AmacaThera doses First In Human Study to Evaluate, AMT-143, for Non-opioid Treatment of Post-operative Pain

AmacaThera doses First In Human Study to Evaluate, AMT-143, for Non-opioid Treatment of Post-operative Pain

May 16, 2024 / Portfolio News

AmacaThera Inc, a clinical stage biotechnology company specializing in the development of advanced sustained release hydrogel formulations, has dosed its first human subject with AMT-143, the company’s lead asset in non-opioid acute pain management.     

AMT-143 is a local slow-release non-opioid local anesthetic that would leverage the AmacaGel platform to provide long-acting post-operative pain relief and is the first therapeutic product formulated to be delivered via AmacaGel, the Company’s patented technology platform.

Previously, the Company had tested the AmacaGel platform without any active pharmaceutical agent.  This trial will test the platform combined with a known anesthetic.  The study will assess the tolerability, safety, pharmacokinetic and clinical activity of the AmacaGel platform with a known compound that has a long history of safety and efficacy.

If approved, AMT-143 could one day be used to reduce post-operative pain without the need for opioids. AMT-143 would be applied in the incisional site at the time of surgery to control local pain.

“A successful outcome will be data demonstrating safety of our asset, and release kinetics showing a sustained release longer than the competitive products,” said Dr. Mike Cooke, Chief Executive Officer, and Co-Founder of AmacaThera, “There is a real need for products that provide longer relief than those currently available on the market.”

Dr. Molly Shoichet is the Company’s Chief Scientific Officer and Co-Founder of AmacaThera. The original technology was developed in her laboratory at the University of Toronto. “My laboratory has demonstrated compatibility of AmacaGel with a wide range of therapeutics – from cells to biologics,” said Shoichet, “With this study, our goal is to provide human data on our technology that will help advance AMT-143 to the clinic. At the same time, we are building out the platform technology so that it can be leveraged for other local-release therapeutics.”

About AmacaThera

AmacaThera is a clinical stage biotechnology company specializing in the development of advanced sustained release hydrogel formulations to solve key therapeutics delivery challenges with both off-patent and proprietary payloads.  AmacaThera is focused on developing an internal pipeline of pain management and oncology assets; and partnering to solve key delivery challenges with advanced proprietary therapeutic modalities.

AmacaThera’s platform technology, AmacaGel, is a fast-gelling physical hydrogel blend of two well-established medical-grade polymers.  It has been designed to liquify under shear force to be delivered by a conventional syringe, rapidly forming a depot at the injection site as it warms to body temperature. AMT-143, the platform’s lead asset, is a slow-release non-opioid local anesthetic that leverages the AmacaGel platform to provide long-acting post-operative pain relief.

For more information, visit www.amacathera.ca

SOURCE AmacaThera Inc.

For further information: Media Contact: Abhaya Khulbe, Investor Relations, info@amacathera.ca

X4 Pharmaceuticals Announces FDA Approval of XOLREMDI™ (mavorixafor) Capsules, First Drug Indicated in Patients with WHIM Syndrome

April 29, 2024 / Portfolio News

X4 Pharmaceuticals (Nasdaq: XFOR), a company driven to improve the lives of people with rare diseases of the immune system, today announced that the U.S. Food and Drug Administration (FDA) has approved XOLREMDI™ (mavorixafor) capsules for use in patients 12 years of age and older with WHIM syndrome (warts, hypogammaglobulinemia, infections and myelokathexis) to increase the number of circulating mature neutrophils and lymphocytes.

XOLREMDI, a selective CXC chemokine receptor 4 (CXCR4) antagonist, is the first therapy specifically indicated in patients with WHIM syndrome, a rare, combined primary immunodeficiency and chronic neutropenic disorder caused by CXCR4 pathway dysfunction. People with WHIM syndrome characteristically have low blood levels of neutrophils (neutropenia) and lymphocytes (lymphopenia) and experience serious and/or frequent infections. The FDA granted Breakthrough Therapy Designation to mavorixafor in WHIM syndrome and evaluated the New Drug Application (NDA) under Priority Review, a designation for therapies that have the potential to provide significant improvement in the treatment, diagnosis, or prevention of serious conditions.

“The approval of XOLREMDI is a transformational milestone both for X4 and, more importantly, for the WHIM syndrome community,” said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals. “We are incredibly grateful to the people living with WHIM syndrome, their families, and the investigators who took part in our clinical program, to U.S. regulators for their continued focus on rare-disease treatment development, and to our dedicated employees for making this targeted breakthrough therapy a reality.”

“Effective and innovative treatments are critical for those diagnosed with a primary immunodeficiency. The approval of XOLREMDI marks an important advancement for people living with WHIM syndrome, who are susceptible to serious and frequent infections,” said Jorey Berry, President and Chief Executive Officer of the Immune Deficiency Foundation (IDF). “We are very pleased to have been a partner to X4 in their journey to bring this much-needed treatment to this underserved rare disease community.”

Teresa K. Tarrant, M.D., Associate Professor of Medicine, Rheumatology, and Immunology at Duke University School of Medicine and a principal investigator in the 4WHIM trial, commented on the news: “Until now, supportive care for people with WHIM syndrome has focused on symptom management and not the underlying cause of disease — the dysfunction of the CXCR4 pathway. I am thrilled that with the approval of XOLREMDI, a therapy designed to address dysregulated CXCR4 pathway signaling, we now have a targeted treatment that has demonstrated the ability to elevate absolute neutrophil and lymphocyte counts, increasing WHIM patients’ ability to fight infections.”

The FDA approval of XOLREMDI was based on results of the pivotal, 4WHIM Phase 3 clinical trial, a global, randomized, double-blind, placebo-controlled, 52-week multicenter study that evaluated the efficacy and safety of XOLREMDI in 31 people aged 12 years and older diagnosed with WHIM syndrome. The efficacy of XOLREMDI was determined by improvement in absolute neutrophil counts (ANC), improvement in absolute lymphocyte counts (ALC), and a reduction in infections. In the 4WHIM trial, XOLREMDI treatment demonstrated increased time above threshold (≥500 cells/microliter) for absolute neutrophil count (TAT-ANC) vs. placebo (p<0.0001) and increased time above threshold (≥1000 cells/microliter) for absolute lymphocyte count (TAT-ALC) v. placebo (p<0.0001). The efficacy of XOLREMDI was further assessed in a composite endpoint consisting of total infection score and total wart change score using a Win-Ratio method. Analyses of the individual components of this composite endpoint showed an approximate 40% reduction in total infection score, weighted by infection severity, in XOLREMDI-treated patients compared with placebo-treated patients. There was no difference in total wart change scores between the XOLREMDI and placebo treatment arms over the 52-week period. Treatment with XOLREMDI also resulted in a 60% reduction in the annualized infection rate compared with placebo-treated patients. The most common adverse reactions reported in the 4WHIM trial (≥10% and more frequently reported than placebo) were: thrombocytopenia, pityriasis, rash, rhinitis, epistaxis, vomiting, and dizziness.

With the FDA approval of XOLREMDI, X4 has received a Rare Pediatric Disease Priority Review Voucher that can be used to obtain priority review for a subsequent application or sold to another drug sponsor.

X4Connect™ Offers Disease and Treatment-Related Support
X4 is committed to helping people with WHIM syndrome access XOLREMDI and announced today the launch of X4Connect, offering eligible U.S. patients dedicated support throughout their XOLREMDI treatment journey, including disease and treatment-related resources, help navigating insurance coverage, and copay assistance. For additional information about X4Connect, call 1-844-X4CNNCT (844-942-6628), Monday-Friday, 8am-8pm ET or visit https://www.xolremdihcp.com/access-and-support.

XOLREMDI will be commercially available in the U.S. through X4’s specialty pharmacy partner PANTHERx® Rare.

Conference Call and Webcast
The company will host a conference call and webcast today at 8:30 am ET. The conference call can be accessed by dialing 1-877-451-6152 from the United States or 1-201-389-0879 internationally, followed by the conference ID: 13746357. The live webcast will be accessible through the investor relations section of X4 Pharmaceuticals’ website at www.x4pharma.com. Following the completion of the call, a webcast replay will be available on the website.

IMPORTANT SAFETY INFORMATION

CONTRAINDICATION

XOLREMDI is contraindicated with drugs highly dependent on CYP2D6 for clearance.

WARNINGS AND PRECAUTIONS

Embryo-Fetal Toxicity: Based on its mechanism of action, XOLREMDI is expected to cause fetal harm. Verify pregnancy status of female patients of reproductive potential prior to starting XOLREMDI. Advise females of reproductive potential to use effective contraception during treatment with XOLREMDI and for three weeks after the final dose.

QTc Interval Prolongation: XOLREMDI causes concentration-dependent QTc prolongation. Correct any modifiable risk factors for QTc prolongation, assess QTc at baseline, and monitor QTc during treatment as clinically indicated in patients with risk factors for QTc prolongation or receiving concomitant medications that increase XOLREMDI exposure and/or drugs with a known potential to prolong the QTc interval. Dose reduction or discontinuation of XOLREMDI may be required.

ADVERSE REACTIONS

The most common adverse reactions (in ≥10% patients and more frequently reported than placebo) were thrombocytopenia, pityriasis, rash, rhinitis, epistaxis, vomiting, and dizziness.

DRUG-DRUG INTERACTIONS

Avoid co-administration of XOLREMDI and strong CYP3A4 inducers. Reduce XOLREMDI daily dosage when administered with strong CYP3A4 inhibitors. Monitor more frequently for adverse reactions associated with an increase in exposure of XOLREMDI when used concomitantly with moderate CYP3A4 inhibitors or P-gp inhibitors and reduce XOLREMDI daily dosage if necessary.

USE IN SPECIFIC POPULATIONS

  • Advise females that breastfeeding is not recommended during treatment with XOLREMDI and for three weeks after the final dose.
  • The safety and effectiveness of XOLREMDI have not been established in pediatric patients younger than 12 years of age.
  • XOLREMDI is not recommended in patients with severe renal impairment, end-stage renal disease, or moderate to severe hepatic impairment.

To report suspected adverse reactions, contact X4 Pharmaceuticals at 1-866-MED-X4MI (1-866-633-9464) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please see the full Prescribing Information for XOLREMDI.

About WHIM Syndrome
WHIM syndrome is a rare, combined primary immunodeficiency and chronic neutropenic disorder caused by CXCR4 receptor dysfunction that results in impaired mobilization of white blood cells from the bone marrow into peripheral circulation. WHIM syndrome is named for its four classic manifestations: warts, hypogammaglobulinemia, infections, and myelokathexis, although only a minority of patients experience all four manifestations in the acronym. People with WHIM syndrome characteristically have low blood levels of neutrophils (neutropenia) and lymphocytes (lymphopenia), and as a result, experience serious and/or frequent infections. It is estimated that at least 1,000 people are currently diagnosed with WHIM syndrome in the U.S.

About XOLREMDI™ (mavorixafor)
XOLREMDI (mavorixafor) is a selective CXCR4 receptor antagonist approved in the U.S. for use in patients 12 years of age and older with WHIM syndrome to increase the number of circulating mature neutrophils and lymphocytes. CXCR4 receptor stimulation by its ligand, CXCL12, has been shown to play a key role in the movement of white blood cells (leukocytes) to and from the bone marrow compartment. Treatment with XOLREMDI results in increased mobilization of neutrophils and lymphocytes from the bone marrow into peripheral circulation.

About X4 Pharmaceuticals
X4 is delivering progress for patients by developing and commercializing innovative therapies for those with rare diseases of the immune system and significant unmet needs. Leveraging our expertise in CXCR4 and immune system biology, we have successfully developed mavorixafor, which has received U.S. approval as XOLREMDI™ (mavorixafor) capsules in its first indication. We are also evaluating the use of mavorixafor in additional potential indications. X4 corporate headquarters are in Boston, Massachusetts and our research center of excellence is in Vienna, Austria. For more information, please visit our website at www.x4pharma.com.

Forward-Looking Statements
This press release contains forward-looking statements within the meaning of applicable securities laws, including the Private Securities Litigation Reform Act of 1995, as amended. These statements may be identified by the words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “target,” or other similar terms or expressions that concern X4’s expectations, strategy, plans, or intentions. Forward-looking statements include, without limitation, implied or express statements regarding X4’s expectations as to the timing of commencement of planned launch, availability, and commercialization of XOLREMDI, which is approved in the U.S. for use in patients 12 years of age and older with WHIM syndrome to increase the number of circulating mature neutrophils and lymphocytes; X4’s plans for commercial launch of XOLREMDI in this indication, including its planned commercial launch in the U.S. through PANTHERx Rare; X4’s belief in its readiness for commercial launch of XOLREMDI; the potential benefit of XOLREMDI in the indicated patient population; the potential number of patients with WHIM syndrome and the potential market for XOLREMDI; the anticipated timing for completion of commercial drug product manufacturing; and the mission and goals for our business.

Any forward-looking statements in this press release are based on management’s current expectations and beliefs. These forward-looking statements are neither promises nor guarantees of future performance, and are subject to a variety of risks and uncertainties, many of which are beyond X4’s control, which could cause actual results to differ materially from those contemplated in these forward-looking statements, including the risks that: X4’s launch and commercialization efforts in the U.S. with respect to XOLREMDI may not be successful, and X4 may be unable to generate revenues at the levels or on the timing we expect or at levels or on the timing necessary to support our goals; the number of patients with WHIM syndrome, the unmet need for additional treatment options, and the potential market for XOLREMDI may be significantly smaller than we expect; XOLREMDI may not achieve the clinical benefit, clinical use, or market acceptance we expect or we may encounter reimbursement-related or other market-related issues that impact the success of our commercialization efforts; we may encounter adverse events for XOLREMDI at any stage that negatively impact commercialization; the need to align with our collaborators may hamper or delay our development and commercialization efforts or increase our costs; our business may be adversely affected and our costs may increase if any of our key collaborators fails to perform its obligations or terminates our collaboration; the internal and external costs required for our ongoing and planned activities, and the resulting impact on expense and use of cash, may be higher than expected, which may cause us to use cash more quickly than we expect or to change or curtail some of our plans or both; and other risks and uncertainties, including those described in the section entitled “Risk Factors” in X4’s Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC) on March 21, 2024, and in other filings X4 makes with the SEC from time to time. X4 undertakes no obligation to update the information contained in this press release to reflect new events or circumstances, except as required by law.

Investor Contact:
Daniel Ferry
Managing Director, LifeSci Advisors
daniel@lifesciadvisors.com
(617) 430-7576

Media Contact:
Kit Rodophele
Account Director, Ten Bridge Communications
krodophele@tenbridgecommunications.com

Charting A New Course For Chronic Cough Treatment

April 23, 2024 / New Investments

Chronic cough, particularly when it extends beyond 8 weeks into what is known as refractory chronic cough (RCC), represents a substantial health challenge. With its prevalence globally ranging from 2% to 18%, RCC places a significant burden on individuals and healthcare systems due to its profound physical and psychological impact on patients. Of the currently recommended treatments, most exhibit limited evidence, and many are used off-label, do not address the underlying pathology, have limited efficacy, and produce significant side-effects.

This highlights a critical unmet need for innovative therapeutic strategies that target the underlying mechanisms of RCC. Underlying the importance of supporting innovation in this space, as it is not only essential for developing effective treatments but also for advancing our understanding of RCC and similar respiratory ailments. Potentially leading to breakthroughs that could transform patient care and improve quality of life for millions.

Nocion Therapeutics is uniquely positioned to develop new treatments for chronic cough patients due to its innovative approach and targeted technology. The company is focusing on developing first and potentially best-in-class, small molecule, charged sodium channel blockers (CSCBs), known as “nocions”. These innovative compounds are designed to selectively silence activated nociceptors that are involved in the transmission of cough, itch, and pain signals.

Their lead program, Taplucanium Dry Powder for Inhalation, is currently being evaluated in a Phase 2b study for patients with chronic cough. This proprietary molecule represents a new class of CSCBs that allows for the specific silencing of activated/inflamed nociceptors. Unlike other investigational cough therapies, such as P2X3 antagonists which target a specific receptor, Taplucanium operates through any open Large Pore Channel (LPC) to inhibit the sodium channels responsible for initiating the pathological cough response. As broad sodium channel inhibitors are already used off-label for pulmonary irritation applications, Taplucanium could expand the clinical benefit as a potential new treatment option for chronic cough.

With a highly differentiated mechanism of action, Nocion presents a great opportunity to leverage recent learnings from chronic cough clinical studies, potentially offering a new therapeutic strategy that could benefit a broader population of chronic cough patients.

“Building upon decades of research of its esteemed scientific founders and long history of clinical experience with broad voltage-gated sodium channel inhibitors, we believe Nocion’ lead program and emerging products from its nociceptor-focused platform is well positioned to provide broad impact across several inflammatory diseases starting with chronic cough” stated Lu Han, Ph.D., Partner, Lumira Ventures. “We are proud to support the mission of the Nocion team alongside a distinguished group of co-investors to develop innovative treatments targeting major unmet needs.”

Building a Potential Cure for Type 1 Diabetes

April 22, 2024 / New Investments

Over 300K Canadians, +1.5M Americans, and +9M individuals globally are living with Type 1 Diabetes. Although a seemingly difficult-to-treat disease, over the last several years there has been a strong wave of innovation and renewed interest in the space from big pharma and investors alike. For Lumira, we see the Type 1 diabetes space as an opportunity to address various components of the disease (improving current insulin regimens, delaying/preventing the destruction of beta cells, replacing beta cells, and regenerating/restoring beta cells) to develop a functional cure within the next 15 years. As a result of our these, Lumira has been proactively engaged in an in-depth evaluation of investment opportunities in the Type 1 Diabetes space for the last two and half years.

COUR Pharmaceuticals was identified through our proactive therapeutic area search process as a company with a compelling technology for the potential delay/prevention of the disease. Late in 2022, the drug TZIELD developed by Provention Bio was approved by the FDA establishing a path to market for drugs in this category, however it is widely recognized that this product will be difficult to access for most patients due to its narrow label, challenging dosing regimen, and immune exhaustive effect which leaves patients vulnerable to infectious diseases after chronic treatment.

The search for a more patient-friendly, scalable, and targeted approach led us to COUR’s antigen-specific tolerization platform, a modality that Lumira has followed for over a decade with broad potential across various autoimmune conditions. The company has developed a nanoparticle that tolerizes the immune system to any antigens it is loaded with, and any autoimmune disease where the disease-causing antigen(s) is known would therefore be a potential area to pursue. The company has generated strong proof-of-clinical concept data in celiac disease through its partnership with Takeda Pharmaceuticals. We believe the approach would be effective in the two lead programs that the company is pursuing – Type 1 Diabetes and Myasthenia Gravis – as well as other autoimmune conditions.

“There is an urgent need for innovative strategies aimed at restoring self-tolerance safely and more effectively in autoimmune disorders. We believe COUR’s proprietary and strongly differentiated platform is a versatile and first-in-class approach to meet this critical medical need. We are dedicated to collaborating with COUR and its partners to help advance these potentially cutting-edge treatments for immune-related diseases.” Benjamin “Beni” Rovinski, Ph.D., Managing Director, Lumira Ventures

After months of in-depth diligence and strategic structuring, we were ultimately able to bring on a strong group of investors to participate, and have created strong competitive dynamics by having around the table six strategic investors. Make no mistake, deals in these markets are hard and take longer to get done, but we believe our significant efforts will be worth the eventual return.

In January 2024, COUR Pharmaceuticals announced the closing of a $105M Series A financing, co-lead by Lumira Ventures and Alpha Wave Ventures, with participation from Roche Venture Fund, Pfizer (as part of the Pfizer Breakthrough Growth Initiative), Bristol Myers Squibb, Angelini Ventures, and the JDRF T1D Fund. This deal signifies (according to Pitchbook metrics); The largest private healthcare deal ever led by a Canadian-headquartered VC, the second largest private deal led by a Canadian-headquartered VC in 2023 across any sector (not just healthcare), the largest private healthcare deal in the US Midwest in 2023 and Lumira’s largest deal as a lead investor.

 

Lumira Ventures Announces Investment in Nocion Therapeutics to Revolutionize Chronic Cough Treatment

April 2, 2024 / Portfolio News

TORONTO, ON / ACCESSWIRE / APRIL 2, 2024 / Lumira Ventures is delighted to announce its latest investment in Nocion Therapeutics, a clinical-stage biopharmaceutical company developing first and potentially best-in-class, small molecule charged sodium channel blockers (CSCBs), called Nocions. These innovative compounds are designed to selectively silence activated nociceptors for the treatment of serious conditions involving cough, itch, and pain. Proceeds from the financing will be used to evaluate the lead program, Taplucanium Dry Powder for Inhalation, in a Phase 2b study in Chronic Cough patients.

“With a highly differentiated mechanism of action delivered via a targeted approach, we are extremely excited about Taplucanium as a potential new treatment modality for refractory chronic cough patients with very limited options today.” Said Lu Han, Ph.D., Partner, Lumira Ventures.

The Series B financing was led by Arkin Bio Capital and Monograph Capital, with participation from additional new investors as well as existing investors. Lumira Ventures is proud to support Nocion Therapeutics alongside this distinguished group of syndicates including a strategic investor with deep domain expertise in inhaled respiratory therapeutic drug development.

Benjamin “Beni” Rovinski, Ph.D., Managing Director at Lumira Ventures added, “With this financing, and together with a broad syndicate of sophisticated investors, we look forward to supporting a strong management team with deep domain expertise in the respiratory field to develop innovative treatments targeting major unmet needs.”

In conjunction with the Series B investment, Lu Han, Ph.D., of Lumira Ventures will join Nocion’s Board as an observer.

“We are excited to have Lumira join our Series B syndicate. Their participation, along with the rest of the syndicate, closes our Series B raise of $70M” said Rick Batycky, Ph.D., CEO of Nocion Therapeutics. “This robust raise will allow for a comprehensive evaluation of Taplucanium in Chronic Cough patients, and completion of additional activities to advance this program to Phase 3 readiness, as we diligently work to get this potentially transformative treatment to patient in a timely fashion.”

For further information, please contact:

Rick Batycky

rick@nociontx.com

About Lumira Ventures:

Founded in 2007, Lumira Ventures is a multi-stage life sciences investment firm with offices in Toronto, Montréal, Vancouver, and Boston. We partner with mission-driven entrepreneurs building companies that harness rapidly evolving advancements in medicine to develop transformative products for patients while delivering strong financial returns for our investors and meaningful economic value and impact for society. For more information, visit www.lumiraventures.com

About Nocion Therapeutics:

Nocion Therapeutics is a biopharmaceutical company developing novel small molecule CSCBs, “nocions,” that selectively affect actively firing nociceptors for the treatment of serious conditions involving cough, itch, and pain. The company’s mission is to safely alleviate suffering for millions of patients with conditions arising from activated sensory neurons. Working with Harvard’s Office of Technology Development, Nocion was founded on an exclusive license to foundational intellectual property from Harvard University and Boston Children’s Hospital. For more information, visit: www.nociontx.com.

Fusion Pharmaceuticals to be Acquired by AstraZeneca, Accelerating Development of Next-Generation Radioconjugates to Treat Cancer

March 19, 2024 / Portfolio News

HAMILTON, ON and BOSTON, March 19, 2024 /PRNewswire/ — Fusion Pharmaceuticals Inc. (Nasdaq: FUSN), a clinical-stage oncology company focused on developing next-generation radioconjugates (RCs) as precision medicines, today announced the Company has entered into a definitive agreement to be acquired by AstraZeneca. The acquisition marks a major step forward in AstraZeneca delivering on its ambition to transform cancer treatment and outcomes for patients by replacing traditional regimens like chemotherapy and radiotherapy with more targeted treatments.

RCs have emerged as a promising modality in cancer treatment over recent years. These medicines deliver a radioactive isotope directly to cancer cells through precise targeting using molecules such as antibodies, peptides or small molecules. This approach has many potential advantages compared to traditional radiotherapy, including minimizing damage to healthy cells and enabling access to tumors not reachable through external beam radiation.

This acquisition complements AstraZeneca’s leading oncology portfolio with the addition of the Fusion pipeline of RCs, including the Company’s most advanced program, FPI-2265, a potential new treatment for patients with metastatic castration-resistant prostate cancer (mCRPC). FPI-2265 targets prostate-specific membrane antigen (PSMA), a protein that is highly expressed in mCRPC, and is currently in a Phase 2 trial.

The acquisition brings new expertise and pioneering R&D, manufacturing and supply chain capabilities in actinium-based RCs to AstraZeneca. It also strengthens their presence in and commitment to Canada.

Fusion Chief Executive Officer John Valliant, Ph.D., said: “This acquisition combines Fusion’s expertise and capabilities in radioconjugates, including our industry-leading radiopharmaceutical R&D, pipeline, manufacturing and actinium-225 supply chain, with AstraZeneca’s leadership in small molecules and biologics engineering to develop novel radioconjugates. Expanding on our existing collaboration with AstraZeneca where we have advanced FPI-2068, an EGFR-cMET targeted radioconjugate into Phase I clinical trials, gives us a unique opportunity to accelerate the development of next-generation radioconjugates with the aim of transforming patient outcomes.”

Susan Galbraith, Executive Vice President, Oncology R&D, AstraZeneca, said: “Between thirty and fifty per cent of patients with cancer today receive radiotherapy at some point during treatment, and the acquisition of Fusion furthers our ambition to transform this aspect of care with next-generation radioconjugates. Together with Fusion, we have an opportunity to accelerate the development of FPI-2265 as a potential new treatment for prostate cancer, and to harness their innovative actinium-based platform to develop radioconjugates as foundational regimens.”

Fusion President and Chief Business Officer Mohit Rawat, said: “Fusion has differentiated itself in the growing radioconjugate space by assembling an industry-leading team with deep expertise and infrastructure to support bringing these much-needed therapies to cancer patients. Together we look forward to building upon our work to impact the landscape of cancer therapy. Deepening our collaboration with AstraZeneca presents an exciting opportunity for the Fusion team.”

Fusion will become a wholly owned subsidiary of AstraZeneca, with operations continuing in Canada and the US.

Financial Considerations

Under the terms of the definitive agreement, AstraZeneca, through a subsidiary, will acquire all of Fusion’s outstanding shares pursuant to a plan of arrangement for a price of $21.00 per share in cash at closing plus a non-transferable contingent value right (CVR) of $3.00 per share in cash payable upon the achievement of a specified regulatory milestone.

The upfront cash portion of the consideration represents a transaction value of approximately $2 billion, a 97% premium to Fusion’s closing market price of $10.64 on March 18, 2024, and an 85% premium to the 30-day volume-weighted average price (VWAP) of $11.37 before this announcement. Combined, the upfront and maximum potential contingent value payments represent, if achieved, a transaction value of approximately $2.4 billion, a 126% premium to Fusion’s closing market price on March 18, 2024, and a 111% premium to the 30-day VWAP. As part of the transaction, AstraZeneca will acquire the cash, cash equivalents and short-term investments on Fusion’s balance sheet, which totaled $234 million as of December 31, 2023.

The proposed acquisition of Fusion is to be completed by way of a statutory plan of arrangement under the Canada Business Corporations Act and subject to customary closing conditions, including approval of (i) 66⅔% of the votes cast by Fusion shareholders and (ii) a simple majority of the votes cast by Fusion shareholders (excluding certain persons required to be excluded in accordance with Multilateral Instrument 61-101 of the Canadian Securities Administrators), in each case, at a special meeting of Fusion shareholders. The transaction is expected to close in the second quarter of 2024, subject to customary closing conditions, including the approval of Fusion shareholders and regulatory clearances, as noted above.

Centerview Partners LLC is serving as exclusive financial advisor to Fusion and Goodwin Procter LLP is serving as legal counsel, with Osler, Hoskin & Harcourt LLP serving as Canadian legal counsel.

Radioconjugates in oncology
RCs combine the precise targeting of antibodies, small molecules or peptides with potent medical radioisotopes to deliver radiation directly to cancer cells. By seeking out cancer cells, RCs provide a more precise mechanism of cancer cell killing compared with traditional radiation therapy, with the goal of improving efficacy while minimizing toxicity on healthy cells. RCs are administered via systemic delivery, which enables their use in tumor types not accessible to external beam radiation and the targeting of cancer cells that have spread from the main tumor to other sites in the body.

About FPI-2265
FPI-2265 is an actinium-225 based PSMA targeting RC, for mCRPC, currently in a Phase II trial. Actinium-225 emits alpha particles and holds the promise of being a next-generation radioisotope in cancer treatment. By delivering a greater radiation dose over a shorter distance, alpha particles such as actinium-225 have the potential for more potent cancer cell killing, and targeted delivery, thereby minimizing damage to surrounding healthy tissue.

About Fusion 
Fusion Pharmaceuticals is a clinical-stage oncology company focused on developing next-generation RCs. Fusion connects alpha particle emitting isotopes to various targeting molecules in order to selectively deliver the alpha emitting payloads to tumors. Fusion’s clinical-stage development portfolio includes lead program, FPI-2265, targeting PSMA for mCRPC and novel RCs targeting solid tumors.

Cautionary Note Regarding Forward-Looking Statements

To the extent any statements made in this communication contain information that is not historical, these statements are forward-looking statements within the meaning of Section 27A of the U.S. Securities Act of 1933, as amended, and Section 21E of the U.S. Securities Exchange Act of 1934, as amended, and forward-looking information under Canadian securities law (collectively, “forward-looking statements”). Certain statements in this communication may constitute forward-looking statements, which reflect the expectations of Fusion’s management regarding the business prospects and opportunities of Fusion and the proposed transaction. The use of words such as “may,” “will,” “could,” “should,” “expects,” “intends,” “plans,” “anticipates,” “believes,” “estimates,” “predicts,” “projects,” “seeks,” “endeavor,” “potential,” “continue” or the negative of such words or other similar expressions can be used to identify forward-looking statements. More particularly and without limitation, this communication contains forward-looking statements and information regarding whether the proposed transaction will be completed, whether the CVR Agreement will be entered into, the anticipated benefits of the proposed transaction for Fusion and its Shareholders, whether approval will be received under the U.S. Hart-Scott-Rodino Antitrust Improvements Act of 1976 and the Competition Act (Canada), each as amended, and whether the milestone under the CVR Agreement will be achieved.

Fusion’s actual results could differ materially from those stated or implied in forward-looking statements due to a number of factors including but not limited to risks related to the satisfaction or waiver of the conditions to closing the proposed transaction (including the failure to obtain necessary regulatory, court and Fusion’s shareholder approvals) in the anticipated timeframe or at all, including the possibility that the proposed transaction does not close; the response of business partners and competitors to the announcement of the proposed transaction, and/or potential difficulties in employee retention as a result of the announcement and pendency of the proposed transaction; significant transaction costs; the failure to realize the expected benefits of the proposed transaction; risks associated with the disruption of management’s attention from ongoing business operations due to the proposed transaction; and unknown liabilities and the risk of litigation and/or regulatory actions related to the proposed transaction. Please also refer to the factors discussed under “Risk Factors” and “Special Note Regarding Forward-looking Information” in Fusion’s Annual Report on Form 10-K for the year ended December 31, 2022, with the U.S. Securities Exchange Commission (“SEC”), each as updated by Fusion’s continuous disclosure filings, which are available at www.sec.gov and at www.sedarplus.com.

Forward-looking statements involve significant risks and uncertainties, should not be read as guarantees of future performance or results, and will not necessarily be accurate indications of whether or not or the times at or by which such performance or results will be achieved. All forward-looking statements herein are qualified in their entirety by its cautionary statement and are made as of the date of this document. Fusion disclaims any obligation to revise or update any such forward-looking statements or to publicly announce the result of any revisions to any of the forward-looking statements contained herein to reflect future results, events or developments, except as required by law.

Additional Information about the Proposed Transaction and Where to Find It

This communication is not intended to and does not constitute an offer to sell or the solicitation of an offer to subscribe for or buy or an invitation to purchase or subscribe for any securities or the solicitation of any vote, consent or approval in any jurisdiction, nor shall there by any sale, issuance or transfer of securities in any jurisdiction in contravention of applicable law. This communication has been prepared in respect of the transaction involving Fusion, AstraZeneca and Purchaser (as defined in the Arrangement Agreement) pursuant to the terms of the Arrangement Agreement, and may be deemed to be soliciting material relating to the transaction. In connection with the transaction, Fusion will file a management information circular and proxy statement on Schedule 14A relating to a special meeting of the shareholders with the SEC and Canadian Securities Administrators (“CSA”). Additionally, Fusion will file other relevant materials in connection with the transaction with the SEC and the CSA. Shareholders of Fusion are urged to read the management information circular and proxy statement and/or consent solicitation documents regarding the transaction and any other relevant materials carefully in their entirety when they become available before making any voting or investment decision with respect to the transaction because they will contain important information about the transaction and the parties to the Arrangement Agreement. The definitive management information circular and proxy statement will be mailed to holders of Fusion’s shares. Shareholders will be able to obtain a copy of the management information circular and proxy statement, as well as other filings containing information about the transaction and the parties to the Arrangement Agreement made by Fusion with the SEC and CSA free of charge on EDGAR at www.sec.gov, on SEDAR+ at www.sedarplus.com, or on Fusion’s website at www.fusionpharma.com. Information contained on, or that may be accessed through, the websites referenced in this communication is not incorporated into and does not constitute a part of this document. We have included these website addresses only as inactive textual references and do not intend them to be active links.

Participants in the Solicitation

Fusion and its directors and executive officers may be deemed to be participants in the solicitation of proxies from the shareholders of Fusion in respect of the transaction. Information about Fusion’s directors and executive officers is set forth in the proxy statement and management information circular for Fusion’s Annual General Meeting of Shareholders, which was filed with the SEC and CSA on April 27, 2023. Investors may obtain additional information regarding the interest of such participants by reading the management information circular and proxy statement regarding the proposed transaction when it becomes available.

Contact:
Amanda Cray
Senior Director of Investor Relations & Corporate Communications
(617) 967-0207
cray@fusionpharma.com

SOURCE Fusion Pharmaceuticals

Lumira Ventures Celebrates Team Promotions, Emphasizes Growth and Development and Honors Female Contributions Ahead of International Women’s Day

March 7, 2024 / Portfolio News

TORONTO, ON / ACCESSWIRE / March 7, 2024 / Lumira Ventures, a life sciences investment firm dedicated to supporting healthcare innovation and patient impact, is proud to announce several significant advancements and promotions within its investment team. “As we commemorate International Women’s Day on March 8th, 2024, it is crucial to recognize the invaluable contributions of women in the workforce,” said Peter van der Velden, Managing Director at Lumira Ventures. “Lumira was among the first VC firms in Canada with a female partner and these team promotions along with the recent new addition to our team showcase the indispensable contributions and influence of women in leadership roles shaping the professional landscape within venture capital.”

Lumira Ventures commends the dedication and accomplishments of three deserving employees who have been promoted to elevated leadership positions. These promotions underscore Lumira’s commitment to nurturing and empowering talent from within our organization. The following team members, all talented women, have been promoted to positions of new responsibility within the firm.

Suman Rao, Ph.D. has been promoted to Senior Associate. Suman has continuously demonstrated strong leadership and strategic insight since joining the Lumira Ventures investment team in 2021. In her new role, she will take on additional responsibilities for sourcing, evaluating and closing new investments and serving on the boards of Lumira portfolio companies. As well she will continue being a core member of the team developing and promoting new strategies with our partner Angelini Ventures to pursue the growing opportunity we see from our investment approach in Europe.

Baye Galligan, MA, HBA, has been promoted to Director of Research. Since joining Lumira in 2017, Baye has been an integral part of our investment team. In 2021 she became one of the internal leads on supporting our Venture Innovation Program, which mentors current Ph.D., M.D. or MBA students through a 6-12 month in-house fellowship in venture capital and entrepreneurship. As this program expanded, and as Lumira continued to deepen its pro-active internal research, it presented a perfect opportunity for Baye to take the lead with respect to prioritizing and developing the research to support new investment themes and portfolio company value creation through the application of traditional and AI-enabled research tools.

Alyssia Watkin has been promoted to Director of Communications and Investor Relations. After joining Lumira first as an intern and then as our first dedicated internal marketing resource in 2021, Alyssia has played a pivotal role in transforming our marketing and communications functions in support of our investor community and external stakeholders. In her new position, she will take on additional responsibilities related to the firm’s fundraising initiatives and limited partner relationship management, as well as coordinating Lumira’s communications initiatives supporting our portfolio companies.

Along with these promotions, Lumira Ventures welcomes the new addition of:

Isabelle Harris, HBA to our team, as an Analyst. Isabelle brings a rich background in life sciences and strategy consulting to the Lumira team. She holds a B.S in genetics from Western University and an HBA from Ivey Business School. In her new role, she will collaborate with the investment team and support the evaluation of new investment opportunities, utilizing her understanding of industry dynamics and analytical acumen to help drive investment decisions across our various investment funds.

Lumira Ventures is excited to have Isabel join the team and looks forward to the leadership of the newly promoted team members. Their diverse backgrounds, distinctive perspectives, and commitment to excellence embody the essence of International Women’s Day. As we join the world in celebrating this important day, Lumira Ventures reaffirms its dedication to fostering gender equality, empowerment, and an inclusive workplace where everyone has the opportunity to flourish, not just today, but every day.

For more information, please visit www.lumiraventures.com

Media Inquiries: info@lumira.vc

www.lumiraventures.com

About Lumira Ventures

Founded in 2007, Lumira Ventures is a multi-stage life sciences investment firm with offices in Toronto, Montréal, Vancouver, and Boston. We partner with mission-driven entrepreneurs building companies that harness rapidly evolving advancements in medicine to develop transformative products for patients while delivering strong financial returns for our investors and meaningful economic value and impact for society.

SOURCE: Lumira Ventures

Reforming Interventional Cardiology

February 22, 2024 / Success Stories

History: Established in 2003, OpSens’ technology was originally used in oil and gas, some industrial applications, and by some medical device manufacturers to measure temperature, pressure, and displacement. Their success in these industries well established their reputation as an expert in measurements using sensor technology. However, the team recognized a greater potential for such uniquely effective systems and their ability to do more at a larger and more medically impactful scale, by focusing on cardiology applications. In 2009, OpSens Medical expanded the business strategy into the global fractional flow reserve (FFR) market. Directly shifting their focus to providing resolutions to cardiologists’ needs for more sophisticated measurement, document, and evaluation instruments for their work and their patients.

Our team had built a relationship with the OpSens team from the inception and although hesitant of the initial indications, we saw the potential to benefit patients and continued to monitor the expansion. Following the logical turn to bring pressure guidewires to market for cardiac care and recognizing the exceptional progress made to achieve this, our support in 2016 was undeniable. Our investment in OpSens Medical was a distinctive opportunity to reinvent the course of care for coronary disease and bring best-in-class solutions that offer better outcomes to healthcare systems and patient lives. Coronary artery disease is the most common type of heart disease and a leading cause of death in North America. OpSens versatile technology continues to outperform the standards of care and provide greater resolutions to many critical clinical limitations. As the company continues to make exceptional progress, we are thrilled to be a part of bringing forward the new gold standard of interventional cardiology care.

Solution: The OptoWire™, OPTOMONITOR™ AND SAVVYWIRE™

OpSens OptoWire - Opsens Medical

The OptoWire™ is an advanced pressure guidewire powered by Fidela, a 2nd generation fiber optic sensor. Allowing physicians to more precisely measure the Fractional Flow Reserve (FFR) and diastolic Pressure Ratio (dPR), to determine the severity of coronary artery blockage. The design and performance mimic a workhorse guidewire. With a small structure and distinct engineering properties, the resistance, flexibility, and sensitivity is enhanced. Physicians are able to use a single device and obtain multiple measurements across complex structures with high confidence in the accuracy of results. The wire durability also allows it to perform stenting and b

alloon angioplasty without the need to replace the wire and lose accuracy. With disconnect and reconnect capabilities, physicians can easily measure and adapt to different anatomies, perform appropriate interventions, and confirm improvements following stent implantation.

OpSens completes European enrolment in SAFE-TAVI study

The SavvyWire™ is the first and only 3-in-1 sensor-guided solution for Transcatheter Aortic-Valve Replacement (TAVR) procedures. Engineered to enhance TAVR efficiency while improving lifetime patient management. The device supports stable aortic valve delivery and positioning with continuous hemodynamic measurements during the procedure, and reliable ventricular pacing without the need for adjunct devices or venous access.

The OptoMonitor™ system, a paired smart integration tool, enables convenient measurement management directly at the tableside or integrated into monitoring systems. Physicians can immediately measure pressure in the artery after blockage and compare it instantaneously with measurements prior. Generating faster, more actionable diagnoses and decisions, allowing patients to go home without suffering from symptoms.

Generating Impact: In various clinical studies, pressure guidewires have proven substantial impacts on patients and clinical practice:

Clinical-Studies

 

Today, the Optowire is the gold standard of care, having been used in the diagnosis and treatment of

Diagnosis-Treatment

 

This rapid adoption has led to the installation of more than 2,000 OptoMonitor units in clinics, and growing use by clinical partners to improve their success rates in challenging coronary interventions. The Optowire is currently approved for sale in the United States, European Union, Japan, and Canada, with channels for sales globally.

OpSens’ demonstrated patient impact does not stop here. The company now intends to capitalize on its expertise in coronary artery disease to extend its activities to structural cardiology, with the SavvyWire. With a global TAVR market of over 200,000 procedures and an expectation to reach 400,000 in 2027, the SavvyWire is on a path for enormous and expansive patient impact. To date, the SavvyWire has been cleared by Health Canada and U.S. FDA and completed procedures in the U.S, Europe, while most recently extending pacific coast presence in New Zealand.

Cutting-Edge Cardiac Care to Save Lives

February 22, 2024 / Success Stories

History: For 27 years, Lorene Bardy, the late wife of Bardy Diagnostics founder Gust Bardy, M.D., had a rare heart rhythm disorder. Lorene had been seen by many expert electrophysiologists, but the lack of ability to visualize and record her P wave left her without answers to her specific type of arrhythmia. At the young age of 52, Lorene suddenly passed by cardiac arrest, which Dr. Bardy attributes to the suboptimal state of diagnostic technologies. As a practicing electrophysiologist since 1983 and motivated by the experiences of his late wife, Dr. Bardy began his relentless mission to take the many unmet medical needs in cardiac care and turn it into a tangible and life-changing medical solution. In 2013, Bardy Diagnostics was formed with the unique perspective of both physician and husband.  Dr. Bardy used his expertise to develop transformative cardiac monitoring solutions that help physicians obtain more accurate information about actionable events in the hopes of saving lives.

Solution: The Carnation Ambulatory Monitor (CAM™)

Bardy Carnation Ambulatory Monitor Patch Decreases Diagnosis Time and Readmissions | DAICThe Carnation Ambulatory Monitor (CAM™) is the first non-invasive, P-wave centric ambulatory cardiac monitor and arrhythmia detection device designed to elevate signal detection, patient compliance, and physician care procedures. Through advanced compression algorithms to more effectively process signals, and short patch vectors to overcome signal-to-noise limitations, the technology is rigorously optimized to ensure a clear P-wave recording and superior detection of heart rhythms. The form and placement of the device are designed to improve comfort for patients, while generating higher rhythm specificity. The sensor is placed over the sternum, on top of the heart, this location prevents any noisy muscle stimulation and enhances the capture of the P-wave signal.

The hourglass form enables comfort in this area and was thoughtfully designed to resolve theAmbulatory ECG Monitoring Solutions Made Simple | Bardy Diagnostics underserved needs of comfort for the female chest. With a lifestyle-enabling form factor patients can remain going about their daily activities comfortably and confidently. CAM™ provides options for a 48-hour, 7- or 14-day wear period and continuous 24/7 monitoring using BDxCONNECT, a versatile patient management portal. When experiencing symptoms, patients simply push the button on the device, and a report is sent to their physician to analyze and determine if intervention is needed. Once the wear period is finished, the data is uploaded and a more in-depth view of ECG data is generated through multiple fields of concise analysis. In an industry-leading two-day report turn-around, physicians have an improved resolution of ECG data, supplying more heart rhythm information and allowing for the detection of many unrecognized patterns and more clinically-actionable diagnoses.

Generating Impact: The CAM patch is rapidly becoming the new standard of care in cardiac monitoring and the trusted cardiac monitor of choice by electrophysiologists, cardiologists, and physicians globally. The 14-day CAM patch in addition to the 2- and 7-day CAM patches are all commercially available and cleared by the FDA, actively being used to improve cardiac care across the U.K., U.S., and Canada. The commercialization of the product and strong clinical evidence are clear indicators of the significance of the technologies impact. With further development and clinic expansions, it is evident the improvements Bardy’s technology will continue to make on greater arrhythmia detection and diagnosis for patients around the world.

The life-saving potential of Bardy’s technology continues to evolve and benefit patients in different avenues. The CAM patch currently extending its use in newly established protocols at hospitals across Seattle, WA, to monitor cardiac rhythms for COVID-19 patients on outpatients on Hydroxychloroquine (HCQ). HCQ has been demonstrated to prolong the QT interval, resulting in a potentially lethal arrhythmia called Torsades de Pointes. The unique ability of the BardyDx CAM Patch to detect and record low frequency, low amplitude cardiac rhythms will be especially valuable in measuring QT intervals and diagnosing Torsades de Pointes.

To date, the CAM patch has been used to monitor 40,000 + patients 

and acceptance of the product continues to rapidly grow as a result of continuously positive patient and physician reception of the unique technology. Promising clinical evidence and peer-reviewed data have additionally demonstrated the advantageous weight of the CAM patch and exemplified a dominant superiority over existing monitors to provide a greater impact on clinical decisions, patient comfort, and arrhythmia diagnosis.

96%
34% and 40%
4X more clinically

 

Advancing Next-Gen Gene Editing to Combat Genetic Diseases

February 22, 2024 / New Investments

Gene editing holds incredible potential to cure a wide range of genetic diseases by directly altering DNA in the genome inside cells. Yet, today’s gene editors remain facing many limitations. “Current gene editing technologies are constrained by low on-target editing efficiency and high unintended off-target effects,” noted Dr. Daniel Hetu, Managing Director at Lumira Ventures. The team at Specific Biologics is shifting this paradigm. Through the development of their novel gene editing therapies, the company is breaking down barriers to better realize the significant opportunity of this therapeutic approach and optimize its application across new therapeutic areas. “Data suggest that Dualase™ has the potential to overcome these well-known challenges in relevant therapeutic indications,” mentioned Dr. Hetu.

Specific Biologics, an early-stage biotechnology company has developed a unique next-generation Dualase™ gene editing platform that can more accurately alter DNA sequences by cutting DNA differently than current editors. The platform gene editing technology cuts DNA twice and leaves non-compatible DNA ends. Other gene editors leave compatible DNA ends when they cut DNA. When a cell repairs compatible ends, it leads to random insertions or deletions or a low frequency of repair, reducing efficiency and target effects. Dualase™ by creating non-compatible ends influences cellular repair and depending on the presence or absence of template DNA, the cell may introduce defined length deletions or increase the frequency of repair. This step is critical to the company’s mission as defined length deletions & increased repair allow targeting of genetic diseases that could not be targeted before.

“Based on our early work with Dualase™, we believe it will have broad utility across a number of diseases with high unmet need,” said Dr. Brent Stead, CEO of Specific Biologics Inc. “This investment by Lumira and adMare, two recognized leaders in life sciences, will help accelerate the advancement of our promising platform, and a pipeline of differentiated Dualase™-based gene editing therapeutics in a diverse range of indications.”

Initial indications for Dualase™ were set to target a set of cystic fibrosis-causing nonsense mutations where patients currently have fewer therapeutic options available. By delivering the gene editing technology using an inhalable lipid nanoparticle carrier, the treatment will aid in delivering the gene editing machinery inside the cells of the lung. Following, if the CFTR nonsense mutations are corrected in enough cells, the gene editing therapeutic will provide a more effective treatment option with long-lasting benefits for CF patients.

The company is on a mission and it does not stop at cystic fibrosis. The promising breadth of the platform and widespread indications allow the company to expand its research and build a pipeline of therapeutics based on Dualase, offering hope to treat many other genetic diseases with no current therapies.

“Built on the work of Dr. David Edgell at Western University’s Schulich School of Medicine & Dentistry, Specific Biologics represents another example of how Canadian health science innovators are at the leading edge of treatment modalities,” added Nikhil Thatte, Principal at Lumira Ventures. “We are excited to work alongside adMare and the Specific team to grow the company and bring transformative therapies to patients.”

Bridging The Gap: A Platform to Address Cognitive Deficits

February 22, 2024 / New Investments

damona-pharma-2Cognitive deficits are the hallmark of a variety of psychiatric and neurodegenerative disorders affecting millions of patients worldwide.   These conditions include; major depressive disorder (MDD), schizophrenia, a number of neurodegenerative disorders including Alzheimer’s Disease, and aging in general.  Treatment options are few and far between and in many cases no treatment options are available to address cognitive deficits across disorders which often leave patients, care givers and family members struggling.  At the forefront of research in the area is Dr Etienne Sibille and his lab at the Centre for Mental Health and Addiction (CAMH) at the University of Toronto, a world leading academic research institute.  Dr. Sibille and his team has been studying how brain circuitry is affected and if there are ways in which the connections between neurons in vital areas responsible for cognition can be strengthened and as we age.

The brain processes reams of information and has mechanisms to put the brakes on much of the stimuli available so as to ensure that the brain does not to become overwhelmed by inputs that can drown out what is important.  In the early 2010’s Dr. Sibille’s lab discovered that some of the circuitry responsible for putting a break on inputs was affected in human tissues of aging individuals and patients with a number of cognitive issues.  The team went on to show that these same circuits were affected in rodents models of disease and aging.  These insight became the foundation of a number of publications and start of a focused process to determine what could be done to reverse the change in the brains circuitry.

Through collaborations with other world renowned groups DAMONA-Pharma Dr. Sibille’s group embarked on a journey to design a drug that could reverse the pathology that they discovered and hypothesized was leading to cognitive decline in patients.  By the summer of 2017, when we first met Dr. Sibille and his associate Dr. Prevost, — they, CAMH and international collaborators had filed patents on a class of compounds that showed promise in laboratory studies to address this issue.  It was easy for us to be engaged by the vision, need and opportunity.  While the team had initially identified a group of novel compounds and very interesting biology, translating that into a drug was still far off.  From 2017 to 2021, through hard work, tenacity, constant collaboration with other drug developers in the field, leveraging connections through various local and international accelerator programs, VC’s like us, clinicians, etc. the team was able to identify a lead drug that had all the characteristics needed.  They also learned the art of approaching and working with investors, contract organizations that do much of the preclinical work, the various regulatory bodies like FDA, and clinicians experienced running clinical studies.  Through this process the company settled on a program to first address the huge unmet clinical need in patients that respond to treatment of MDD using antid epressants but continue to have lingering cognitive issues.  It is estimated that in the US and Europe alone that there are more than 4.3 million suffering from cognitive impairment that have been successfully treated for MDD.  Therapeutic strategies currently available today include expensive psychotherapy, cognitive remediation and more recently transcranial magnetic stimulation and currently there are no approved therapeutics to treat the cognitive symptoms.

During these early years, like many typical startups, the technology development program was funded through non-dilutive grants and other sources of government support.  And like many startups with promise we provided our early input and guidance on metrics of success and what the company would need including clinical program, company structure, IP and license strategy and team to move the program to the next level to be a leading biotech company poised for success.  In late 2020 and early 2021 the founders formalized a process to raise equity capital to take the lead their lead into the clinic.  Seeing many of the hallmarks of a founding team that could move to the next level, we more formally re-engaged with the founder in the latter half of 2021 knowing the technology well and decided to undertake a more formal diligence effort through which we were to help fully refine the business plan and work closely with the founder to raise capital in 2022.  Despite the challenging markets in 2022 we were able to identify a co-lead, The Noetic Fund, and close the Seed round in September 2022.

Since the close of the Seed financing the company has strengthened the team, enhanced its investor group, hired a world class CEO, John Riley and is now in a position to be in the clinic in 2024.

Iterion Therapeutics Announces First Patient Dosed in Phase 1b/2a Clinical Trial of Tegavivint in Patients with Advanced Hepatocellular Carcinoma Who Have Failed One or More Systemic Treatments

February 20, 2024 / Portfolio News

Iterion Therapeutics, an oncology-focused biopharmaceutical company developing small molecule inhibitors of Transducin beta-like protein 1 (TBL1), a downstream target in the Wnt/beta-catenin signaling pathway, announced today it is actively enrolling a Phase 1b/2a clinical trial of its lead molecule, tegavivint, in patients with advanced hepatocellular carcinoma (HCC) that have failed at least one line of systemic therapy.

Activation of the Wnt/beta-catenin pathway is a hallmark of several forms of cancer, including HCC, in which approximately half of patients have tumors with Wnt-activating mutations. Unfortunately, key members of this pathway (including beta-catenin) have either been resistant to conventional drug development or plagued with off-target toxicities. Extensive pre-clinical study results across multiple tumor types suggest that TBL1 is a downstream target that is necessary for Wnt/beta-catenin-activated oncogenesis. TBL1 binds nuclear beta-catenin thus stabilizing the transcription complex necessary to turn on cancer-causing genes. Tegavivint’s direct binding to TBL1 displaces beta-catenin and disrupts this complex, allowing the degradation of free nuclear beta-catenin and preventing downstream oncogenic gene transcription.

“By targeting TBL1, tegavivint promotes potent inhibition of nuclear beta-catenin oncogenic activity without inducing toxicities observed for other drugs that are upstream in the Wnt-pathway” stated Rahul Aras, Ph.D., President & CEO for Iterion Therapeutics. “HCC is a devastating cancer with a high prevalence of Wnt-activated tumors, and we are committed to developing tegavivint for this patient population that otherwise has very few therapeutic options”.

HCC is the sixth most commonly diagnosed cancer and the third leading cause of cancer death globally. In 2023, the National Cancer Institute (NCI) estimated 41,210 new HCC cases diagnosed in the US, with 29,380 deaths. The overall prognosis is poor, with a 5-year survival rate of 21.6%. Wnt-activation plays multiple roles in the pathogenesis of HCC by initiating tumorigenesis, promoting metastasis, and enhancing an immunosuppressive tumor microenvironment. Patients with elevated nuclear beta-catenin and TBL1 demonstrate particularly poor outcomes. Despite this, there are no FDA-approved therapies targeting this pathway.

“There is a desperate need for novel targeted therapies to address the large unmet clinical need in HCC” expressed Casey Cunningham, M.D., Chief Medical Officer for Iterion Therapeutics. “Tegavivint has demonstrated excellent tolerability and has the potential to address a large portion of this population by directly inhibiting beta-catenin’s oncogenic activity”.

The ongoing clinical trial will enroll approximately 35 patients in dose escalation and optimization cohorts of patients with unresectable locally advanced or metastatic HCC that have failed at least one line of systemic treatment. The study will evaluate safety and clinical efficacy in addition to pharmacokinetic and pharmacodynamic markers to determine a Recommended Phase 2 Dose (RP2D) in HCC. Tegavivint has demonstrated safety, clinical and pharmacodynamic activity in a Phase 1 clinical study of patients with desmoid tumors.

For more information about this Phase 1b/2a clinical trial of tegavivint in patients with advanced HCC, please visit www.ClinicalTrials.gov using the identifier NCT05797805.

About Iterion Therapeutics

Iterion Therapeutics is a clinical-stage biotechnology company developing novel cancer therapeutics. The company’s lead product, tegavivint, is a potent and selective small molecule that binds to Transducin beta-like protein 1 (TBL1), a newly characterized target in the Wnt/beta-catenin pathway. TBL1 binds to nuclear beta-catenin, forming an active transcription complex that prevents nuclear degradation of beta-catenin and activates genes necessary for the Wnt/beta-catenin oncogenic activity. Tegavivint binds to TBL1 and disrupts the interaction between beta-catenin and TBL1, thus promoting the degradation of nuclear beta-catenin and preventing the transcription of Wnt/beta-catenin targeted oncogenes. Iterion is currently advancing multiple clinical programs investigating tegavivint in cancer indications where nuclear beta-catenin overexpression is a known factor. These include Iterion-sponsored programs in hepatocellular carcinoma (HCC), as well as investigator-sponsored programs in non-small cell lung cancer (NSCLC), diffuse large B-cell lymphomas (DLBCL), and pediatric solid tumors. Iterion is the recipient of combined a $18.9 million in Product Development Awards from the Cancer Prevention and Research Institute of Texas (CPRIT). For more information on Iterion, please visit  www.iteriontherapeutics.com.

SOURCE Iterion Therapeutics

enGene Announces Oversubscribed $200 Million Private Placement Financing

February 14, 2024 / Portfolio News

Financing included participation from new and existing investors

Pro forma cash and cash equivalents expected to fund current operating plan into 2027

enGene Holdings Inc. (Nasdaq: ENGN or “enGene” or the “Company”), a clinical-stage genetic medicines company whose non-viral lead program EG-70 is in a pivotal study for BCG-unresponsive non-muscle invasive bladder cancer (NMIBC), today announced that it has agreed to sell 20 million of its common shares at a price per share of $10.00, representing a 31% premium over the closing price on February 13, 2024. The financing is expected to close on February 16, 2024, subject to customary closing conditions. enGene anticipates the gross proceeds from the private placement to be $200 million, before deducting any offering-related expenses.

The financing included participation from new and existing investors, including Adage Capital Partners, LP, Blue Owl Healthcare Opportunities, Boxer Capital, Commodore Capital, Cormorant Asset Management, Deep Track Capital, an affiliate of Deerfield Management Company, Foresite Capital, Janus Henderson Investors, Logos Capital, Lumira Ventures, Marshall Wace, Perceptive Advisors, Soleus Capital, Surveyor Capital (a Citadel company), Venrock Healthcare Capital Partners, and a large investment management firm.

enGene intends to use the proceeds from this financing to fund the continued development of EG-70, the Company’s genetic medicine therapeutic candidate being evaluated in an ongoing pivotal study for BCG-unresponsive NMIBC, evaluation of expanded EG-70 development opportunities, potential new R&D programs and for working capital and general corporate purposes. The proceeds from this financing, combined with current cash and cash equivalents are expected to be sufficient to fund the current operating plan into 2027.

Leerink Partners, Guggenheim Securities and Wells Fargo Securities are acting as placement agents on the offering.

The securities described above have not been registered under the Securities Act of 1933, as amended. Accordingly, these securities may not be offered or sold in the United States, except pursuant to an effective registration statement or an applicable exemption from the registration requirements of the Securities Act. enGene has agreed to file a registration statement with the U.S. Securities and Exchange Commission (the “SEC”) registering the resale of the common shares issued in this private placement. Any offering of the securities under the resale registration statement will only be made by means of a prospectus.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such jurisdiction.

About enGene

enGene is a late-stage biotechnology company mainstreaming genetic medicines through the delivery of therapeutics to mucosal tissues and other organs, whose lead program EG-70 is being evaluated in an ongoing pivotal study for patients with non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (Cis) who are unresponsive or naïve to treatment with Bacillus Calmette-Guérin (BCG). EG-70 was developed using enGene’s proprietary Dually Derivatized Oligochitosan (DDX) platform, which enables penetration of mucosal tissues and delivery of a wide range of sizes and types of cargo, including DNA and various forms of RNA. enGene became a publicly traded company effective November 1, 2023, upon the completion of a business combination with Forbion European Acquisition Corporation, a special purpose acquisition company.

Forward-Looking Statements

Some of the statements contained in this press release may constitute forward-looking statements within the meaning of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, and “forward-looking information” within the meaning of Canadian securities laws (collectively, “forward-looking statements”). enGene’s forward-looking statements include, but are not limited to, statements regarding enGene’s expectations, hopes, beliefs, intentions, goals, strategies, forecasts and projections. The words “anticipate”, “appear”, “approximate”, “believe”, “continue”, “could”, “estimate”, “expect”, “foresee”, “intend”, “may”, “might”, “plan”, “possible”, “potential”, “predict”, “project”, “seek”, “should”, “would”, and similar expressions may identify forward-looking statements, but the absence of these words does not mean that a statement is not forward-looking. Forward-looking statements may include, for example, statements about: the timing and expectation of the closing of the private placement; the satisfaction of customary closing conditions related to the private placement and the anticipated use of proceeds therefrom; and the period over which enGene estimates the proceeds from the private placement, combined with its existing cash and cash equivalents, will be sufficient to fund its current operating plan.

Many factors, risks, uncertainties and assumptions could cause the Company’s actual results, performance or achievements to differ materially from those expressed or implied by the forward-looking statements, including, without limitation, the Company’s ability to recruit and retain qualified scientific and management personnel; establish clinical trial sites and enroll patients in its clinical trials; execute on the Company’s clinical development plans and ability to secure regulatory approval on anticipated timelines; and other risks and uncertainties detailed in filings with Canadian securities regulators on SEDAR+ and with the U.S. Securities and Exchange Commission (“SEC”) on EDGAR, including those described in the “Risk Factors” section of the Company’s Annual Report on Form 10-K for the fiscal year ended October 31, 2023 (copies of which may be obtained at www.sedarplus.ca or www.sec.gov).

You should not place undue reliance on any forward-looking statements, which speak only as of the date on which they are made. enGene anticipates that subsequent events and developments will cause enGene’s assessments to change. While enGene may elect to update these forward-looking statements at some point in the future, enGene specifically disclaims any obligation to do so, unless required by applicable law. Nothing in this press release should be regarded as a representation by any person that the forward-looking statements set forth herein will be achieved or that any of the contemplated results of such forward-looking statements will be achieved.

SOURCE enGene

Transposon Announces Final Results from a Phase 2 Study of its LINE-1 Reverse Transcriptase Inhibitor TPN-101 for the Treatment of Progressive Supranuclear Palsy and Interim Results from a Phase 2 Study of TPN-101 for the Treatment of C9orf72-Related Amyotrophic Lateral Sclerosis and/or Frontotemporal Dementia

February 13, 2024 / Portfolio News

TPN-101 is the first PSP treatment to reduce NfL and IL-6 levels, key biomarkers of neurodegeneration and neuroinflammation in PSP

Participants treated with TPN-101 for the entire 48-week study in PSP showed a stabilization of their clinical symptoms as measured by the PSPRS

In the Phase 2 study of C9orf72-related ALS/FTD, TPN-101 showed effects on key biomarkers of neurodegeneration, neuroinflammation and microglial activation, including NfL, Tau, UCHL1, YKL-40 and osteopontin

TPN-101 also showed a clinical effect on Vital Capacity, an objective respiratory measure that correlates with mortality in patients with ALS

Transposon plans to advance TPN-101 to a Phase 3 registration study for PSP and is expanding the development of TPN-101 into Alzheimer’s disease

Transposon Therapeutics, a biotechnology company developing a platform of novel, orally administered therapies for the treatment of neurodegenerative and aging-related diseases including Alzheimer’s disease, today announced final results from its Phase 2 study of TPN-101 for the treatment of progressive supranuclear palsy (PSP). The company also announced interim results from its Phase 2 study of TPN-101 in patients with amyotrophic lateral sclerosis (ALS) and/or frontotemporal dementia (FTD) related to hexanucleotide repeat expansion in the C9orf72 gene (C9orf72-related ALS/FTD). Transposon will present final results from its Phase 2 study in PSP in a poster presentation at the hybrid AD/PD™ 2024: 18th International Conference on Alzheimer’s and Parkinson’s Diseases. The meeting will take place online and in Lisbon, Portugal, from March 5-9, 2024.

Final Phase 2 PSP Study Results

Final 48-week results from the Phase 2 study in PSP confirm and extend the 24-week interim results that showed TPN-101 is the first treatment for PSP to reduce levels of neurofilament light chain (NfL), a key biomarker of neurodegeneration in tauopathies such as PSP and Alzheimer’s disease. Participants treated with placebo from weeks 1 to 24 and then switched to 400 mg TPN-101 at week 24 experienced a reduction of NfL in the cerebrospinal fluid (CSF) from weeks 24 to 48 that was similar to that observed in the group of participants receiving the 400 mg dose from weeks 1 to 24. Participants treated with 400 mg TPN-101 for the entire 48-week treatment period showed no increase of NfL levels in the CSF from weeks 1 to 48. In contrast, NfL levels in the CSF are reported to increase by 9-18% annually in natural history studies of PSP.

The 48-week results also showed further dose-related reductions in interleukin 6 (IL-6) cytokine levels, as well as reductions in levels of osteopontin. IL-6 is a biomarker of neuroinflammation that is elevated in PSP and correlates with disease progression and severity. Osteopontin levels correlate with cognitive deficit in Alzheimer’s disease patients and lowering of osteopontin may predict cognitive improvement.

Participants treated with TPN-101 for the entire 48-week trial duration showed a stabilization of their clinical symptoms as measured by the PSP Rating Scale (PSPRS) between weeks 24 and 48. In contrast, participants who had been on placebo from weeks 1 to 24 continued to show a worsening of the PSPRS between weeks 24 and 48, suggesting a delay of clinical benefit onset of at least 24 weeks after start of drug treatment, and lagging behind the early effects on biomarkers seen in weeks 1 to 24.

“There are no effective treatments for PSP, a uniformly fatal disease that is similarly prevalent to ALS,” said Adam Boxer, M.D., Ph.D., Endowed Professor in Memory and Aging in the Department of Neurology at the University of California, San Francisco and principal investigator in the Phase 2 study of TPN-101 for PSP. “The effects of TPN-101 on CSF NfL concentrations, as well as other exploratory CSF biomarkers, have not previously been observed in any PSP trial and support further investigation of clinical treatment effects in a larger study.”

Interim Phase 2 C9orf72-Related ALS/FTD Study Results

Results from the pre-defined interim analysis of the Phase 2 study in C9orf72-related ALS/FTD demonstrated the impact of TPN-101 treatment on key biomarkers of neurodegeneration, neuroinflammation, and microglial activation including NfL, tau, UCHL1, YKL-40, and osteopontin from baseline to 24 weeks. The data also showed a clinical effect on Vital Capacity, an objective respiratory measure that correlates with mortality in patients with ALS. Notably, C9orf72-related ALS/FTD is a TDP-43 protein pathology associated with nuclear displacement of TDP-43 in the central nervous system. TDP-43 pathology also occurs in about 50% of patients with Alzheimer’s disease.

“Transposon’s founding mission was to establish human proof-of-concept that dysregulation of retrotransposable elements in neurodegenerative diseases such as PSP, ALS and Alzheimer’s disease can be addressed with a new class of LINE-1 reverse transcriptase inhibitors,” stated Eckard Weber, M.D., Founder and Chief Innovation Officer of Transposon. “The results obtained with TPN-101 in both PSP and ALS/FTD show for the first time that neurodegenerative diseases that involve LINE-1 dysregulation are treatable with a specific inhibitor of this important novel target. We look forward to advancing the development of TPN-101 into registration studies to address this devastating and rapidly growing disease category.”

“Given the large unmet needs in PSP and ALS, these promising results highlight the therapeutic potential of TPN-101 for both disorders,” added Murali Doraiswamy, MBBS, FRCP, Professor of Psychiatry and Geriatrics at Duke University and chair of Transposon’s clinical advisory board. “These data also provide key validation of targeting LINE-1 to treat other tauopathies such as Alzheimer’s disease.”

“Based on the groundbreaking study results shown by TPN-101 in both PSP and ALS/FTD, we plan to rapidly advance TPN-101 into a Phase 3 registration study for the treatment of PSP, and potentially ALS/FTD as well, pending final confirmatory study results,” said Dennis Podlesak, Chairman and Chief Executive Officer of Transposon. “Importantly, the totality of the results across both studies also provides compelling scientific rationale for targeting LINE-1 to treat Alzheimer’s disease. Based on these findings, we are expanding our development efforts into Alzheimer’s disease with the goal of bringing a novel drug treatment to patients that remain in dire need of an effective new therapeutic option.”

About AD/PD 2024

The hybrid AD/PD 2024: 18th International Conference on Alzheimer’s and Parkinson’s Diseases will take place online and in Lisbon, Portugal, from March 5-9, 2024. Transposon will present final results from its Phase 2 study in PSP in a poster presentation (abstract #433) at AD/PD 2024 entitled: “A Phase 2a Study of TPN-101, a Nucleoside Reverse Transcriptase Inhibitor, in Patients with Progressive Supranuclear Palsy.” For more information, please visit the AD/PD 2024 website.

About the Phase 2 Study in PSP

The Phase 2 study in PSP is a multi-center, randomized, double-blind, placebo-controlled, parallel-group, 4-arm study with an open-label treatment phase in patients with PSP. Participants (n=42) were randomized to receive daily doses of 100 mg, 200 mg or 400 mg of TPN-101, or placebo. The study includes a 6-week screening period, a 24-week double-blind treatment period, a 24-week open label treatment period, and a follow-up visit 4 weeks post-treatment. All phases of the study, including the 24-week open label treatment period, have been completed. Further information on the study can be accessed at ClinicalTrials.gov.

About the Phase 2 Study in C9orf72-related ALS/FTD

The Phase 2 study in C9orf72-related ALS/FTD is multi-center, randomized, double-blind, placebo-controlled parallel-group, 2-arm study with an open-label treatment phase in patients with C9orf72-related ALS and/or FTD. Participants (n=42) were randomized to receive daily doses of 400 mg of TPN-101 or placebo. Similar to the Phase 2 study in PSP, this study includes a 6-week screening period, a 24-week double-blind treatment period, a 24-week open-label treatment period, and a follow-up visit 4 weeks post-treatment. The predefined interim analysis was performed after all patients completed the 24-week double-blind portion of the study. The open-label extension of the study is ongoing, with subjects and investigators blinded to the original treatment assignment. Further information on the study can be accessed at ClinicalTrials.gov.

About TPN-101

TPN-101 specifically inhibits the LINE-1 reverse transcriptase that promotes LINE-1 replication. LINE-1 elements are a class of retrotransposable elements that in humans are uniquely capable of replicating and moving to new locations within the genome. When this process becomes dysregulated, LINE-1 reverse transcriptase drives overproduction of LINE-1 DNA, triggering innate immune responses that contribute to neurodegenerative, autoimmune and aging-related disease pathology.

About PSP

PSP is a rare and fatal tauopathy with no approved treatment options. PSP mainly affects people in their mid- to late-60s and is characterized by early postural instability and falls, vertical gaze palsy, akinesia, rigidity, pseudobulbar palsy, and frontal dysfunction with cognitive and behavioral changes. The mean survival for individuals with PSP is 6 to 7 years.

About ALS and FTD

ALS is a neurodegenerative disease characterized by progressive muscle weakness, loss of ability to speak, eat, move or breathe. FTD is a progressive frontal / temporal cortex disease associated with behavior and personality changes, emotional problems, and difficulty walking, communicating or working. With onset commonly in middle age or earlier, patients with ALS have a mean survival of only 3 years. Patients with FTD have a mean survival of 9 years. 

About Transposon

Transposon Therapeutics, Inc. is a clinical-stage biopharmaceutical company developing a platform of novel therapies for the treatment of neurodegenerative and aging-related diseases, including Alzheimer’s disease. The company’s lead clinical compound, TPN-101, is first-in-class to address LINE-1 reverse transcriptase for treating neurodegenerative and autoimmune diseases. The company also has a discovery platform supporting a deep pipeline of novel therapies to address additional indications. Dr. Doraiswamy chairs the company’s clinical advisory board and has received compensation/stock options for his role.

Contact:
Rick Orr 
Transposon Therapeutics, Inc.
(858) 535-4821
rorr@transposonrx.com

SOURCE Transposon Therapeutics

Endogenex Receives IDE Approval to Initiate Pivotal Clinical Study

February 1, 2024 / Portfolio News

A Prospective, Randomized, Multicenter Study Assessing the Safety and Efficacy of the ReCET™ System in Adult Patients with Type 2 Diabetes

Endogenex, Inc., a clinical stage medical device company dedicated to improving outcomes in patients with Type 2 Diabetes (T2D), announced that the U.S. Food and Drug Administration (FDA) granted an Investigational Device Exemption (IDE) to initiate a clinical study of the ReCET System for the treatment of T2D in adult patients, inadequately controlled by non-insulin, glucose lowering medications.

The ReCET Clinical Study is a multicenter, prospective, randomized, double blinded, sham controlled study assessing the safety and effectiveness of the ReCET™ System. The pivotal study will enroll up to 350 patients at clinical sites in the United States and Australia. 

“Endogenex is excited to achieve this important clinical milestone,” said Stacey Pugh, Endogenex CEO. “This is a critical next step in advancing the ReCET Procedure as a treatment that addresses the underlying causes of T2D that are not targeted by current diabetes medications.”

“Many people living with T2D struggle to remain in control of their blood sugar levels, even while using modern glucose lowering medications. This presents a significant challenge for patients and their care providers,” stated Richard Pratley, MD, Medical Director of the Advent Health Diabetes Institute in Orlando, Florida and Co-Principal Investigator for the ReCET Study. “Preliminary results of the ReCET Procedure have been very encouraging, so we look forward to participating in this study and expanding the understanding of how it may fulfill a significant need in the care of this patient population.”

“The role of the duodenum in metabolic function and signaling is well-established,” stated Dr. Barham Abu Dayyeh, MD, MPH, the Director of Advanced and Metabolic Endoscopy at Mayo Clinic in Rochester, Minnesota, and Co-Principal Investigator for the ReCET Study. “Regenerating duodenal tissue through the ReCET Procedure has the potential to target underlying pathophysiologic factors associated with Type 2 Diabetes. The evaluation of this procedure within the ReCET Study may pave the way for a novel treatment approach to this disease.”

The ReCET Clinical Study will be enrolling patients at up to 30 sites in the United States and up to 10 sites in Australia.

The ReCET™ Procedure:
ReCET is a novel, endoscopic, outpatient procedure that targets the underlying cellular pathology of the duodenum that may contribute to the development and progression of Type 2 Diabetes.

Through the application of highly controlled, non-thermal pulsed electric fields, the ReCET System is designed to initiate the body’s natural regenerative process to restore proper cellular signaling from the duodenum and improve metabolic function, including better control of blood glucose levels.

The ReCET System has been evaluated as part of feasibility clinical studies including REGENT-1 US, REGENT-1 Australia, and EMINENT in the Netherlands. The clinical studies assessed safety and efficacy in adults with Type 2 Diabetes whose blood glucose levels were inadequately controlled, despite the use of insulin and non-insulin medications. Publication of preliminary study outcomes has been presented at clinical conferences globally.

The ReCET System has received Breakthrough Device Designation from the FDA for treatment of Type 2 Diabetes in adult patients inadequately controlled by glucose lowering medications.

About Endogenex:
Endogenex is a privately held, clinical stage company headquartered in Minneapolis, MN. Endogenex was founded, in partnership with Mayo Clinic, to develop therapies to improve outcomes for people living with Type 2 Diabetes. Endogenex’s focus is centered on the principle that effective treatment for Type 2 Diabetes can extend beyond pharmaceutical options, correct metabolic function, and help people regain control of their blood glucose levels.

For more information, please visit our website at www.endogenex.com or www.twitter.com/endogenex

Media Inquiries:
Brent Collins
bcollins@endogenex.com
+1(612)-227-6949

SOURCE Endogenex

COUR Pharmaceuticals Secures $105 Million in Series A Financing Co-Led by Lumira Ventures and Alpha Wave Ventures

January 30, 2024 / Portfolio News
    • Funds will be used to support the advancement of COUR’s Myasthenia Gravis (MG) and Type 1 Diabetes (T1D) product candidates into Phase 2a clinical trials
    • Participating investors include Roche Venture Fund, Pfizer, Bristol Myers Squibb, Angelini Ventures, and JDRF T1D Fund

    CHICAGO, Jan. 30, 2024 (GLOBE NEWSWIRE) — COUR Pharmaceuticals, a clinical-stage biotechnology company focused on the development of first-in-class, disease modifying therapies designed to induce antigen-specific tolerance for immune-mediated diseases, today announced the closing of a Series A investment round, securing approximately $105 million in financing. The investment was co-led by Lumira Ventures and Alpha Wave Ventures, with participation from Roche Venture Fund, Pfizer (as part of the Pfizer Breakthrough Growth Initiative), Bristol Myers Squibb, Angelini Ventures, and the JDRF T1D Fund. In connection with the financing, Benjamin “Beni” Rovinski, Ph.D., Managing Director of Lumira Ventures, and Simon Greenwood, Senior Investment Director of Roche Venture Fund, will join the COUR Board of Directors.

    The proceeds will enable COUR to advance multiple, wholly owned product candidates that leverage the company’s immune tolerance platform, including Phase 2a proof-of-concept clinical studies in Myasthenia Gravis and Type 1 Diabetes, and other pipeline opportunities. These product candidates, in addition to existing Phase 2 partnered programs with Takeda Pharmaceuticals in Celiac Disease and Ironwood Pharmaceuticals in Primary Biliary Cholangitis, continue to strengthen COUR’s position as the leader in antigen-specific immune tolerance.

    “The impressive syndicate of thought-leading investors and prominent strategic investors is a testament to COUR’s platform technology and our paradigm-changing potential for patients impacted by a variety of autoimmune diseases,” said John J. Puisis, founder and chief executive officer of COUR. “We are excited to have this support as we advance our pipeline and revolutionize antigen-specific immune tolerance while avoiding immune system suppression in our mission to potentially bring to market life changing therapies for patients.”

    Benjamin “Beni” Rovinski, Ph.D., managing director of Lumira Ventures added, “There is an urgent need for innovative strategies aimed at restoring self-tolerance safely and more effectively in autoimmune disorders. We believe COUR’s proprietary and strongly differentiated platform is a versatile and first-in-class approach to meet this critical medical need. We are dedicated to collaborating with COUR and its partners to help advance these potentially cutting-edge treatments for immune-related diseases.”

    About COUR Pharmaceuticals: COUR Pharmaceuticals is a clinical-stage biotechnology company developing therapies to treat patients with autoimmune and inflammatory diseases. COUR’s first-in-class therapies are based on our proprietary antigen-specific immune tolerance platform and are designed to reprogram the immune system to address the underlying root cause of immune-mediated diseases. Data from multiple clinical and preclinical programs have demonstrated the ability of COUR’s product candidates to induce antigen-specific immune tolerance and have the potential to treat a wide range of autoimmune and inflammatory diseases.

    COUR is developing product candidates in Myasthenia Gravis and Type 1 Diabetes in addition to having partnered products in Celiac Disease (with Takeda Pharmaceuticals), and Primary Biliary Cholangitis (with Ironwood Pharmaceuticals).

    For more information, please visit www.courpharma.com

    About Lumira Ventures: Founded in 2007, Lumira Ventures is a multi-stage life sciences investment firm with offices in Toronto, Montréal, Vancouver, and Boston. We partner with mission-driven entrepreneurs building companies that harness rapidly evolving advancements in medicine to develop transformative products for patients while delivering strong financial returns for our investors and meaningful economic value and impact for society.

    For more information, visit www.lumiraventures.com

    About Alpha Wave Global: Alpha Wave is a global investment company with offices in New York, Miami, London, Abu Dhabi, Tel Aviv, Bangalore, Jakarta, and Sydney. Its flagship global venture and growth fund, Alpha Wave Ventures, aims to invest in best-in-class venture and growth-stage companies and endeavors to be helpful long-term partners to the founders and management teams. Alpha Wave manages a variety of investment partnerships that cover several asset classes, themes, and geographies.

    For more information, please visit www.alphawaveglobal.com

    Contacts

    For Investor Relations:

    Brian Bock, Chief Financial Officer

    bbock@courpharma.com

    For Media:

    Jason Braco, Ph.D.

    jbraco@lifescicomms.com

    Cyrano Therapeutics Secures $9.0 Million Series B Financing to Advance Clinical Development of CYR-064 for Treatment of Post-Viral Smell Loss

    January 16, 2024 / Portfolio News

    Phase 2 FLAVOR clinical trial currently enrolling 150 patients to assess the safety and effectiveness of CYR-064 in treating patients with chronic smell loss following recovery from a viral infection (post-viral hyposmia)

    No FDA-approved drug therapy available for this increasingly prevalent and serious chronic sensory condition

    Cyrano Therapeutics, Inc., a regenerative medicine company pioneering the development of treatments for smell loss, announced today that it has secured a $9.0 million Series B financing to advance the development of CYR-064 as a potential first-ever treatment for post-viral smell loss (hyposmia). Co-lead investors participating in the financing include the Florida Opportunity Fund managed by DeepWork Capital and existing investors Lumira Ventures and Remiges Ventures.

    Cyrano intends to use the proceeds from its Series B financing to advance FLAVOR, its Phase 2, double-blind, randomized clinical trial of CYR-064, an intranasal theophylline spray therapy to treat patients who have lost their sense of smell following recovery from a viral infection. The FLAVOR Phase 2 trial will be conducted at up to 15 sites in the US and the Series B financing is to fund the trial through data readout.

    “This Series B financing enables us to advance what we believe to be a first-of-its-kind treatment for patients suffering from long-term smell loss due to a viral infection,” said Rick Geoffrion, President and CEO of Cyrano Therapeutics, Inc. “Smell loss correlates to functional loss of taste, which can both diminish quality of life and present significant health and safety risks, thus underscoring the urgency in advancing this important therapeutic. We also plan to conduct exploratory research for patients with Parkinson’s disease, 95 percent of whom often experience loss of smell and flavor as the first symptom of the disease.”

    Jackson Streeter, MD, Venture Partner at DeepWork Capital, stated: “Cyrano Therapeutics represents an important investment for DeepWork and the Florida Opportunity Fund as we seek to identify and cultivate emerging biotechnology companies in Florida that combine executive and scientific expertise with therapeutic opportunities that offer the potential to address significant, unmet medical needs. We look forward to supporting the entire Cyrano team as the company advances CYR-064 through the FLAVOR Phase 2 trial.”

    The number of patients experiencing long-term smell and flavor loss has increased 10-fold in the wake of the COVID-19 pandemic to more than 40 million patients in the US and Europe. Thirty percent of those suffering from smell loss will experience a hazardous event such as food poisoning or the inability to detect hazardous fumes. There is currently no FDA-approved drug therapy to treat smell loss from any cause.

    About Hyposmia
    Hyposmia, including post-viral hyposmia, is an increasingly prevalent and serious chronic sensory condition for which there is no approved drug therapy and limited treatment options. Hyposmia causes significant impairment in quality of life for many sufferers. Moreover, in older individuals, hyposmia is associated with an increased risk of cognitive impairment and mortality. Prior to the COVID-19 pandemic, an estimated 8 million individuals in the US and Europe suffered from long-term post-viral hyposmia. The COVID-19 pandemic has resulted in dramatically increased prevalence, with an estimated 40 million individuals in the US and Europe currently suffering from long-term post-viral hyposmia.

    About Cyrano Therapeutics
    Cyrano Therapeutics is a private, venture-backed clinical stage regenerative medicine company. Since our foundation, we have been working diligently to develop therapies for people struggling with the loss of smell and taste. To learn more, please visit cyranotherapeutics.com.

    About the FLAVOR Trial
    The Phase 2 FLAVOR trial is a 150 subject randomized, double-blinded, placebo-controlled, multi-dose, multi-site clinical trial of CYR-064 for the treatment of post-viral hyposmia. For more information, please visit ClinicalTrials.gov

    About DeepWork Capital
    DeepWork Capital invests professionally managed committed venture capital in growth-oriented, early-stage companies in the technology and life science sectors. We partner with visionary entrepreneurs building disruptive companies. DeepWork works closely with other investment groups and takes a hands-on approach with its portfolio companies. For additional information, please see www.deepworkcapital.com

    Media Contacts:
    Tiberend Strategic Advisors, Inc.

    Dave Schemelia
    dschemelia@tiberend.com
    +1 609-468-9325

    Eric Reiss
    ereiss@tiberend.com
    +1 802-249-1136

    SOURCE Cyrano Therapeutics

    World’s First Patients Treated With Novel Edison® Histotripsy System

    January 9, 2024 / Portfolio News

    HistoSonics, (www.histosonics.com), the manufacturer of the non-invasive Edison® Histotripsy System, announced today a patient from the University of Rochester Medical Center has received the world’s first targeted liver tumor treatments utilizing the Edison System following its recent FDA De Novo clearance. In the same week Cleveland Clinic treated their initial patients suffering from liver tumors utilizing histotripsy. HistoSonics’ image guided sonic beam therapy system uses proprietary technology and advanced imaging to deliver non-invasive, personalized treatments with precision and control. The science of histotripsy uses focused sound energy to produce controlled acoustic cavitation that mechanically destroys and liquifies targeted tissue at sub-cellular levels.

    On December 18th, Roberto Hernandez-Alejandro MD, Chief of the Division of Transplantation, Koji Tomiyama, MD, PhD Associate Professor of Surgery & Transplant, and team were first to use Edison to perform a histotripsy liver treatment on a patient suffering from recurrent liver tumors originating in the colon. “There is an enormous potential applicability of histotripsy in patients with liver tumors. By destroying targeted liver tumors, histotripsy opens the opportunities for patients to be downstaged and bridged for surgical resections and transplantation,” said Dr. Hernandez-Alejandro. He added, “One of the most interesting findings in our early experience is the preservation of the vessels and blood flow, which is unique and may open a new era in liver therapies.”

    C.H. David Kwon, MD, PhD, Director of Minimally Invasive Liver Surgery at Cleveland Clinic, and his team performed their initial procedure on a patient with primary colorectal disease with metastasis to the liver that has continued to progress despite getting chemotherapy. “In our early experience, we have found that patients have the potential to benefit from histotripsy because it is less invasive than other treatment options and our patients reported a lack of pain following the procedure,” said Dr. Kwon. The Cleveland Clinic team led by Dr. Kwon, Federico Aucejo, MD, and Jaekeun Kim MD has treated three patients with liver tumors of multiple different diseases.

    “Our purpose has always been to make a meaningful change in the lives of patients,” said Joe Herman MD, Medical Director. Herman added, “Working with the University of Rochester Medical Center and Cleveland Clinic as the first to offer non-invasive histotripsy to treat their patients’ liver tumors as part of standard clinical practice supports our ongoing mission.” HistoSonics is in the process of training key staff and launching over a dozen of its first partners programs around the United States. The company believes that the novel mechanism of action of their proprietary technology provides significant advantages to patients, including the ability of the treatment site to recover and resorb quickly. Uniquely, the HistoSonics’ platform also provides physicians the ability to monitor the destruction of tissue under continuous real-time visualization and control, unlike any modality that exists today.

    For more information on the University of Rochester Medical Center Histotripsy and Liver Surgery Team: University of Rochester Medical Center Liver Surgery and Transplant

    For more information on the Cleveland Clinic Liver Surgery Team: Cleveland Clinic Liver Surgery and Transplant

    The Edison® System is intended for the non-invasive mechanical destruction of liver tumors, including the partial or complete destruction of unresectable liver tumors via histotripsy. The FDA has not evaluated the Edison System for the treatment of any specific disease or condition. Use of Edison System in kidney applications is limited by federal law to investigational use.

    About HistoSonics

    HistoSonics is a privately held medical device company developing non-invasive platforms and proprietary sonic beam therapy utilizing the science of histotripsy, a novel mechanism of action that uses focused ultrasound to mechanically destroy and liquify unwanted tissue and tumors. The company is currently focused on commercializing their Edison System in the US and select global markets for liver treatment while expanding histotripsy applications into other organs like kidney, pancreas, and others. HistoSonics has offices in Ann Arbor, Michigan and Minneapolis, MN.

    For more information please visit: www.histosonics.com/

    Contacts

    For Media Inquiries
    Media contact:
    Josh King
    Vice President of Global Market Access
    Email : Joshua.king@histosonics.com
    Phone : +1 608.332.8124

    AmacaThera Closes Series A Extension to Advance Clinical Development of Long Acting, Localized, Non-Opioid Therapeutics to Improve Post-Surgery Patient Care

    November 29, 2023 / Portfolio News

    AmacaThera, a leader in the development of novel injectable, localized therapeutics based upon its AmacaGel delivery platform, announced the closing of a CAD$4.0 million financing round, with a new lead investor, supported by existing investors; BDC Capital’s Women in Technology Venture Fund, Inveready, Lumira Ventures, StandUp Ventures, and MaRS IAF. The proceeds will be used to accelerate the clinical development of the lead clinical candidate, AMT-143, and also to advance multiple pipeline programs targeting the local, injectable, sustained release market.

    Despite the continuing opioid epidemic, which results in ~80,000 deaths annually in the US alone, the treatment of pain continues to be frequently managed through opioid-based medications. This includes in post-surgical settings, where it can be a gateway to addiction and the abuse of prescription pain medicines and illicit narcotics. Leveraging the AmacaGel platform, AmacaThera is developing AMT-143 with the objective of it being a best-in-class, non-opioid, therapeutic able to provide an extended period of post-surgical pain relief and reduce with the aim of eliminating the need for opioids in the postsurgical recovery period. Pre-clinical studies have shown superior release kinetics to the current standard of care, resulting in rapid onset pain relief followed by long-acting pain control for up to 3 days, thereby providing relief over the most severe pain window following surgery. The Phase Ib clinical trial is scheduled to commence in early 2024.

    The Company’s unique, injectable, hydrogel AmacaGel platform is effective for the delivery of a wide range of therapeutics, from small molecules to antibodies to lipid nanoparticles to stem cells, as demonstrated in over 40 peer-reviewed publications. With the demonstrated safety in humans, the funding will also be used to expand the application of the platform and to develop additional products for the pipeline.

    Lead investor Paul Austin said, “AmacaThera is developing a product that fulfills both societal and medical needs for a large market.”

    Lumira Ventures, Lu Han, agreed, “AmacaThera continues to build an innovative portfolio of therapeutics that have the potential to establish a new standard of care across a variety of indications.”

    “We are excited to welcome our new lead investor to our board, as they share our enthusiasm for AmacaThera’s vision and potential for long-term success”, said Dr. Michael Cooke, CEO and Co-Founder, “This investment will enable us to expand and accelerate our efforts in developing new long-acting injectable products”.

    “This financing provides further evidence of the importance of therapeutic delivery in the life science innovation cycle”, said Dr. Molly Shoichet, CSO, Co-Founder, and University Professor at the University of Toronto. “The commitment of our existing investors reflects their recognition of the platform opportunity and the potential product portfolio.”

    About AmacaThera

    AmacaThera Inc., a resident company of Johnson & Johnson Innovation, JLABS @ Toronto is a clinical-stage company transforming therapeutics to make a difference in patient health. AmacaGel, our unique, injectable hydrogel platform, provides localized sustained drug delivery for improved patient outcomes across multiple therapeutic areas, including post-surgical pain management, cancer and other hard-to-reach, unmet medical needs.

    For more information, please visit www.amacathera.ca.

    SOURCE AmacaThera Inc.

    For further information: Michael Foorer, mike_foorer@amacathera.ca

    Summer 2024 Analyst Position Open

    November 21, 2023 / Lumira News

    Looking to make your mark in the world of venture capital and life sciences innovation? Lumira Ventures is accepting applications for exceptional business students to join our team as a Summer Analyst. By working hand in hand with seasoned industry professionals from all dimensions of the life sciences ecosystem, you will gain first-hand knowledge and insights into the challenges, opportunities, processes, and people shaping the future of healthcare innovation in Canada and around the globe.

    The Summer Analyst will work closely with Lumira’s Toronto-based investment team to learn all aspects of the venture business, with a particular focus on financial modeling, business diligence, and proactive market analysis.

    Term: Full-time May 13th – August 23rd, 2024

    Location: 141 Adelaide Street West, Lumira Ventures Office, Downtown Toronto

    Requirements:

    • This position is open to upper-year undergraduates, MBAs, and Masters students with a strong financial analysis skill set and business competencies.
    • Candidates should also have a demonstrated interest in life sciences and healthcare innovation. Work, volunteer, or academic experience in the life sciences would be an asset.
    • Candidates must be a Canadian citizen or Permanent Resident of Canada and be able to be located in Toronto for the full term (4-5 days per week in office).

    Application Deadline: December 15th, 2023

    Application: https://airtable.com/appaUyWYqi2T6AIoV/shrMihXUpc8llNyHc

    Lumira Ventures

    Lumira Ventures is a leading, globally focused life sciences investment firm. We are a triple bottom-line investor that builds transformative companies developing life-changing and life-saving health solutions for patients, while also generating superior realized returns for our investors. As an impact-focused investor, we believe we give our employees the unique opportunity to combine passion and vocation in a highly collaborative environment with a group of highly aligned and motivated colleagues. The breadth of our investment success stories spans from the first cure for hepatitis C infection, to robotic surgical platforms in orthopeadics, to breakthrough treatments for cancer, neurological disorders and cardiovascular disease. Our portfolio companies have developed and brought to market dozens of therapies and innovative healthcare products, impacting the lives of over 1 billion patients globally and generating cumulative sales in excess of $80 billion.

    Our dynamic investment team draws from backgrounds in company formation and management, research science, business development, entrepreneurial and consulting backgrounds. We pride ourselves on our collaborative, results-oriented culture and we strive to identify candidates who are well suited to our unique, team-based environment.  Lumira is committed to having a workforce that reflects the communities in which we live and work. We are an equal opportunity employer committed to a barrier-free recruitment and selection process. At Lumira Ventures inclusion and diversity means belonging, and we believe our high retention and very low turnover reflect the quality of the experience and environment we have built for our employees.

    Lumira Ventures Portfolio Company enGene (Nasdaq: ENGN) Debuts as Publicly Traded Genetic Medicines Company

    November 2, 2023 / Portfolio News

    CAD$191 million gross proceeds from the transaction to support the advancement of the clinical program and pipeline expansion

    Pivotal clinical trial for lead program EG-70 targeting BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) currently enrolling patients

    Lumira Ventures portfolio company enGene Holdings Inc., (“enGene” or the “Company”), a clinical-stage biotechnology company developing genetic medicines, has completed a business combination with a special purpose acquisition company and a concurrent oversubscribed private investment in public equity (PIPE) financing. As a result of this combination, enGene has become a publicly traded company whose shares are now listed on the Nasdaq market under the symbol “ENGN.” Gross proceeds from this transaction totaled approximately CAD$191 million.

    With operations in Montréal and Boston, enGene is currently enrolling patients in a pivotal trial of its lead program EG-70 (detalimogene voraplasmid) in patients with BCG-unresponsive non-muscle-invasive bladder cancer with carcinoma in situ.  EG-70 was developed using the company’s proprietary Dually Derivatized Oligochitosan (DDX) platform, which enables a carrier to penetrate mucosal tissue and deliver a wide range of sizes and types of cargo, including DNA and various forms of RNA, by turning mucosal cells into therapeutic production “factories” that generate proteins and RNAs for a desired local effect.

    “We are extremely proud of the progress enGene has made since our foundational investment in the company in 2015 via our Merck Lumira Biosciences Fund,” stated Peter van der Velden, Managing General Partner of Lumira Ventures. “That fund focused on identifying and building transformative biotechnology companies in Quebec, and enGene is certainly fulfilling that promise. Our commitment to the company runs deep and we are pleased to have provided the continuity of capital that is so critical for success in our sector, via investments in enGene from multiple Lumira-managed funds, including most recently via Lumira Ventures Fund IV’s participation in the PIPE transaction.”

    “Lumira Ventures financial support and strategic guidance have been critical to enGene’s ability to develop our therapeutic platform, build a world-class team, and complete this important transaction,” said Jason Hanson, Chief Executive Officer of enGene. “We now look forward to advancing our shared mission to expand genetic medicine into the mainstream of clinical practice and address the high disease burden faced by patients in our areas of focus.”

    Gerry Brunk, Managing Director of Lumira Ventures and enGene board member, added: “We extend our appreciation to the entire enGene management team and board in achieving this important milestone, as well as to our longtime enGene co-investors, Fonds de solidarité FTQ and Forbion. We also note the support of this transaction by other Lumira limited partners Northleaf Capital Partners, Investissement Québec, and other new investors including BVF Partners, Omega Funds, Cowen Healthcare Investments, and Vivo Capital.”

    Based on data from Pitchbook and the CVCA, we believe enGene’s entry onto the Nasdaq exchange represents the first Canadian biotechnology company to debut in the public markets since 2020. We also believe that the company’s oversubscribed PIPE is the largest equity financing for a private or public Canadian biotechnology company in 2023.

    For complete details on this transaction, please visit this link.

    About Lumira Ventures

    Founded in 2007, Lumira Ventures is a multi-stage life sciences investment firm with offices in Toronto, Montréal, Vancouver, and Boston. We partner with mission-driven entrepreneurs building companies that harness rapidly evolving advancements in medicine to develop transformative products for patients while delivering strong financial returns for our investors and meaningful economic value and impact for society. For more information, visit www.lumiraventures.com

    About enGene

    enGene is a clinical-stage biotechnology company mainstreaming genetic medicines through the delivery of therapeutics to mucosal tissues and other organs, with the goal of creating new ways to address diseases with high clinical needs. enGene’s lead program is EG-70 for patients with non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (Cis) who are unresponsive or naïve to treatment with Bacillus Calmette-Guérin (BCG) – a disease with a high clinical burden. EG-70 is being evaluated in an ongoing Phase 2 pivotal study. EG-70 was developed using enGene’s proprietary Dually Derivatized Oligochitosan (DDX) platform, which enables penetration of mucosal tissues and delivery of a wide range of sizes and types of cargo, including DNA and various forms of RNA. For more information, visit www.enGene.com.      

    Forward-Looking Statements

    Some of the statements contained in this press release may constitute forward-looking statements within the meaning of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, and “forward-looking information” within the meaning of Canadian securities laws (collectively, “forward-looking statements”).  enGene’s forward-looking statements include, but are not limited to, statements regarding enGene’s expectations, hopes, beliefs, intentions, goals or strategies regarding the future. In addition, any statements that refer to projections, forecasts or other characterizations of future events or circumstances, including any underlying assumptions, are forward-looking statements. The words “anticipate”, “appear”, “approximate”, “believe”, “continue”, “could”, “estimate”, “expect”, “foresee”, “intend”, “may”, “might”, “plan”, “possible”, “potential”, “predict”, “project”, “seek”, “should”, “would”, and similar expressions (or the negative versions of such words or expressions) may identify forward-looking statements, but the absence of these words does not mean that a statement is not forward-looking. Forward-looking statements may include, for example, statements about: enGene’s expected future business plans and expansion of gene therapy into clinical practice; the potential benefits of the DDX platform and the future results and timing of Phase 1 and Phase 2 studies; the expected use of net proceeds of the Business Combination and the associated private funding; the effectiveness and use cases of enGene’s EG-70 Program; the expected timeline for the interim Phase 2 results from the EG-70 program. Readers are cautioned that forward-looking statements are not guarantees of future performance and that actual results or developments may differ materially from those expressed or implied in the forward-looking statements.

    All forward-looking statements, including, without limitation, our examination of historical operating trends, are based upon our current expectations and various assumptions. Certain assumptions made in preparing the forward-looking statements include the Company’s ability to recruit and retain qualified scientific and management personnel, establish clinical trial sites and patient registration for clinical trials and acquire technologies complimentary to, or necessary for, its programs; the Company is able to enroll a cohort of patients in the Phase 2 LEGEND trial to assess EG-70’s efficacy and safety in the BCG-naïve patient population to evaluate its ultimate potential as a monotherapy in first line patients and expanding EG-70’s opportunity; the Company is able to file a Biologics License Application in 2025 with the FDA for approval to market EG-70 in the United States as a monotherapy to treat BCG-unresponsive NMIBC; EG-70’s product profile can be integrated seamlessly into community urology clinics where the vast majority of NMIBC patients are treated; the Company is able to retain commercial rights to EG-70 in the United States and commercialize EG-70 independently, while selectively partnering outside of the United States; the Company is able to execute the “pipeline-in-a-product” development strategy for EG-70; and the Company is able to utilize the dually derived chitosan gene delivery platform to develop effective, new agents for the delivery of genetic medicines to mucosal tissues.

    All forward-looking statements are subject to risks, uncertainties and other factors that may cause actual results to differ materially from those that we expected and/or those expressed or implied by such forward-looking statements. These risks and uncertainties include: the expected benefits of the Business Combination;  enGene’s financial performance following the Business Combination, including financial projections and business metrics and any underlying assumptions thereunder; the ability to maintain the listing of enGene securities on Nasdaq following the Business Combination; enGene’s success in recruiting and retaining, or changes required in, officers, key personnel or directors following the completion of the Business Combination;  enGene’s plans and ability to execute product development, manufacturing process development, preclinical and clinical development efforts successfully and on anticipated timelines; enGene’s ability to design, initiate and successfully complete clinical trials and other studies for its product candidates and its plans and expectations regarding its ongoing or planned clinical trials;  enGene’s plans and ability to obtain and maintain marketing approval from the U.S. Food and Drug Administration and other regulatory authorities, including the European Medicines Agency, for its product candidates; enGene’s plans and ability to commercialize its product candidates, if approved by applicable regulatory authorities; the degree of market acceptance of enGene’s product candidates, if approved, and the availability of third-party coverage and reimbursement; the ability of enGene’s external contract manufacturers to support the manufacturing, release testing, stability analysis, clinical labeling and packaging of enGene’s products; enGene’s future financial performance and the sufficiency of  enGene’s cash and cash equivalents to fund its operations; the outcome of any known and unknown litigation and regulatory proceedings; enGene’s ability to implement and maintain effective internal controls; or other risks and uncertainties detailed in filings with Canadian securities regulators on SEDAR+ and U.S. Securities and Exchange Commission on EDGAR.

    Any forward-looking statement speaks only as of the date on which it was made. enGene anticipates that subsequent events and developments will cause enGene’s assessments to change. While enGene may elect to update these forward-looking statements at some point in the future, enGene specifically disclaim any obligation to do so, unless required by applicable law. Nothing in this press release should be regarded as a representation by any person that the forward-looking statements set forth herein will be achieved or that any of the contemplated results of such forward-looking statements will be achieved. You should not place undue reliance on forward-looking statements, which speak only as of the date they are made.

    Specific Biologics Announces Additional Investment and Appointment of Experienced Biotechnology Leader Steven Kanner, PhD, to the Board of Directors

    October 17, 2023 / Portfolio News

    Specific Biologics Inc. (“Specific”), a venture-backed, early-stage genome editing company, is pleased to announce it has appointed Steven Kanner, PhD, as an Independent Director. Dr. Kanner joins Brent Stead, PhD, MBA, as well as seasoned company builders Daniel Hétu, MD, MBA of Lumira Ventures, Nikhil Thatte, B.Eng of Lumira Ventures and Frédéric Lemaître Auger, MSc, PhD, MBA of adMare BioInnovations. The appointment coincides with renewed investment from Lumira Ventures and adMare BioInnovations to advance the preclinical development of therapeutics based on Specific’s proprietary Dualase® platform gene editors.

    “We are thrilled to have the renewed commitment from Lumira and adMare and to welcome Dr. Kanner to the Board of Directors,” said Dr. Stead, CEO of Specific Biologics Inc. “He brings decades of experience in executive leadership roles in genome editing and rare disease companies from preclinical research to commercially launched products. He will be an immense asset to Specific as we advance our gene editing technology toward the clinic and, most importantly, bring potentially life-changing therapeutics to patients with serious genetic diseases.”

    “This is an exciting time to be joining Specific Biologics as the potential for gene editing technologies to transform the lives of patients is starting to be realized in the clinic,” said Dr. Kanner. “Specific’s differentiated Dualase gene editor has promise to modify a number of disease-causing mutations in vivo that are not easily targeted with today’s gene editors, and I look forward to helping the team implement Dualase’s potential.”

    Dr. Kanner serves as Chief Scientific Officer at Caribou Biosciences where he is leading preclinical discovery, technical operations, and translational development of genome-edited cell therapies.  He has held research leadership positions at prominent life sciences organizations, including Bristol-Myers Squibb, Astex Pharmaceuticals, Agensys/Astellas Pharma, and Arrowhead Pharmaceuticals. Prior to joining Caribou Biosciences, Dr. Kanner served as Vice President of Discovery Biology at Arrowhead Pharmaceuticals, where he helped lead the discovery and development of novel RNAi-based therapeutic candidates for oncology and genetic diseases. At Bristol-Myers Squibb, he facilitated a discovery program ultimately leading to the development and launch of Sprycel® (dasatinib), a treatment for chronic myeloid leukemia. Dr. Kanner graduated from the University of California, Berkeley with a B.A. in genetics and earned his Ph.D. in immunology and microbiology from the University of Miami’s Miller School of Medicine. Dr. Kanner completed his postdoctoral fellowship in oncology at the University of Virginia’s Department of Microbiology, Immunology, and Cancer Biology.

    Dualase’s unique two-site mechanism of action enables efficient and precise disruption and repair in therapeutic areas that are not easily addressed with existing gene editors. Specific has demonstrated the benefit of this two-site mechanism in externally validated studies in disease-relevant models at a broad and diverse set of genetic mutations where Dualase uniquely addresses the mutation or has a differentiated advantage. The additional investment from Lumira Ventures and adMare BioInnovations will be used to advance preclinical testing of Dualase gene editors in liver, ocular and CNS disorders. In addition, Specific will continue the development of Dualase gene editors for Cystic Fibrosis with preclinical studies supported by the Cystic Fibrosis Foundation.

    “Our additional investment in Specific demonstrates our belief that Dualase gene editors have a unique advantage at many targets for therapeutic gene editing,” noted Dr. Daniel Hetu, Managing Director at Lumira Ventures. “The efficiency and precision of Dualase edits in disease-relevant cell models generated by third-party researchers support this thesis.”

    “adMare is excited to continue to partner with Specific and provide them with the necessary financial, commercial, and scientific resources to advance their preclinical data packages in various therapeutic areas with significant unmet medical needs,” shared Dr. Frédéric Lemaître Auger, Vice President of Investments at adMare BioInnovations. “We look forward to working together to select the most promising clinical applications of the Dualase technology.”

    About Specific Biologics Inc.

    Specific Biologics Inc. (“Specific”) is a venture-backed early-stage biotechnology company on a mission to develop novel gene editing technologies to treat diseases through efficient and precise gene editing. Our differentiated two-site Dualase® platform gene editors cut DNA in a way that optimally exploits the cell’s naturally occurring DNA repair pathways. This enables two gene editing outcomes, precise DNA deletions to disrupt genes or increased repair to correct genes to target new therapeutic areas for gene editing. Specific also develops lipid nanoparticles to deliver the gene editor to target cells and is developing a pipeline of Dualase®-based therapeutics in areas of high unmet medical need. To learn more, visit www.specificbiologics.com.

    About Lumira Ventures

    Lumira Ventures is a North American healthcare venture capital firm with decades of experience of investing in and helping to build transformative biomedical companies.  We are a multi-stage investor that partners with mission-driven entrepreneurs and like-minded investors to build innovative healthcare companies.  These companies are harnessing rapidly evolving innovations in genomics, cell therapy, gene therapy, bioengineering, robotics and artificial intelligence to develop high impact, often transformative products for patients while generating exceptional returns for their investors and meaningful economic value to society. To date, Lumira’s companies have brought dozens of biomedical innovations to the market, impacting the lives of patients worldwide. Lumira Ventures manages its activities from offices in Toronto, Montréal, Vancouver and Boston.  For more information, please visit www.lumiraventures.com

    About adMare BioInnovations

    With a strong track record of globally-competitive scientific discovery, Canadian life sciences are primed to lead the world. To make this a reality, adMare BioInnovations uses its scientific and commercial expertise, specialized R&D infrastructure, and seed capital to build strong life sciences companies, develop robust ecosystems, and foster industry-ready talent. It re-invests its returns into the Canadian industry to ensure its long-term sustainability. adMare currently has 29 portfolio companies that have attracted $2.3 billion of risk capital, have a combined value of $4 billion, and have created over 1,000 jobs in Canada. For more information, please go to www.admarebio.com.

    SOURCE Specific Biologics Inc.

    Edesa Biotech to Receive Up To C$23 Million in Funding from Federal Government

    October 12, 2023 / Portfolio News

    Edesa Biotech, Inc. (Nasdaq:EDSA), a clinical-stage biopharmaceutical company focused on inflammatory and immune-related diseases, has secured a commitment of up to C$23 million from the Government of Canada for a pivotal Phase 3 clinical study of the company’s first-in-class therapeutic candidate.

    Edesa’s experimental drug, called EB05 (paridiprubart), represents a new class of emerging therapies called Host-Directed Therapeutics (HDTs) that are designed to modulate the body’s own immune response when confronted with infectious diseases or even chemical agents. Importantly, these therapies are agnostic to the causal agent and can be stockpiled preemptively for seasonal outbreaks and unexpected emergencies and threats.

    “This project has the potential to increase survival rates, reduce ICU costs and improve outcomes for critically ill patients,” said Par Nijhawan, MD, Chief Executive Officer of Edesa Biotech. “With this validation and the continued support from the federal government, we believe we are in a position to accelerate research, expand our reach to more hospitals and move another significant step closer to commercialization.”

    Edesa’s current project builds on the success of a government-supported Phase 2 clinical study completed during the pandemic which demonstrated that paridiprubart reduced mortality by 84% among critically ill patients with a severe form of respiratory disease called Acute Respiratory Distress Syndrome (ARDS). A parallel in vitro study at the University of Toronto also demonstrated recently that paridiprubart inhibits inflammation from influenza and other pathogens.

    “We are proud of our track record of delivering successful results on time and on budget for our government-supported projects,” said Dr. Nijhawan. “The development of breakthrough medicines – especially in the critical care fields – is key to building a strong biopharma sector, creating jobs and most importantly improving patient outcomes at home and abroad. We are honored to be a part of these efforts.”

    The Honorable Francois-Philippe Champagne, Minister of Innovation, Science and Industry said that the Strategic Innovation Fund (SIF) funding announced today is part of the government’s plan to grow a strong and competitive life sciences sector, and ensure the nation’s readiness for future pandemics or other health emergencies.

    “This project is a prime example of Canada’s determination to the development of the next generation of medicine, while creating good jobs and securing long-term economic growth,” said Minister Champagne.

    Edesa intends to use the SIF funding toward study expenses, including hospital and physician expenditures, as well as scale-up of commercial drug product should the development program be successful. Funding is provided under the federal government’s Strategic Innovation Fund (SIF) following a competitive review process.

    Additional information regarding the funding are outlined in the company’s Current Report on Form 8-K, which Edesa expects to file with the U.S. Securities and Exchange Commission and on the SEDAR+ system in Canada.

    About ARDS

    ARDS involves an exaggerated immune response leading to inflammation and injury to the lungs that prevents the lungs from oxygenating blood and ultimately deprives the body of oxygen. For moderate to severe cases, there are currently few meaningful treatments, other than supplemental oxygen and mechanical ventilation, and patients suffer high mortality rates. In addition to virus-induced pneumonia, ARDS can be caused by smoke/chemical inhalation, sepsis, chest injury and other causes. Prior to the pandemic, ARDS accounted for 10% of intensive care unit admissions, representing more than 3 million patients globally each year.

    About EB05 (Paridiprubart)

    Paridiprubart is a first-in-class monoclonal antibody developed for acute and chronic disease indications that involve dysregulated innate immune responses. This host-directed therapeutic (HDT) candidate inhibits toll-like receptor 4 (TLR4), a key immune signaling protein that has been shown to be activated both by viruses, like SARS-CoV2, SARS-CoV1 and Influenza, as well as in the pathogenesis of chronic autoimmune diseases.

    About Phase 3 Clinical Study

    Edesa’s Phase 3 study of EB05 (paridiprubart) is a multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of EB05 in critical-care patients. The current protocol calls for treatment of ARDS subjects hospitalized with SARS-CoV2 infections who are on invasive mechanical ventilation, both with and without additional organ support. The primary endpoint is the mortality rate at 28 days. In addition to SARS-CoV2 induced ARDS, Edesa is currently exploring various approaches to evaluate EB05 in a general ARDS population.

    About Edesa Biotech, Inc.

    Edesa Biotech, Inc. (Nasdaq:EDSA) is a clinical-stage biopharmaceutical company developing innovative ways to treat inflammatory and immune-related diseases. The company’s most advanced drug candidate is EB05 (paridiprubart), a monoclonal antibody developed for acute and chronic disease indications that involve dysregulated innate immune responses. Edesa is currently evaluating EB05 in a Phase 3 study as a potential treatment for Acute Respiratory Distress Syndrome (ARDS), a life-threatening form of respiratory failure. In addition, Edesa is developing an sPLA2 inhibitor, EB01 (daniluromer), as a topical treatment for chronic Allergic Contact Dermatitis (ACD), a common occupational skin condition. The company has also received regulatory approval to conduct a Phase 2 trial its EB06 monoclonal antibody as a treatment for vitiligo, a life-altering autoimmune disease that causes skin to lose its color in patches. Edesa is also planning to file an investigational new drug application for a future Phase 2 study of paridiprubart for systemic sclerosis (scleroderma), an autoimmune rheumatic disorder that causes fibrosis, (scarring/hardening) of skin and internal organs such as the lungs, heart and kidneys. Sign up for news alerts. Connect with us on Twitter and LinkedIn.

    Edesa Forward-Looking Statements

    This press release may contain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. Forward-looking statements may be identified by the use of words such as “anticipate,” “believe,” “plan,” “estimate,” “expect,” “intend,” “may,” “will,” “would,” “could,” “should,” “might,” “potential,” or “continue” and variations or similar expressions. Readers should not unduly rely on these forward-looking statements, which are not a guarantee of future performance. There can be no assurance that forward-looking statements will prove to be accurate, as all such forward-looking statements involve known and unknown risks, uncertainties and other factors which may cause actual results or future events to differ materially from the forward-looking statements. Such risks include: the ability of Edesa to obtain regulatory approval for or successfully commercialize any of its product candidates, the risk that access to sufficient capital to fund Edesa’s operations may not be available or may be available on terms that are not commercially favorable to Edesa, the risk that Edesa’s product candidates may not be effective against the diseases tested in its clinical trials, the risk that Edesa fails to comply with the terms of license agreements with third parties and as a result loses the right to use key intellectual property in its business, Edesa’s ability to protect its intellectual property, the timing and success of submission, acceptance and approval of regulatory filings, and the impacts of public health crises, such as Covid-19. Many of these factors that will determine actual results are beyond the company’s ability to control or predict. For a discussion of further risks and uncertainties related to Edesa’s business, please refer to Edesa’s public company reports filed with the U.S. Securities and Exchange Commission and the British Columbia Securities Commission. All forward-looking statements are made as of the date hereof and are subject to change. Except as required by law, Edesa assumes no obligation to update such statements.

    Contact

    Gary Koppenjan
    Edesa Biotech, Inc.
    (289) 800-9600
    investors@edesabiotech.com

    SOURCE: Edesa Biotech

    Life Sciences BC Announces Companies and Mentors for Fourth Annual Investor Readiness Program

    October 12, 2023 / Lumira News

    Life Sciences BC welcomes the companies and mentors participating in its 2024 Investor Readiness Program (IRP). Through a focused six-month program, small and mid-size enterprises (SMEs) in British Columbia’s life sciences sector are paired with entrepreneurs-in-residence (EIRs) to become investor-ready. The industry expertise and acumen of the mentors provide the companies with invaluable insight and support, culminating in a final pitch session with investors and community stakeholders at the end of the program.

    Companies and mentors are paired according to skillset, experience, and interests. The SMEs and EIRs selected for the 2024 IRP are:

    EIR Mentors

    The program runs from October to March and emphasizes personalized coaching, educational sessions, goal setting, peer-to-peer feedback, networking opportunities, and group meetings. Applications for the 2025 program will open in spring 2024.

    *This program is supported in part by funding from the National Research Council of Canada Industrial Research Assistance Program (NRC IRAP).

    We are delighted to launch the 2024 Investor Readiness Program, dedicated to empowering our newest startups through education and mentorship and enabling them to acquire the skills and knowledge necessary to attract investors. The vibrant life sciences community in British Columbia is rapidly expanding, and we thank the NRC IRAP for their invaluable support of this important initiative. We also thank our dedicated entrepreneurs-in-residence for their exceptional leadership and commitment to giving back to our sector. We look forward to another thrilling investor journey with our incredible BC SMEs, who represent the bright future of our sector.”

    Wendy Hurlburt, President and CEO, Life Sciences BC

    The IRP program empowered me with fundraising knowledge and provided me with a safe space to practice my pitch. The best part for me was the connection with my mentor, who continues to offer valuable guidance, unwavering support, and encouragement. She has made me a better leader of my company and team.”

    Chris Tam, CEO, Integrated Nanotherapeutics and IRP Graduate 2023

    The IRP is an extraordinary program. It’s inspiring and energizing for the mentors to spend time with bright entrepreneurs with fresh new ideas and a lot of passion. It’s also a great opportunity for entrepreneurs to learn from their peers and a wide range of experienced mentors. These relationships continue well beyond the program’s completion and can last for many years into the future.”

    David Helliwell, Co-Founder & Executive Board Chair, Thrive Health and EIR

    I thought that the program was great. We really grew up as a company and became far more professional. Our program had great mentors and great presenters. Highly recommended.”

    Gordon Eberwein, CEO, Derm-Biome Pharmaceuticals and IRP Graduate 2023

    For more information about LSBC’s Investor Readiness Program, please contact William Burrows, Director, Business Operations and Programs: wburrows@lifesciencesbc.ca 604-669-9099 ext: 101

    About Life Sciences BC

    Life Sciences BC is a non-profit industry association committed to championing and representing British Columbia’s vibrant life sciences community. Our multifaceted approach encompasses local, national, and international initiatives to foster collaboration across diverse sectors, drive economic development, and facilitate vital connections, investments, and global partnerships.

    At Life Sciences BC, we implement impactful programs and projects that advocate for public policies, elevate regional and international visibility, and help expand BC’s thriving life sciences sector. Our representation extends to various stakeholders, including academic and research organizations, established and emerging life sciences companies, and the professional services supporting this dynamic ecosystem.

    Contacts

    Johanna Mills
    Manager, Marketing & Communications, Life Sciences BC
    Email: jmills@lifesciencesbc.ca
    Web: www.lifesciencesbc.ca

    Cyrano Therapeutics Enrolls First Patient in Phase 2 Trial of CYR-064 for Post-Viral Smell Loss (Hyposmia)

    October 12, 2023 / Portfolio News

    There is currently no FDA-approved therapy for this increasingly prevalent and serious chronic sensory condition.

    Cyrano Therapeutics, Inc., a regenerative medicine company pioneering the development of treatments for smell loss, announced today that it has commenced enrollment in the Phase 2 FLAVOR trial, a randomized, double-blinded, placebo-controlled, multi-dose clinical trial of CYR-064, its novel, soft-mist nasal-spray product candidate, for the treatment of post-viral hyposmia. Post-viral hyposmia is an increasingly prevalent and serious chronic sensory condition for which there is currently no approved drug therapy.

    “Hyposmia, along with the associated alterations in taste results in a significant reduction in quality of life, as this common and serious condition manifests as a loss of two of the five basic human senses,” stated Mas Takashima, MD FACS, Professor and Chairman of the Department of Otolaryngology – Head and Neck Surgery at Houston Methodist Hospital and the principal investigator for the FLAVOR trial. “CYR-064 has the potential to become the first pharmaceutical product to restore the senses of smell and taste in patients with post-viral hyposmia and thereby address a major unmet clinical need.”

    Cyrano expects to enroll approximately 150 subjects in the FLAVOR trial, which is designed to assess the local nasal safety, tolerability, and effectiveness of CYR-064 as compared to placebo in the treatment of post-viral hyposmia over a six-month period. The FLAVOR trial is a multi-institutional prospective study being conducted at fifteen clinical sites in the United States. 

    “We are excited to announce a significant milestone in our commitment to advancing medical science and improving the lives of those affected by post-viral smell loss,” said Rick Geoffrion, President and CEO of Cyrano Therapeutics, Inc. “With the enrollment of the first patients in our Phase 2 trial of CYR-064, we take a giant step forward in addressing a pressing health concern. In a world where post-viral smell loss continues to affect an ever-growing number of individuals, we’re dedicated to pioneering solutions that make a difference.”

    About Hyposmia

    Hyposmia, including post-viral hyposmia, is an increasingly prevalent and serious chronic sensory condition for which there is no approved drug therapy [and limited treatment options]. Hyposmia causes significant impairment in quality of life for many sufferers. Moreover, in older individuals, hyposmia is associated with an increased risk of cognitive impairment and mortality. Prior to the COVID-19 pandemic, an estimated 8 million individuals in the U.S. and Europe suffered from long-term post-viral hyposmia. The COVID-19 pandemic has resulted in dramatically increased prevalence, with an estimated 40 million individuals in the U.S. and Europe currently suffering from long-term post-viral hyposmia. 

    About Cyrano Therapeutics

    Cyrano Therapeutics, Inc. is a private, venture-backed, clinical-stage regenerative medicine company. Since our inception, we have been working diligently to develop therapies for people struggling with the loss of smell and taste. For more information, please visit cyranotherapeutics.com.

    SOURCE Cyrano Therapeutics

    Endotronix Receives IDE Approval for the Market Expanding PROACTIVE-HF 2 Clinical Trial

    October 11, 2023 / Portfolio News

    Endotronix, Inc., a digital health and medical technology company dedicated to advancing the treatment of heart failure (HF), announced it has received Investigational Device Exemption (IDE) approval from the FDA for a subsequent multicenter study, PROACTIVE-HF 2, which will evaluate the company’s Cordella Sensor for pulmonary artery (PA) pressure-guided therapy. The prospective, dual-arm trial aims to expand access to New York Heart Association (NYHA) class II HF patients and support efficient and scalable remote patient management with a clinician-directed, patient self-management strategy.

    “The evidence in favor of PA pressure-guided therapy for NYHA class III HF patients has been consistently validated over the last 15 years. However, questions remain as to the therapy benefit in NYHA class II patients and how to best scale effective remote HF management,” stated Lynne W. Stevenson M.D., Heart Failure Specialist at Vanderbilt University Medical Center in Nashville, TN, and Global Principal Investigator (PI) of the PROACTIVE-HF 2 clinical trial. “The randomized arm of PROACTIVE-HF 2 will be the first study to compare PA pressure-guided therapy to a telehealth control arm. The single-arm cohort evaluates the power of engaged patients and subsequent impact on outcomes. Together these studies help us better understand the impact of pulmonary hypertension on right ventricular function over time and provide guidance on how to scale this important therapy with patient engagement.”

    The PROACTIVE-HF 2 prospective, open-label randomized controlled clinical trial will enroll up to 1,500 patients in the U.S. and Europe. The dual arm study design was presented over the weekend at HFSA and the first patient is expected to be enrolled later this year. The randomized arm will assess the safety and efficacy of PA pressure-guided therapy using Cordella in NYHA class II patients at risk for congestion. In both cohorts, patients and clinicians will have access to daily trended telehealth data (i.e. blood pressure, heart rate, and weight) and the treatment cohort will also have access to daily PA pressure data. The study will evaluate safety and efficacy, using a compositive first HF event or death rate, for up to 24 months. The single-arm study will assess the impact of clinician-directed patient self-management at home in NYHA class III HF patients who are at risk for poor outcomes using a 12-month endpoint for safety and incidence of HF hospitalization or death.  Both arms will collect data on secondary endpoints including changes in right ventricular function in relation to PA pressure, and patient engagement.

    Additionally, the Company presented 12-month sub-study data from its initial pivotal PROACTIVE-HF trial at the HFSA conference, demonstrating a low HF hospitalization rate of 0.34 at 12 months in NYHA class III HF patients.

    In December 2021, the prospective, multi-center pivotal PROACTIVE-HF trial was redesigned from a randomized controlled trial, with patients and providers in a control arm blinded to PA pressure values, to a single-arm study where both groups had access to patient data. The data for 63 former control arm patients was evaluated before and during the 12-month period following the unblinding, demonstrating significant improvements in mean PA pressure (mPAP) and outcomes, as well as strong patient interest in having access to PA pressure data.

    • In the unblinded period, average seated mPAP for patients above target (>20 mmHg) decreased significantly (28.1 mmHg vs. 23.6 mmHg, p=0.03)
    • 12-month unblinded HF hospitalization rate was significantly lower than the 12 months prior to implant (0.3 ±0.9 vs. 3±0.9, p<0.0001)
    • 78% of patients made lifestyle changes based on their mPAP trends
    • 86% of patients rated a positive impact of PA pressure management on their health

    This data dovetails the announcement that the PROACTIVE-HF study has completed its primary endpoint follow-up period and the data is being prepared for pre-market approval (PMA) submission to the FDA by the end of the year and data presentation in the first half of next year.

    “We are establishing a strong foundation of compelling clinical evidence for Cordella with early PROACTIVE-HF data. The team is driving towards PMA submission before the end of this year and looks forward to sharing the results from the full study cohort in 2024,” commented Harry Rowland, CEO and co-founder of Endotronix. “We remain confident in the benefits Cordella brings to patients and clinicians to improve heart failure outcomes and remain on track for a mid-2024 launch.”

    About Endotronix

    Endotronix innovates at the intersection of Medtech & Digital Health to improve care for people living with heart failure (HF).  The comprehensive Cordella solution enables proactive, data-driven HF management that engages patients, reduces and prevents congestion, and improves outcomes. The Cordella Sensor is an implantable pulmonary artery (PA) pressure sensor that directly measures the leading indicator of congestion, allowing early, targeted therapy. The Cordella HF System is a patient health management platform, which combines comprehensive vital sign data from non-invasive devices to support patient-clinician engagement and care decisions. Combining trended insights, the versatile and scalable Cordella enhances current clinical practice and supports guideline-based care across the entire HF continuum. Learn more at www.endotronix.com.

    The Cordella PA Pressure Sensor System is an investigational device and is not currently approved for clinical use in any geography. CAUTION – Investigational Device. Limited by Federal (or United States) Law to Investigational Use. Exclusively for Clinical Investigation. The Cordella HF System, without the sensor, is available for commercial use in the U.S. and E.U.

    Cautionary Statement Regarding Forward-Looking Statements

    This press release may contain predictions, estimates or other information that might be considered forward-looking statements. Such forward-looking statements are not a guarantee of future performance.

    MEDIA CONTACT:

    Carla Benigni

    SPRIG Consulting, LLC

    +1 (847) 951-7430

    carla@sprigconsulting.com

    OpSens, Inc Acquired by Haemonetics for $345 Million

    October 10, 2023 / Portfolio News

    Haemonetics Corporation (NYSE: HAE), a global medical technology company focused on delivering innovative medical solutions to drive better patient outcomes, and OpSens, Inc. (TSX:OPS) (OTCQX:OPSSF), a medical device cardiology-focused company delivering innovative solutions based on its proprietary optical technology, today announced that they have entered into a definitive agreement under which Haemonetics will acquire all outstanding shares of OpSens for CAD $2.90 per share in an all-cash transaction representing a fully diluted equity value of approximately USD $253 million at current exchange rate.

    OpSens offers commercially and clinically validated optical technology for use primarily in interventional cardiology. OpSens’ core products include the SavvyWire®, the world’s first and only sensor-guided 3-in-1 guidewire for TAVR procedures, that acts as pacing and pressure monitoring wire advancing the workflow of the procedure and enabling potentially shorter hospital stays for patients, and the OptoWire®, a pressure guidewire that aims to improve clinical outcomes by accurately and consistently measuring Fractional Flow Reserve (FFR) and diastolic pressure ratio (dPR) to aid clinicians in the diagnosis and treatment of patients with coronary artery disease. OpSens also manufactures a range of fiber optic sensor solutions used in medical devices and other critical industrial applications. 

    Stewart Strong, President, Global Hospital at Haemonetics, said, “With the acquisition of OpSens, we expand our leadership in interventional cardiology and strengthen our foundation for additional growth and diversification. By leveraging OpSens’ proprietary optical sensor technology, our global commercial infrastructure, and our relationships with the top US hospitals performing TAVR and PCI procedures, we have a powerful opportunity to improve standards of care for more physicians and patients worldwide. We are excited to welcome OpSens’ talented team and look forward to advancing our shared commitment to maximizing patient benefits and value for our customers.”

    This transaction creates compelling financial and strategic benefits for Haemonetics:

    • Expands Hospital business unit portfolio with innovative fiber optic sensor technology in the attractive interventional cardiology market. OpSens’ portfolio utilized in TAVR and PCI procedures offers strong competitive advantages with a total addressable market of approximately $1 billion. OpSens technology is also being used across a range of medical and industrial applications, representing additional avenues for growth and diversification.
    • Leverages Haemonetics’ commercial and geographic breadth to accelerate adoption. OpSens’ product portfolio has already demonstrated commercial success and is well-positioned for long-term growth. Haemonetics’ commercial success with its VASCADE® Vascular Closure portfolio, combined with extensive existing commercial and clinical infrastructure, will accelerate customer access to OpSens’ products with the potential to make SavvyWire the leading guidewire for TAVR procedures in the U.S. Additionally, Haemonetics’ presence in high-growth international markets will enable further penetration of OpSens products in these regions.
    • Augments long-term growth with additional product and market expansion opportunities. Haemonetics plans to build on the OpSens acquisition to further expand its Hospital business through internal and external R&D, clinical, and other business development efforts. Over the past several quarters, Haemonetics has made additional strategic investments, which would further complement OpSens’ portfolio and strengthen Haemonetics’ leadership in interventional cardiology, including in VivaSure Medical®, the company that developed PerQSeal®, an innovative percutaneous large-bore vessel closure technology.
    • Delivers immediate and longer-term financial benefits. The transaction is expected to be immediately accretive to Haemonetics’ revenue growth. On a GAAP basis, Haemonetics expects this transaction to be slightly dilutive to earnings per diluted share in fiscal year 2024 due to transaction and integration costs and accretive thereafter. Haemonetics expects this transaction to be immediately accretive to adjusted earnings per diluted share.

    Transaction Details and Financing: The transaction will be affected by way of an arrangement under the Business Corporations Act (Québec). Completion of the acquisition is subject to the approval of OpSens shareholders, receipt of court and regulatory approval, as well as certain other closing conditions customary for transactions of this nature. The transaction is expected to close by the end of January 2024.

    Haemonetics plans to finance this acquisition through a combination of cash and a revolving credit facility. Following this acquisition, Haemonetics’ net debt to EBITDA ratio, per the terms set forth in the Company’s existing Credit Agreement, is expected to be approximately 2.1x.

    Advisors: Goldman Sachs & Co. LLC served as financial advisor for Haemonetics and DLA Piper as legal advisor. Piper Sandler LLC served as OpSens’ financial advisor, while Norton Rose Fulbright served as its legal advisor.

    Supplemental Information: Haemonetics posted supplemental slides with additional information about this transaction to Haemonetics’ investor relations website. These slides can also be accessed by following this link: https://haemonetics.gcs-web.com/static-files/7e0041fd-89d4-4f20-bbd0-947c00118c05 

    About Haemonetics

    Haemonetics (NYSE: HAE) is a global healthcare company dedicated to providing a suite of innovative medical products and solutions for customers, to help them improve patient care and reduce the cost of healthcare. Our technology addresses important medical markets: blood and plasma component collection, the surgical suite, and hospital transfusion services. To learn more about Haemonetics, visit www.haemonetics.com.

    Cautionary Note Regarding Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Forward-looking statements do not relate strictly to historical or current facts and may be identified by the use of words such as “may,” “will,” “should,” “could,” “would,” “expects,” “plans,” “anticipates,” “believes,” “estimates,” “projects,” “predicts,” “forecasts,” “foresees,” “potential” and other words of similar meaning in conjunction with statements regarding, among other things, (i) plans and objectives of management for the operation of Haemonetics, (ii) statements regarding the timing of completion of the acquisition and the consummation of the acquisition, (iii) the anticipated financing of the transaction and the Company’s net debt to EBITDA ratio following completion of the acquisition, (iv) the anticipated benefits to Haemonetics arising from the completion of the acquisition, (v) the impact of the acquisition on Haemonetics’ business strategy and future business and operational performance, and (vi) the assumptions underlying or relating to any such statement. Such forward-looking statements are not meant to predict or guarantee actual results, performance, events or circumstances and may not be realized because they are based upon Haemonetics’ current projections, plans, objectives, beliefs, expectations, estimates and assumptions and are subject to a number of risks and uncertainties and other influences. Actual results and the timing of certain events and circumstances may differ materially from those described by the forward-looking statements as a result of these risks and uncertainties.

    Factors that may influence or contribute to the inaccuracy of the forward-looking statements or cause actual results to differ materially from expected or desired results may include, without limitation, the failure to realize the anticipated benefits of the acquisition or the acquisition, its announcement or pendency having an unanticipated impact, Haemonetics’ ability to predict accurately the demand for products and products under development by it or OpSens and to develop strategies to successfully address relevant markets, the impact of competitive products and pricing, technical innovations that could render products marketed or under development by Haemonetics or OpSens obsolete, risks related to the use and protection of intellectual property, the risk that the transaction may not be completed in a timely manner or at all (whether due to the failure of OpSens to obtain shareholder approval, the failure to obtain approval of the Québec Superior Court or otherwise), and the risk that using debt to finance, in part, the acquisition will increase Haemonetics’ indebtedness. These and other factors are identified and described in more detail in Haemonetics’ filings with the U.S. Securities and Exchange Commission (“SEC”). Haemonetics does not undertake to update these forward-looking statements.

    Non-GAAP Financial Measures

    This press release contains financial measures that are considered “non-GAAP” financial measures under applicable SEC rules and regulations. Management uses non-GAAP measures to monitor the financial performance of the business, make informed business decisions, establish budgets and forecast future results. Performance targets for management are also based on certain non-GAAP financial measures. These non-GAAP financial measures should be considered supplemental to, and not a substitute for, the Company’s reported financial results prepared in accordance with U.S. GAAP. In this release, supplemental non-GAAP measures have been provided to assist investors in evaluating the expected impact of the Company’s acquisition of OpSens and provide a baseline for analyzing trends in the Company’s underlying businesses. We strongly encourage investors to review the Company’s financial statements and publicly-filed reports in their entirety and not rely on any single financial measure.

    When used in this release, adjusted earnings per diluted share excludes restructuring costs, restructuring related costs, digital transformation costs, amortization of acquired intangible assets, asset impairments, accelerated device depreciation and related costs, costs related to compliance with the European Union Medical Device Regulation (“MDR”) and In Vitro Diagnostic Regulation (“IVDR”), integration and transaction costs, certain tax settlements and unusual or infrequent and material litigation-related charges, and the tax impact of these items. Because non-GAAP financial measures are not standardized, it may not be possible to compare these financial measures to similarly titled measures used by other companies.

    The Company does not attempt to provide reconciliations of forward-looking adjusted earnings per diluted share guidance to the comparable GAAP measures because the combined impact and timing of recognition of certain potential charges or gains, such as restructuring costs and impairment charges, is inherently uncertain and difficult to predict and is unavailable without unreasonable efforts. In addition, the Company believes such reconciliations would imply a degree of precision and certainty that could be confusing to investors. Such items could have a substantial impact on GAAP measures of the Company’s financial performance.

    Additional Information Regarding the Transaction

    In connection with the proposed transaction, OpSens will file with the Canadian Securities Administrators in each of the provinces of Canada (the “CSA”) and mail or otherwise make available to its shareholders a management information circular (the “Proxy Circular”) regarding the proposed transaction. BEFORE MAKING ANY VOTING DECISION, OPSENS’ SHAREHOLDERS ARE URGED TO READ THE PROXY CIRCULAR IN ITS ENTIRETY WHEN IT BECOMES AVAILABLE AND ANY OTHER DOCUMENTS FILED WITH THE CSA IN CONNECTION WITH THE PROPOSED TRANSACTION OR INCORPORATED BY REFERENCE THEREIN BECAUSE THEY WILL CONTAIN IMPORTANT INFORMATION ABOUT THE PROPOSED TRANSACTION. Investors and security holders may obtain a free copy of the Proxy Statement and other documents that OpSens files with the CSA (when available) from the CSA’s website at www.sedarplus.ca and from OpSens’ website at www.opsens.com.

    Investor Contacts:

    Olga Guyette, Sr. Director-Investor Relations & Treasury

    (781) 356-9763    olga.guyette@haemonetics.com 

    Media Contact:

    Josh Gitelson, Director-Global Communications 

    (781) 356-9776  josh.gitelson@haemonetics.com

    David Trenk, Manager-Investor Relations

    (203) 733-4987 david.trenk@haemonetics.com

    SOURCE Haemonetics Corporation

    FDA Awards HistoSonics Clearance of its First-of-a-Kind Edison® Histotripsy System

    October 10, 2023 / Portfolio News

    HistoSonics®, (www.histosonics.com), the manufacturer of the Edison® System and novel histotripsy therapy platforms, announced today the marketing authorization of its “Breakthrough” platform via the U.S. Food and Drug Administration’s (FDA) De Novo Classification Request process, a rigorous pre-market review pathway for medical devices with no existing predicate. Marketing authorization makes Edison the first and only histotripsy platform available in the Unites States.

    “This is HistoSonics’ most meaningful milestone to date and represents over two-decades of tireless efforts, from its inception in 2001, overcoming what was once thought to be impossible”

    Mike Blue, President, and CEO of HistoSonics

    FDA authorization was based, in part, on data from the #HOPE4LIVER Trials in 13 trial sites across the US and Europe. Data pooled from both the US and European/UK trials were used to assess the clinical safety and efficacy of histotripsy in destroying targeted primary and secondary liver tumors. Histotripsy was noted to have achieved both primary safety and efficacy endpoints in the pooled data where 44 subjects were evaluated for safety and 44 tumors treated were evaluated for efficacy. Important to note was the heterogeneity of the treated subjects, 18 of which had hepatocellular carcinoma (HCC) tumors and 26 had metastatic tumors to the liver from the colon, rectum, breast, and other primary origins.

    As recently presented at the annual CIRSE Congress in Copenhagen, a technical success rate of 95.5% was achieved indicating that physicians can precisely target and destroy liver tissue and unresectable liver tumors. Also, only 3 procedure related CTCAE Grade 3 or higher adverse events through 30 days post-histotripsy were observed across all 44 subjects treated, representing a complication rate of 6.8% with each event being common to focal liver therapies and not specific to histotripsy.

    “This is HistoSonics’ most meaningful milestone to date and represents over two-decades of tireless efforts, from its inception at the University of Michigan in 2001, overcoming what was once thought to be impossible – integrating the many complexities of histotripsy into a completely non-invasive clinical platform,” commented Mike Blue, President, and CEO of HistoSonics. The Company noted it has expanded its commercial and operational capacity over the previous year in preparation for commercial activities. Blue added, “We have been thoughtfully adding professionals with deep domain experience in operations, market development and education and are prepared to begin scheduling physician training immediately. This is a fantastic day for patients who will benefit from the novel advantages of histotripsy, and I commend the FDA for working so expeditiously with us throughout the review process.”

    The science of histotripsy uses focused sound energy to produce controlled acoustic cavitation that mechanically destroys and liquifies targeted liver tissue, including tumors, at sub-cellular levels. HistoSonics’ Edison System uses proprietary technology and advanced imaging to deliver personalized, non-invasive histotripsy treatments with precision and control. The company believes that the novel mechanism of action of their proprietary technology may provide significant advantages to patients, including the ability of the treatment site to recover and resorb quickly. Uniquely, the HistoSonics’ platform also provides physicians the ability to monitor the destruction of tissue under continuous real-time visualization and control, unlike any modality that exists today.

    “As a surgeon, it’s rewarding to be able to offer a procedure where we can precisely destroy liver tumors without using a scalpel or needles, hopefully enabling the patient’s quick recovery while avoiding certain complications like surgical site infections or radiation illness common with other modalities,” commented Joe Amaral MD, VP Medical Affairs for HistoSonics. “Based on the data and patient experiences in our studies we are confident histotripsy will have a meaningful impact for patients suffering from unresectable liver disease, including liver tumors, and we look forward to the role histotripsy will play in treatment strategies going forward,” added Amaral.

    The Edison System is indicated for the non-invasive destruction of liver tumors, including unresectable liver tumors, using a non-thermal, mechanical process of focused ultrasound.

    About HistoSonics

    HistoSonics is a privately held medical device company developing a non-invasive platform and proprietary sonic beam therapy utilizing the science of histotripsy, a novel mechanism of action that uses focused ultrasound to mechanically destroy and liquify unwanted tissue and tumors. The company is currently focused on commercializing their Edison System in the US and select global markets for liver treatment while expanding histotripsy applications into other organs like kidney, pancreas, and others. HistoSonics has offices in Ann Arbor, Michigan and Minneapolis, MN.

    For more information please visit: www.histosonics.com/.

    Contacts

    Media contact:
    Josh King
    Vice President of Marketing
    Email: Joshua.king@histosonics.com
    Phone: 608.332.8124

    Deka Biosciences Closes USD $20 Million Series B2 Financing Led by MPM BioImpact

    September 28, 2023 / Portfolio News

    Sept. 28, 2023 — Maryland-based biotech company Deka Biosciences (“Deka”) today announced that it has successfully closed a USD $20 Million Series B2 financing with a syndicate of life science investors led by MPM BioImpact, and joined by additional investors including Leaps by Bayer, Lumira Ventures, O-Bio (Echo Investment Capital), Viva BioInnovator, Alexandria Venture Investments, Amana Investments, Plains Ventures, ATEM Capital and CEO John Mumm. The proceeds of the financing will support the advancement of Deka’s pipeline and drug product manufacturing as they continue clinical trials following the receipt of a notice to proceed letter from the FDA for their investigational new drug (IND) application to evaluate DK210 (EGFR). Additionally, Detlev Biniszkiewicz, Ph.D. of MPM-BioImpact, will join the Deka board.

    “This financing round is a significant milestone. We appear to be the only company that has dissociated IL-2 potency from toxicity,” said Dr. John Mumm, CEO of Deka. “It is gratifying to be supported by investors who recognize the potential of our innovative science to radically improve immunotherapy treatments by evaluating each patient’s immune system and combining biology with the power of precision medicine.

    “Deka’s unique vision of rationally combining multiple cytokines in a single therapeutic, and the potential impact this could have for patients suffering from cancer and autoimmunity, was deeply intriguing to the MPM BioImpact team,” said Dr. Detlev Biniszkiewicz, Managing Director at MPM BioImpact. “We are impressed by the ability of DK210 to improve the safety parameters of IL-2 while maintaining potent anti-tumor responses in cancer patients, and we are thrilled to be working with the Deka team in developing their innovative approach.”

    Deka Biosciences Pre-filled Syringes containing DK2^10 (EGFR) (PRNewsfoto/Deka Biosciences)

    The funding follows the seed round investment of USD $5 million led by CEO John Mumm, Series A financing of USD $20 million announced in November 2021, co-led by Leaps by Bayer and ECHO Investment Capital, and a USD $21.5 million Series B1 co-led by Lumira and Leaps by Bayer in 2022. The previous financings have enabled Deka to advance a pipeline of multiple DiakinesTM, conduct critical IND enabling experiments, manufacture drug substance/drug product with Cytovance Biologics, start the first Phase I clinical trial, as well as to expand into a new facility.

    About Deka Biosciences
    Deka Biosciences is a biotech company led by entrepreneur Dr. John Mumm, who is backed by a team of experienced academic, biopharma and CDMO innovators with expertise in drug discovery, product development, characterization, and testing. Deka has developed disease specific Diakines™ designed to maximize patient benefits through improved pharmacokinetics / pharmacodynamics function by the targeted accumulation of dual and complimentary cytokines into affected tissues. Through developing a better understanding of each patient’s immune response to different Diakines™, Deka hopes to maximize the impact of its Diakines™ by building specific targeted therapies for everyone.

    SOURCE Deka Biosciences

    enGene Appoints Richard Bryce, MBChB, MRCGP, MFPM, as Chief Medical Officer

    September 7, 2023 / Portfolio News

    enGene, Inc., a clinical-stage biotechnology company mainstreaming gene therapy through its novel platform for the delivery of therapeutics to mucosal tissues and other organs, today announced the appointment of Richard Bryce, MBChB, MRCGP, MFPM as its Chief Medical Officer, effective September 19, 2023. Dr. Bryce will oversee the clin­i­cal develop­ment of EG-70, enGene’s lead product candidate for non-muscle invasive bladder cancer (NMIBC), as well as the devel­op­ment strat­e­gy for enGene’s ther­a­peu­tic pipeline of tissue-targeted non-viral gene therapies.

    “We are thrilled to have Dr. Bryce join enGene during a pivotal period in our growth as we advance our Phase 1/2 registrational LEGEND study for EG-70 in NMIBC,” said Jason Hanson, CEO of enGene. “We believe that the potential efficacy and ease of administration of EG-70 could transform how NMIBC is managed, and Dr. Bryce’s extensive track record advancing oncology therapeutics through late-stage trials and approval makes him well positioned to lead this program, while also leveraging our Dually Derivatized Oligochitosan (DDX)® platform to expand our pipeline.”

    Before joining enGene, Dr. Bryce was most recently Chief Medical Officer at Rain Oncology, a late-stage company developing precision oncology therapeutics, where he built a clinical development team and oversaw multiple clinical studies, including a global Phase 3 registrational study in dedifferentiated liposarcoma. Prior to that, he was Chief Medical & Scientific Officer at Puma Biotechnology, where he led the development strategy for neratinib. His leadership of this program resulted in FDA, EMA and multiple other global approvals, a diverse clinical development program, and an active translational program with several hundred scientific and clinical publications during his tenure. Earlier in his career, Dr. Bryce was Senior Director of Clinical Science for Onyx Pharmaceuticals, where he oversaw the Phase 3 registrational studies for carfilzomib.

    “I am excited to join the enGene team and share in its relentless focus on patient needs and dedication to the translation of innovative science into practical non-viral genetic medicines,” said Dr. Bryce. “I believe EG-70 has the potential to change the treatment paradigm for NMIBC patients and clinicians, and I look forward to advancing the pivotal LEGEND study and expanding our pipeline of non-viral genetic medicines.”

    Dr. Bryce obtained his Bachelor of Medicine and Bachelor of Surgery (MBChB) Degrees from the University of Edinburgh and is certified in the EU in primary care/general practice and pharmaceutical medicine. He holds numerous postgraduate specialist clinical qualifications including those from the Royal College of Obstetricians & Gynaecologists (RCOG) and the Royal College of Physicians (RCP). He also served as a Surgeon Lieutenant Commander in the Royal Navy.

    About enGene Inc.

    enGene Inc. is a clinical-stage biotechnology company mainstreaming gene therapy through its novel platform for delivery of therapeutics to mucosal tissues and other organs, potentially creating new ways to address diseases with high unmet clinical needs. Our proprietary Dually Derivatized Oligochitosan (DDX)® platform is designed to enable a carrier to penetrate mucosal tissue and deliver a wide range of sizes and types of cargo, including DNA and various forms of RNA, by turning mucosal cells into therapeutic production “factories” that generate proteins and RNAs for a desired local effect. This platform will enable localized treatment specifically to the target site without the immunogenicity and systemic effects typical of viral vector-based genetic medicines, potentially expanding gene therapy to multiple clinical settings.

    About enGene’s EG-70 Program

    enGene’s lead product candidate, EG-70, is a non-viral immunotherapy comprised of three gene cargos delivered via our proprietary DDX platform; it is being evaluated as a monotherapy in a registrational study to treat NMIBC patients with carcinoma-in-situ (Cis) who are unresponsive to treatment with Bacillus Calmette-Guérin (BCG). It is also being studied as a monotherapy to treat NMIBC patients with Cis who are naïve to BCG. EG-70 is applied by intravesical administration and encodes two RIG-I agonists intended to stimulate the innate immune system and IL-12 to stimulate the adaptive immune system. With cargoes selected to stimulate both arms of the immune system, EG-70 is designed to generate a strong local immune reaction in proximity to tumors, enabling the immune system to reduce or clear the tumor and develop memory to resist recurrence without significant systemic toxicities.

    About the LEGEND study

    The LEGEND study, both first-in-human and first-in-class, is a Phase 1/2 open-label, monotherapy, multi-center, dose-escalation trial evaluating the safety and tolerability, pharmacokinetics, pharmacodynamics, and efficacy of EG-70 administered by intravesical instillation. To learn more about the first-in-human clinical trial of EG-70 in BCG-unresponsive NMIBC, please visit ClinicalTrials.gov. For additional information about the LEGEND study, please visit TheLegendStudy.com.

    Note regarding forward-looking statements

    This press release contains certain “forward-looking statements” that reflect enGene’s beliefs and assumptions based on currently available data and information. These forward-looking statements can be identified by words such as: “target,” “believe,” “expect,” “will,” “may,” “anticipate,” “estimate,” “would,” “positioned,” “future,” and other similar expressions that predict or indicate future events or trends or that are not statements of historical matters. Forward-looking statements in this press release may include, for example, statements regarding: enGene’s research and development programs, regulatory and business strategy, future development plans, and management; enGene’s ability to advance product candidates and the timing or likelihood of regulatory filings and approvals. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based only on enGene’s current beliefs, expectations, and assumptions that, by definition, involve risks and uncertainties that are difficult to predict and are subject to factors outside of management’s control, and that could cause actual results to differ substantially from statements made including but not limited to: risks associated with the success of preclinical studies, clinical trials, research and development programs, as well as regulatory approval processes. Actual results and outcomes may differ materially from those indicated in the forward-looking statements. enGene has no approved drugs available for sale marketing at this time and may never have an approved drug. You are cautioned not to rely on enGene’s forward-looking statements, which are only made as of the date hereof. enGene is under no obligation to update these statements.

    Additional Information and Where to Find It

    The information provided herein may be relevant to the proposed business combination between enGene and Forbion European Acquisition Corp. (“FEAC”) (Nasdaq: FRBN), In connection with the business combination agreement and the proposed transaction, enGene Holdings Inc. (“Newco”), which will be the surviving public company of the business combination, has filed with the U.S. Securities and Exchange Commission (the “SEC”) a registration statement on Form S-4, which includes a preliminary proxy statement/prospectus, and this communication is not intended to be, and is not, a substitute for the proxy statement/prospectus or any other document that Newco or FEAC has filed or may file with the SEC in connection with the proposed business combination. After the registration statement on Form S-4 has been declared effective, the definitive proxy statement/prospectus will be mailed to shareholders of FEAC as of a record date to be established for voting on the proposed business combination and the other proposals regarding the proposed business combination set forth in the proxy statement/prospectus. Before making any voting or investment decision, investors and shareholders of FEAC are urged to carefully read the entire proxy statement/prospectus, when available, as well as any amendments or supplements thereto, because they will contain important information about the proposed business combination. FEAC investors and shareholders will also be able to obtain copies of the preliminary and definitive proxy statements/prospectuses, without charge, once available, at the SEC’s website at www.sec.gov, or by directing a request to: Forbion European Acquisition Corp., Gooimeer 2-35, 1411 DC Naarden, The Netherlands, Attention: Cyril Lesser.

    Participants in the Solicitation

    FEAC, enGene, Newco and their respective directors, managers, executive officers, other members of management and employees may be deemed participants in the solicitation of proxies from FEAC’s shareholders with respect to the proposed business combination under the rules of the SEC. FEAC’s investors and security holders may obtain more detailed information regarding the names and interests in the proposed business combination of FEAC’s directors and officers, without charge, in FEAC’s filings with the SEC, including the preliminary proxy statement/prospectus and the amendments thereto, the definitive proxy statement/prospectus, and other documents filed with the SEC. Such information with respect to enGene’s and Newco’s directors and executive officers will also be included in the proxy statement/prospectus.

    No Offer or Solicitation

    This press release is not a proxy statement or solicitation of a proxy, consent or authorization with respect to any securities or in respect of the proposed business combination and will not constitute an offer to sell or exchange, or a solicitation of an offer to buy or exchange, any securities (including securities of enGene, FEAC, Newco or the combined company), nor will there be any sale of securities in any states or jurisdictions in which such offer, solicitation, sale or exchange would be unlawful prior to registration or qualification under the securities laws of any such jurisdiction.

    SOURCE enGene

    Molly Shoichet Joins PRIME as New Scientific Director for Precision Medicine Initiative

    August 28, 2023 / Portfolio News

    AmacaThera Co-Founder, Chief Scientific Officer and University Professor Molly Shoichet is the new Scientific Director of PRiME Next-Generation Precision Medicine, a University of Toronto (U of T) Strategic Initiative based at the Leslie Dan Faculty of Pharmacy. As an accelerator for precision medicine, PRiME brings together multi-disciplinary research talent and innovators to tackle unmet needs in drug discovery, diagnostics, and disease biology.

    “It is such an exciting time in precision medicine,” says Shoichet, who is the Michael E Charles Professor of Chemical Engineering at U of T. “We know that a one size fits all approach doesn’t work well in medicine. We can now take advantage of advances in omics – genomics, metabolomics, proteomics – AI, and engineered materials to design therapeutics more precisely for individual needs. From a research and translation perspective, it’s crucial that we address not just the complexity of disease but how we can diagnose and do a better job delivering these new treatments to patients.”

    Since launching in 2019, PRiME has grown to include more than 90 faculty from 16 departments across U of T’s three campuses, with more than 200 graduate trainees connected to the initiative. Under the leadership of Shoichet, PRiME will focus on expanding translation of research advances through collaboration with U of T’s partner hospitals and industry colleagues, growing PRiME into a hub  to enable researchers to develop new solutions for key clinical challenges.

    “U of T is a powerhouse in biomedical research that is recognized throughout the world,” says Shoichet. “This is strengthened not only by researchers in fields like pharmacy and engineering, but by being part of a broad ecosystem that is uniquely poised to accelerate new solutions for unmet needs in human disease. PRiME brings the diversity of ideas into solutions-focused, multidisciplinary research that will help us move the needle.”

    For example, PRiME principal investigators Professors Stéphane Angers and Molly Shoichet are collaborating with SickKids Senior Scientist Dr. Peter Dirks to tackle glioblastoma – the deadliest primary tumour for which there are no therapies. Working with commercialization partner TIAP and industry partner Amgen, PRiME scientists are identifying new therapeutic targets for glioblastoma by combining their expertise in gene editing and specifically-designed hydrogels. Shoichet is known internationally for innovative research in drug delivery, drug discovery and hydrogels. Materials and techniques invented by Shoichet and her team aim to both promote tissue repair in the brain and spinal cord and discover new drugs in cancer. An Officer of the Order of Canada and Fellow of the Royal Society (UK), Shoichet was awarded Canada’s most prestigious award for science and engineering in 2020 – the Gerhard Herzberg Canada Gold Medal for Science.

    As the new Scientific Director of PRiME, with a cross-appointment at the Leslie Dan Faculty of Pharmacy, Shoichet is excited to bring her passion for research and translation to the initiative.

    “The Toronto biotech ecosystem is thriving,” says Carolyn Cummins, Associate Professor in the department of pharmaceutical sciences at the Leslie Dan Faculty of Pharmacy and the Associate Scientific Director of PRiME. “I am excited to work closely with Professor Shoichet to deliver the potential of PRiME at this pivotal time.”

    In the coming months, PRiME will launch many new programs and events for PIs and trainees to build inter-disciplinary and translational collaborations. This includes the new PRiME Inter-Disciplinary Catalyst Program and Fellowships in fall 2023, with partners from across the Toronto drug discovery community.

    Fall 2023 Lumira Venture Innovation Program (VIP): Venture Fellow Applications Open

    August 23, 2023 / Lumira News

    Looking to make a career in life sciences innovation? Our Venture Innovation Program (VIP) is accepting applications for exceptional students across Canada who want to gain exposure to life sciences venture capital and start-ups, and are committed to pushing the future of life sciences innovation forward. By working hand in hand with seasoned industry professionals from all dimensions of the life sciences ecosystem, you will gain first-hand knowledge and insights into the challenges, opportunities, processes, and people shaping the future of healthcare innovation in Canada and around the globe.

    Venture Fellow:

    The Lumira VIP Fellow Position is a 6-12 month program for Ph.D., MD, and MBA students who are motivated build careers in life sciences innovation. The program is designed to be completed part-time alongside your academic program and to offer you the opportunity to participate in the full lifecycle of life sciences venture innovation and investment via different engagements across science, medicine, business, strategy, and operations. The program can be completed remotely from anywhere in Canada, with periodic opportunities to interact in-person at one of our Canadian offices. 

    Application Deadline: September 17th, 2023

    Frequently Asked Questions

    Who is Lumira Ventures?

    Lumira Ventures is an impact investor that has consistently delivered first quartile financial returns to investors in its funds, while also delivering medical innovations that impact patient lives and healthcare ecosystems globally. Our portfolio companies have brought more than 50 biomedical innovations to the market impacting the lives of over 1 billion patients, and generating over $70 billion of cumulative revenue.

    Why did Lumira Ventures launch the LV VIP?

    From the seed investment all the way through to growth stage investment, we have partnered with entrepreneurs in Canada and throughout North America to develop and commercialize truly transformative products that address unmet patient needs and improve the lives of patients worldwide. As Canada is at the foundation of Lumira’s business, we feel privileged to be an active builder of Canada’s biotech and life sciences ecosystem. As a function, we recognize the growing need to identify and support the development of next generation of leaders and innovators within Canada’s high-growth life sciences ecosystem.

    Am I eligible to apply for LV VIP?

    • Canadian citizen or Permanent Resident of Canada
    • Student in the final years of a PhD, MBA or MD program or recent graduates engaged in post-doctoral research or residency programs, with a strong background in the life sciences.

    How much of a time commitment is ideal and is the program paid?

    To get the true benefit of the program, the candidates are expected to participate for at least 6 months, and commit 10-15h per week. Participants must be available to join some meetings during business hours, the timing of which can be flexible. Participants will also be entitled to a monthly stipend.

    Where does the program take place?

    Candidates can work remotely from anywhere in Canada and will have opportunities for in-person interactions with the Lumira team if logistics allow.

    VIP Alumni Experiences

    Thryv Therapeutics announces FDA clearance for Phase 1 Study of THRV-1257 in Anaplastic Thyroid Cancer and acceptance of Late-Breaking Poster Presentation at American Thyroid Association Meeting.

    August 15, 2023 / Portfolio News

    Montreal, Quebec – August 15, 2023 – Thryv Therapeutics Inc., a clinical stage biotechnology company developing therapies for rare diseases including Congenital Long QT Syndrome (LQTS), atrial fibrillation, and resistant cancers, announced today FDA clearance of its Investigational New Drug application (IND) for THRV-1257.  THRV-1257 is being investigated for the treatment of advanced Anaplastic Thyroid Cancer (ATC), including those patients with the common BRAF mutation V600E.  The first in human study will determine the optimal dosing of THRV-1257 in patients with solid tumors, followed by treatment in combination with approved cancer therapies.   

    At the same time, the company announces acceptance of a late breaking poster that will be presented at the upcoming American Thyroid Association (ATA) annual meeting in Washington DC, on September 30th, 2023, from 10:00 a.m. to 1:00 p.m. The presentation will highlight in vitro and in vivo preclinical results of SGK1 inhibition in models of ATC.

    “Preclinical evaluation of our SGK1 inhibitors uncovered novel biology and synergy with existing cancer treatments to extend their activity and reverse resistance.  These studies have revealed a significant opportunity to intervene in several oncology treatment paradigms with an SGK1 inhibitor” Eric Campeau, Vice President, Translational Research.

    According to the ATA, approximately 64,000 people in the United States are diagnosed with thyroid cancer each year. ATC makes up approximately 2% of these cases.  Although ATC is rare compared to other thyroid cancers, it is one of the fastest growing and most aggressive of all cancers. Rapid evaluation and diagnosis are critical for ATC patients, as the disease manifests as a rapidly growing neck mass that impairs speech, swallowing and breathing. Activation of Serine and Glucocorticoid Kinase 1 (SGK1) was determined to be a critical component of ATC cell proliferation, including in tumor cell lines with mutated BRAF. Inhibition of SGK1 was unique in its capacity to suppress ATC cell proliferation compared to other inhibitors tested. Thryv Therapeutics is collaborating with expert scientists and oncologists in ATC to evaluate its SGK1 inhibitors as a potential treatment option to improve the outcome of people with this devastating disease.

    “We are excited to advance our portfolio of potent SGK1 inhibitors into the treatment of aggressive, treatment resistant cancers.  SGK1 has been implicated in a number of treatment-resistance oncology pathways and our work has demonstrated the potential to delay resistance and restore activity of approved therapies and ultimately improve progression free and overall survival outcomes,” Debra Odink, President, and Chief Development Officer.

    About Thryv Therapeutics Inc.

    Thryv Therapeutics Inc. (previously LQT Therapeutics Inc.) is a privately owned company based in Montreal, Quebec, Canada.  Thryv Therapeutics is pioneering a precision medicine approach to treat genetic and drug-induced Long QT Syndromes, atrial fibrillation, and resistant cancers with potent and selective inhibitors of Serum Glucocorticoid inducible Kinase. For more information, please visit www.thryvtrx.com.

    Media Inquiries

    daphne@thryvtrx.com +1 (514) 973 0915

    A 5-Year, $50k Pledge: Supporting Mélanie’s Way on Their Mission to Impact the Lives of Women with Metastatic Cancer and Their Families

    June 13, 2023 / Lumira News

    Mélanie’s Way is a Canadian charity dedicated to granting personalized ‘wishes’ to send women battling cancer and their families on trips of a lifetime. The mission was inspired by Mélanie Chalmers who refused to be defined by the cancer that took her life. Mélanie had the courage to choose the exceptional, and to pursue amazing experiences in the face of great personal cost and risk, which she continued to do up until her final days. Today, Mélanie’s Way is run by over 30 volunteers across Canada and serves young women between the ages of 18 and 45 who have metastatic or relapsed cancer.

    In 2022, Lumira Ventures provided a donation to Mélanie’s Way to send Kristen, a young woman with metastatic breast cancer, and her family on a trip that allowed them to create incredible memories to last a lifetime. Recognizing the profound impact that the wishes have on these families, Lumira Ventures is now partnering with Mélanie’s Way to fund one wish per year for the next 5 years, committing to a total donation of $50,000. Lumira Ventures is thrilled to continue our support for this mission that will help grant more transformative experiences, create unforgettable memories, reignite hope, and instill a renewed sense of joy for these brave women and their families.

    “Mélanie’s Way is so grateful to Lumira Ventures for the support they have given – and their commitment to continue their support in the years ahead. Thanks to Lumira’s support, each year Mélanie’s Way will be able to create a personalized ‘wish’ experience for a young woman with terminal cancer to share with her loved ones. These wish experiences will create moments of joy for these women – and lasting memories for those they will leave behind. On a personal level, we are grateful to Nikhil Thatte from Lumira who created the ‘spark’ that led to the generous commitment from Lumira. What started with his personal attendance to our events and generous support led to conversations with Lumira’s leadership team, a shared understanding of the impact we could have together, and now a tangible impact on the lives of one family a year for the next several years. It’s a great example of what can happen when an individual decides to make a difference,”

    Rob Chalmers, Co-Founder of Mélanie’s Way

    Our pledge to contribute $10,000 annually over the next five years is a commitment to not only help execute the immediate mission of Mélanie’s Way but also to create a ripple effect in the lives of countless others and bring forward the power of philanthropy with the potential for positive change. With that, on Tuesday, July 20th, 2023, Mélanie’s Way is hosting its Spring into Summer Social aimed to bring community together and share stories from recently-completed and soon-to-happen wish experiences they have helped to create. We are also thrilled to have Senior Principal, Nikhil Thatte share a few words on our journey with Mélanie’s Way. Lumira Ventures invites its LPs, partners, and friends to join us at this event to see the profound positive impact that Mélanie’s Way is having on women with cancer and their families.

    Mélanie’s Way Spring into Summer Social Details: https://stacktmarket.com/event/spring-into-summer

    DAMONA Pharmaceuticals Appoints John Reilly as Chief Executive Officer

    June 13, 2023 / Portfolio News

    TORONTO, June 13, 2023 — DAMONA Pharmaceuticals, a preclinical biopharmaceutical company focused on the discovery and development of first-in-class small molecules for the treatment and prevention of cognitive symptoms associated with brain disorders and aging, today announced the appointment of John Reilly as the company’s Chief Executive Officer and member of the Board of Directors.

    Mr. Reilly brings two decades of leadership experience building and creating value for biotechnology companies, with deep knowledge and expertise in corporate strategy, business development, and leading teams in preclinical R&D and clinical development of innovative therapies.

    “We feel very fortunate to have attracted John into the role of CEO for Damona. He brings a wealth of biotechnology company-building experience and a vast network of North American investor and partner relationships, which will be essential in bringing our lead asset toward IND and clinical human proof-of-concept trials,” said Etienne Sibille, Ph.D., Co-Founder and Chief Scientific Officer of DAMONA.

    “I’m ecstatic to have John join the DAMONA team as CEO. John brings a great background to DAMONA with a proven track record of building and managing high-performance preclinical and clinical biopharmaceutical teams that deliver results,” said Jacki Jenuth, Partner and Chief Operating Officer of Lumira Ventures.

    John Reilly is the new Chief Executive Officer of DAMONA Pharmaceuticals

    Prior to joining DAMONA, Mr. Reilly co-founded and served as President and CEO of Apic Bio, a gene therapy company developing first-in-class treatments for rare neurological and liver diseases. At Apic, he secured multiple funding rounds, oversaw development programs, and orchestrated a global licensing agreement with uniQure for the company’s lead program to treat superoxide dismutase 1 (SOD1), a rare, genetic form of ALS. Previously, Mr. Reilly held a number of senior management positions at various biopharmaceutical companies, including Vice President of Business Development at Tetragenetics, which was acquired by Abcellera (Nasdaq: ABCL). Prior to Tetragenetics, he co-founded and served as CEO of Gendyne Therapeutics. Mr. Reilly holds a B.A. from Hamilton College and M.S. and M.B.A. degrees from Cornell University.

    “I am very excited to join DAMONA as CEO and look forward to working with the board, management, and scientific team to continue the company’s impressive progress as we advance the preclinical pipeline and lead program for the treatment of cognitive symptoms associated with depression and diseases of aging into a Phase 1 clinical study,” said John Reilly, CEO of DAMONA Pharmaceuticals.

    About DAMONA Pharmaceuticals
    DAMONA is a privately held preclinical biopharmaceutical company developing small molecule therapeutics that transform the treatment of cognitive deficits and restore normal brain functions for underserved and understudied mental health conditions and for aging populations. DAMONA was founded by Etienne Sibille, Ph.D., and the Centre for Addiction and Mental Health (CAMH). In 2022, DAMONA closed a seed financing round co-led by Lumira Ventures and the Noetic Fund. For more information, please visit: www.damonapharma.com.

    Contact:
    Mary Moynihan
    M2Friend Biocommunications
    +1 (802) 951-9600
    mary@m2friend.com

    SOURCE DAMONA Pharmaceuticals

    Managing General Partner, Peter van der Velden Wins the 2023 Barry Gekiere Lifetime Legacy Award

    May 24, 2023 / Lumira News

    Throughout his more-than-30-year career, Peter van der Velden’s focus has not only been on investing, but also on making a lasting impact in healthcare. Although he initially contemplated becoming a doctor, he soon realized that he was more interested in the business side of healthcare innovation than he was in applied medicine. After completing his graduate studies, he began his career working in a business operations role for a large pharmaceutical company before eventually pivoting into venture capital. 

    “I’ve spent the past 30 years as an operator, investor, and partner in innovative life sciences companies,” says van der Velden. ​“What I love about what I do is that it combines performance-based investing with high impact. Many of the CEOs I’ve worked with have built transformative medical products that have literally changed people’s lives.”

    After stints at Vencap Equities Alberta LTD and Bedford Capital, in 2005, van der Velden was recruited to MDS Capital to restructure the firm. Having done so successfully, he led a management buyout of MDS and formed Lumira Ventures two years later. Today, Lumira is the most active life sciences investor in Canada with an impressive array of portfolio companies across North America. In 2021, the firm raised $350 million via two new funds, one of which is the largest dedicated life sciences fund ever raised in Canada by a Canadian GP. 

    Over the years, van der Velden has been actively involved in an array of successful private and public companies, as well as other entities and initiatives aimed at supporting and promoting life sciences innovation. These include Life Sciences Ontario, OBio, Ontario Hospital Innovation Council, and The World Health Innovation Network to name just a few. ​“Peter is an exceptional strategic thinker, a visionary, and an extraordinary operator,” says colleague Benjamin Rovinski, a Managing Director at Lumira. ​“He’s one of the main pioneers of the life sciences VC industry in Canada.”

    Between 2008 and 2016, van der Velden also held senior roles at the CVCA, where he worked closely with the Federal Government on the implementation of the first Venture Capital Action Plan (VCAP). ​“As the head of the CVCA at the time, Peter worked closely with all stakeholders to ensure that the VCAP program reflected the needs of the community and that it could achieve its goal of helping to restart the Canadian venture capital industry after the disengagement of traditional sources of LP capital,” notes Rovinski. ​“He was deeply committed to the idea of a performance-driven VC industry as the foundation for Canada building a thriving innovation-driven ecosystem.”

    During his time at the CVCA, van der Velden also led a transformative reorganization and repositioning of the CVCA that included transitioning from third-party data providers to the CVCA owning and controlling its own data. He also led the search committee that hired Mike Woollatt as the CVCA’s new CEO, and worked closely with Woolatt and the Executive Committee to implement the new vision for the CVCA, leading to significant membership growth and community engagement.

    In addition to leading Lumira’s investment strategy, van der Velden is also a key driver of Lumira’s philanthropic strategy. In 2018, for example, the firm launched an initiative whereby the firm’s partners donated 10 percent of their Fund III carry to a dozen of Canada’s leading healthcare foundations. More recently, the firm has also actively supported more than 30 businesses and charities in its local ecosystem impacted by Covid and also established the Lumira Foundation to support the firm’s ongoing philanthropic work. 

    “Peter is a proud Canadian who cares about making an impact in Canada,” says Rovinski. ​“He fundamentally believes in the importance of giving back to the community through philanthropic acts that have a material impact.”

    For his tireless efforts over the past three decades, van der Velden has been named the winner of the 2023 Barry Gekiere Lifetime Legacy Award. ​“I’ve been fortunate to be surrounded by fantastic people my entire career,” he says. ​“It’s always been a team effort and I’m just grateful to have been a part of it.”

    For van der Velden, winning the Barry Gekiere Lifetime Legacy Award is particularly touching. ​“Barry was a good friend and someone who I truly respected. He was someone whose personal passion and life’s work were completely aligned, and who always wanted to do the right thing. It’s an honor to receive an award named after him.”

    To that end, van der Velden announced that the Lumira Foundation will be donating $10,000 to the Barry Gekiere Memorial Fund in support of The Leukemia and Lymphoma Society of Canada and The Upside Foundation. 

    Watch the full announcement: https://youtu.be/fVgAv80qIUU

    Cadence Neuroscience Secures $26 Million in Series B Financing for Clinical Trials of Epilepsy Neuromodulation Therapy

    May 23, 2023 / Portfolio News

    Cadence Neuroscience announced that it has secured $26 million in Series B financing. The round was led by Angelini Lumira Biosciences Fund (co-managed by Lumira Ventures and Angelini Ventures) and included other new investors F-Prime Capital, LivaNova USA, Angelini Ventures, Spectrum Financial Services, and Mayo Clinic, as well as the company’s Series A lead investor JAZZ Venture Partners. Gerry Brunk of Lumira Ventures and Kevin Chu of F-Prime Capital have joined the company’s board of directors.

    Cadence is a clinical-stage company developing a novel neuromodulation therapy for treating pediatric and adult patients with focal drug resistant epilepsy based on research by a Mayo Clinic Neurology and Neurosurgery team, led by Gregory Worrell M.D., Ph.D., lead investigator and Mayo Clinic epileptologist. The therapy utilizes chronic subthreshold cortical stimulation to modulate EEG biomarkers associated with epilepsy to reduce or eliminate seizures. The company will use the Series B funds to complete pivotal clinical studies and pursue FDA clearance.

    “The Cadence team has extensive domain expertise in developing active neural implants and conducting human clinical studies in the field,” said Kent Leyde, Cadence’s co-founder and Chief Executive Officer. “During the last three years, we have been closely collaborating with Mayo Clinic in the design, manufacture, and engineering tests of our therapy system. These efforts are nearing completion and will enable us to begin clinical testing.”

    “As an active investor in both medical devices and pharmaceutical therapies for epilepsy, we believe Cadence is well-positioned to drive important advances in the field of neuromodulation, with the potential to revolutionize the treatment of drug refractory epilepsy, a disease which affects millions of people across the world,” said Gerry Brunk, Managing Director at Lumira Ventures. “We believe Cadence’s platform solution has the potential to become an important standard of care in drug-resistant epilepsy as well as other difficult-to-treat brain disorders,” said Paolo Di Giorgio, CEO of Angelini Ventures. 

    “We think the precise, patient-specific stimulation method being developed by Cadence is unique and exciting,” said Kevin Chu, healthcare team Principal at F-Prime Capital. “We are thrilled to join the team on its important mission to bring life-changing therapies to this debilitating and difficult-to-treat disease.”

    About Cadence Neuroscience
    Cadence Neuroscience is a medical device company developing new therapies for the treatment of epilepsy and other neurological disorders. The company’s core technology was developed at Mayo Clinic and is under clinical evaluation. Founded in 2017 and headquartered in Redmond, Washington, Cadence is led by seasoned executives with extensive backgrounds in neural implant product development and clinical studies.

    About Angelini Lumira Biosciences Fund
    Lumira Ventures and Angelini Ventures partnered to create the Angelini Lumira Biosciences Fund in 2021 to invest in early-stage companies in North America developing innovative therapies for disorders of the central nervous system. Founded in 2007, Lumira Ventures is a multi-sector healthcare venture capital firm with offices in Toronto, Boston, Montreal and Vancouver. Angelini Ventures is the corporate venture capital arm of Angelini Industries, an Italian multi-sector group that operates in 21 countries in the health, industrial technology and consumer goods sectors, with 5,800 employees and revenues of €2 billion. With €300M in investment capital, Angelini Ventures creates and invests in companies developing innovative solutions in the fields of biotechnology, life sciences and digital healthcare.

    Related Links
    https://www.cadenceneuro.com/  
    https://www.lumiraventures.com/ 
    https://fprimecapital.com/
    https://www.jazzvp.com/ 
    https://www.livanova.com/ 
    https://www.angeliniventures.com/ 
    https://businessdevelopment.mayoclinic.org/ 

    Mayo Clinic and Dr. Worrell have a financial interest in the technology referenced in this news release. Mayo Clinic will use any revenue it receives to support its not-for-profit mission in patient care, education and research.

    Media Contact:
    Kent Leyde
    425-298-3184
    359168@email4pr.com 

    Endotronix Extends Positive Outcomes with 12-month Data Release from the SIRONA 2 Trial for Remote Heart Failure Management Using the Cordella Pulmonary Artery Sensor

    May 23, 2023 / Portfolio News

    The study met secondary study endpoints and long-term sensor use was associated with significant improvements in patient quality-of-life metrics and low event rates for heart failure hospitalization and death.

    Endotronix, Inc., a digital health and medical technology company dedicated to advancing the treatment of heart failure (HF), today announced positive 12-month data from the SIRONA 2 clinical trial that demonstrated long-term use of the Cordella Pulmonary Artery (PA) Sensor was associated with significant improvements in patient quality-of-life metrics and low HF hospitalization and death (HFH/D) event rates for New York Heart Failure (NYHA) class III patients. The results expand on the previously published positive 90-day primary endpoint data on device safety and pressure accuracy for the Cordella PA Sensor. The 12-month results were presented on May 20th at the European Society of Cardiology’s annual conference, Heart Failure 2023, by Professor Birgit Aßmus, a heart failure cardiologist at the University of Giessen in Giessen, Germany.

    “Adding to a robust foundation of clinical evidence supporting the use of Cordella, the 12-month SIRONA 2 data further validates the benefit of PA pressure-guided therapy to improve patient outcomes for NYHA class III patients,” stated Prof.  Aßmus. “Cordella’s comprehensive approach consists of PA pressure data plus non-invasive data, including blood pressure, weight, and heart rate, which enables my team to efficiently titrate key HF medications remotely and help patients feel better. By actively monitoring the trended health data, we can bring down the PA pressures to avoid acute HF hospitalizations which delivers meaningful benefits to our overall health system and to patient lives.”

    The Cordella solution is the first and only patient management platform to provide both critical PA pressure data with an implanted sensor, and noninvasive vitals for comprehensive clinical management delivered in the patient’s home. The easy-to-use devices securely transmit a patient’s daily health status for trended insights that support optimal dosing of guideline-directed medical therapy (GDMT) across all types of heart failure. The patient-friendly system engages the patient and allows them to view their trended health data which has been shown in clinical evaluation to support healthy lifestyle choices. The streamlined workflow enhances current clinical practice, providing a scalable solution that enables new treatment standards to reduce and prevent HF congestion.

    SIRONA 2 is an open-label, single-arm trial of 70 European patients that met its primary endpoints for safety and accuracy with the previously reported 90-day results. The 12-month results extended both the strong safety profile, with no PA sensor failures and no additional device/system-related complications (DSRC=1.3%), as well as accurate PA pressure measurement, with good agreement with the gold standard fluid-filled reference catheter (Swan Ganz). The recent results also met secondary endpoints for validated patient quality-of-life metrics and demonstrated low event rates for HF Hospitalization / Death (HFH/D) as indicated below.

    12-Month SIRONA 2 Key Findings

    • Significant improvement in patient quality-of-life metrics, including NYHA classification over 12 months (p<0.0001) and 6-minute walk test distance at both 3 months (p=0.005) and 12 months (p=0.02)
    • Low event rates for HF Hospitalization/Death and only 5 deaths (7.1%) at 12 months
      • Cumulative hazard rate for HFH (0.27) and HFH/D (0.33)
    • 78.6% reduction in HFH rate in the year following implant compared to the year prior
    • Consistently high patient compliance with daily submissions of heath data – 95% at 12 months

    “We are thrilled with the significant improvement in patient quality of life and low event rates demonstrated in the 12-month SIRONA 2 data, which sets the stage for upcoming PROACTIVE-HF trial results in early next year,” commented Harry Rowland, CEO of Endotronix. “Together, this clinical evidence will serve as the foundation for our upcoming commercial launch and enable clinicians to improve patients’ lives for the better.”

    About Endotronix

    Endotronix innovates at the intersection of Medtech & Digital Health to improve care for people living with heart failure (HF).  The comprehensive Cordella solution enables proactive, data-driven HF management that engages patients, reduces and prevents congestion, and improves outcomes. The Cordella Sensor is an implantable pulmonary artery (PA) pressure sensor which directly measures the leading indicator of congestion, allowing early, targeted therapy. The Cordella HF System is a patient health management platform, which combines comprehensive vital sign data from non-invasive devices to support patient-clinician engagement and care decisions. Combining trended insights, the versatile and scalable Cordella enhances current clinical practice, and supports guideline-based care across the entire HF continuum. Learn more at www.endotronix.com.

    The Cordella Sensor is under clinical investigation and is not available for commercial use in any geography. The Cordella HF System, without the sensor, is available for commercial use in the U.S. and E.U.

    Cautionary Statement Regarding Forward-Looking Statements

    This press release may contain predictions, estimates or other information that might be considered forward-looking statements. Such forward-looking statements are not a guarantee of future performance.

    SOURCE Endotronix, Inc.

    Antiva Biosciences Closes $53 Million Series E Equity Financing Led by MPM-BioImpact Capital and Names Kristine Ball President and CEO

    April 27, 2023 / Portfolio News

    Proceeds to Support Key Efficacy Studies for ABI-2280 including Phase 2 Trial in High-Grade Cervical Intraepithelial Neoplasia (CIN 2,3) and Exploratory Study as Treatment for High-Risk HPV Infections

    Antiva Biosciences, Inc. biopharmaceutical company developing novel, topical therapeutics for the treatment of pre-cancerous lesions caused by human papilloma virus (HPV) infection, today announced the closing of a $53 million Series E equity financing. The financing was supported by a syndicate of premier life science investors led by MPM-BioImpact Capital, and joined by the company’s existing investors including Canaan Partners, Sofinnova Investments, Adjuvant Capital, GV and Lumira Ventures, among others. In conjunction with the financing, president and chief executive officer Gail Maderis is transitioning to chairman of the company’s board of directors and is being succeeded as CEO by Kristine Ball. Additionally, Ms. Ball, along with Florencia Segal, M.D., and Brian Goodman, Ph.D., both of MPM-BioImpact Capital, will join the Antiva board.

    Proceeds from the financing, which will fund the company into late 2025, will support the advancement of the company’s lead development candidate, ABI-2280, into key efficacy studies following completion of its ongoing Phase 1 trials. Planned studies include a Phase 2 clinical trial in high-grade cervical intraepithelial neoplasia (CIN 2,3) and an exploratory study as a potential treatment for high-risk HPV infection, specifically in subtypes that can lead to cancer. ABI-2280 has potent antiviral activity across all serotypes of HPV and works by both directly blocking HPV replication and inducing apoptosis in HPV-infected lesions, while sparing normal cells. Antiva has leveraged its development expertise to formulate a vaginal tablet of ABI-2280 that will enable self-administration at diagnosis and facilitate worldwide distribution, increasing impact potential for patients.

    The treatment of high-risk HPV infections with ABI-2280 holds the potential to address a significant global health challenge. HPV infections account for nearly all cases of cervical cancer around the world and an accessible and effective treatment holds the promise to dramatically reduce these cancers. Currently, there are no treatments available to resolve HPV infections and prevent their development into cancer. This remains a crucial unmet medical need with more than 5.5 million women in United States alone affected by high-risk HPV infections each year despite ready access to vaccines.

    “We believe that Antiva has the opportunity to make a transformative impact on global health across several large, unmet therapeutic indications with its ABI-2280 program and are thrilled to be able to lead this round of financing. The company has advanced the development of ABI-2280 to patients with CIN 2,3 and is now poised to expand clinical development into high-risk HPV infection. ABI-2280 can make the World Health Organization’s screen-and-treat approach to HPV infection a reality,” said Dr. Segal.

    Dr. Goodman added, “With MPM-BioImpact’s growing focus on the field of virology, Antiva represents the type of impactful investment that we are looking to make in this space. We look forward to supporting Kristine in her new role as CEO and seeing the continued progress that is made under her leadership.”

    Ms. Ball joins Antiva with more than 20 years of executive experience in the life sciences industry across companies of all stages ranging from discovery to clinical to commercial. She has significant transactional expertise, having participated in multiple initial public offerings, corporate M&A deals, partnerships and financings. Ms. Ball most recently served as chief executive officer of Soteria Biotherapeutics, a venture backed immuno-oncology company and previously held leadership positions at Menlo Therapeutics, Relypsa, Inc., and KAI Pharmaceuticals, and served on the board of Forty Seven, Inc., until its acquisition by Gilead.  Ms. Ball currently sits on the board of Atreca, Inc., a publicly traded, clinical-stage oncology company.

    “I am deeply grateful for this opportunity to join Antiva at such an exciting time in the company’s maturation. ABI-2280 has tremendous potential to play a significant role in advancing both global health and women’s health, bringing about a long-needed breakthrough in HPV infections and HPV-related cancers in both men and women,” stated Ms. Ball. “I am eager to carry forward the significant momentum that has been generated by the Antiva team and look forward to the important upcoming clinical development milestones that will move us closer to this goal. I am also excited to be able to work closely with Gail in her new role as board chair, leaning on the experience and knowledge she has gained during her time as president and CEO.”   

    “I take great pride in turning the CEO role over to Kristine with the company in such a strong position following this transformative financing. She is a talented and experienced life science executive who is perfectly suited to lead Antiva as it expands clinical development of ABI-2280,” said Ms. Maderis. “I look forward to remaining intimately connected to the company in the role of board chair and offer a special thanks to Steve James, who has provided Antiva with great leadership and guidance as board chair and will remain an executive advisor to the company.”

    About HPV-Related Diseases and Cervical Cancer

    Human Papilloma Virus (HPV) is so common that nearly all sexually active men and women are infected with the virus at some point in their lives. Many of these are transient infections the body is capable of clearing, but this typically takes months to years.  When the infections persist, they are known to drive the formation of malignancies, including cervical, anal, vulvar, penile, and head and neck cancers.

    The introduction of prophylactic vaccines for HPV was a major step forward in the fight against HPV-associated cancers by preventing infection by certain high-risk HPV subtypes. However, due to low adoption rates in the US, EU, and Japan, and limited access to the vaccines in developing countries, HPV infections and the disease states driven by such infections remain a major unmet clinical need.

    Globally, cervical cancer is the fourth most common cancer in women and as such represents a major public health problem. According to the World Health Organization, an estimated 604,000 women were diagnosed with cervical cancer worldwide and approximately 342,000 women died from the disease in 2020.

    About Antiva Biosciences
    Antiva Biosciences, Inc. is a clinical-stage biopharmaceutical company developing novel, topical therapeutics for the treatment of diseases caused by HPV infection. The company, based in South San Francisco, was founded in 2012 by Dr. Karl Hostetler of The University of California San Diego. The company’s lead drug candidate, ABI-2280 is being developed as a topical treatment for high-grade cervical intraepithelial neoplasia (HSIL, CIN 2,3) and for women with high-risk HPV infection.

    For more information, please visit: www.antivabio.com.

    Contact Information:

    Kristine Ball
    Antiva Biosciences, Inc.
    650-822-1400
    info@antivabio.com

    Tim Brons
    Vida Strategic Partners (media)
    646-319-8981
    tbrons@vidasp.com

    PIC Therapeutics Presents Pre-Clinical Data Update on eIF4E Regulators at AACR Annual Meeting 2023

    April 19, 2023 / Portfolio News

    PIC Therapeutics, Inc., a biotechnology company pioneering the discovery and development of first-in-mechanism, first-in-class allosteric small molecule therapies targeting eIF4E, is presenting pre-clinical data today on the company’s advancing eIF4E program for breast cancer in a poster at the American Association for Cancer Research (AACR) Annual Meeting 2023, taking place in Orlando, FL.

    Advanced or metastatic breast cancers represent a large patient population with limited long-term solutions that address both the heterogenous nature of resistant tumors and the aggressiveness of their proliferation. The aim of our therapies is to drive strong mechanistic responses, in a majority of breast cancer tumor subtypes, that induce a rapid commitment to cell death. To accomplish this, our approach addresses a key fundamental mechanism in protein translation at the convergence of many oncogenic signaling pathways through which resistance arises.

    eIF4E is a key component of protein translation that often becomes dysregulated in breast cancer, supporting cell survival and metastasis alongside therapeutic resistance. PIC compound regulation of eIF4E induces rapid commitment to apoptosis and selective proteomic shifts that are consistent with canonical eIF4E regulation including cell cycle impacts, e.g. effects on cyclin family members and CDKs, DNA repair regulation and metabolic proteins. PIC Compounds also potently impact multiple clinical drivers for ER+ and Triple Negative Breast Cancers, which are evidenced by mRNA signatures that include changes consistent with proteomic impacts to key transcription factors.

    Previous communications revealed our eIF4E regulators potently cause cell death in primary breast cancer organoid models, but spared models derived from healthy tissue. Expanding our efforts in this domain, the impact on normal immune cells was evaluated by exposing PBMCs, CD4+ T cells, and bone marrow mononuclear cells to our compounds. Our eIF4E regulators did not impair survival of these cells up to a maximum tested dose of 25 micromolar. Primary CD4+ T cells were also found to be functional in the presence of eIF4E regulators up to 5 micromolar, which included assessments of CD3/CD28 stimulated cytokine production and proliferative responses, highlighting its remarkable differentiated impact on normal cells compared to tumor cells.

    “We continue to uncover interesting and distinctive preclinical data sets that underscore the potential for unique therapeutic advantages of allosterically regulating eIF4E, as a promising strategy for patients with various subtypes of breast cancer,” said Kathy Bowdish, Ph.D., President and Chief Executive Officer of PIC Therapeutics.

    Our findings suggest that allosteric regulation of eIF4E provides a unique and efficient way to address multiple resistance mechanisms while sparing normal immune cellular function. Our eIF4E regulators represent a potential beneficial therapeutic approach to address multiple resistant cancer patient populations and fulfill the promise of this elusive target.

    Presentation details:
    Title:
    eIF4E allosteric regulators cause rapid commitment to apoptosis in cancer cells while sparing immune cells
    Session Category: Experimental and Molecular Therapeutics
    Session Title: Novel Antitumor Agents 3
    Session Date and Time: Monday April 17, 9:00 AM – 12:30 PM
    Location: Section 17
    Poster Board Number: 14
    Abstract Number: 1624

    About PIC Therapeutics, Inc.

    PIC Therapeutics is a preclinical-stage biotechnology company pioneering the discovery and development of first-in mechanism, first-in-class small molecule medicines focused on fundamentally changing how we treat cancer by developing therapeutics that regulate protein translation in a context dependent manner. PIC Therapeutics targets a “master switch” of cancer signaling pathways, selectively impacting oncogene protein production by altering the Pre-Initiation Complex (PIC) that drives their mRNA translation. PIC Therapeutics’ selective approach regulates cancer cell proteomes, impacting multiple dysregulated oncogenic drivers leading to a powerful new generation of cancer-treating therapeutics. Our agents offer the opportunity to address both drug resistance and tumor heterogeneity, issues that plague many existing treatments. For more information visit www.pictherapeutics.com

    Fusion Pharmaceuticals Announces IND Clearance For FPI-2068, A Jointly Developed Novel Targeted Alpha Therapy

    April 12, 2023 / Portfolio News

    Fusion Pharmaceuticals Inc. (Nasdaq: FUSN), a clinical-stage oncology company focused on developing next-generation radiopharmaceuticals as precision medicines, today announced the clearance of investigational new drug (IND) applications for [225Ac]-FPI-2068 (FPI-2068) and corresponding imaging analogue [111In]-FPI-2107 (FPI-2107) to the U.S. Food and Drug Administration (FDA). Fusion is jointly developing FPI-2068 with AstraZeneca (LSE/STO/Nasdaq: AZN) under the companies’ multi-asset collaboration agreement.

    FPI-2068 is a targeted alpha therapy (TAT) designed to deliver actinium-225 to various solid tumors that express EGFR and cMET. EGFR and cMET are both validated targets that are co-expressed in multiple tumor types, including head and neck squamous cell carcinoma, non-small cell lung cancer, colorectal cancer, and pancreatic ductal adenocarcinoma.

    “The IND filing for FPI-2068 is an important milestone for Fusion as we advance this novel TAT, created by combining our radiopharmaceutical expertise, actinium supply and manufacturing infrastructure with AstraZeneca’s bispecific antibody which preferentially binds to cancer cells that express both EGFR and cMET,” said Fusion Pharmaceuticals Chief Executive Officer John Valliant, Ph.D. “FPI-2068, which we believe will be the first TAT for two validated targets to enter the clinic, was designed to provide enhanced tumor specificity resulting from the co-expression of the two targets when compared to individual monoclonal antibodies against each of these targets. We are excited about the innovative work with AstraZeneca as we advance this and other programs under our broad collaboration agreement.”

    Fusion’s radiopharmaceuticals are a type of precision medicine whereby the cancer-targeted vector (e.g., the bispecific antibody) can be used to screen patients for expression of a tumor biomarker when combined with a corresponding imaging isotope (e.g., indium-111), and subsequently used for therapy when combined with the alpha-emitting radionuclide, actinium-225. Using imaging to identify patients who show uptake of the drug in tumors increases the likelihood of response to therapy. Fusion plans to provide additional guidance on timelines for the FPI-2068 program following initial experience with patient screening in order to better predict the cadence of patient enrollment. 

    FPI-2068 will be the first program to enter clinical development under the Company’s previously announced collaboration agreement with AstraZeneca, which includes joint discovery, development and the option to co-commercialize novel TATs leveraging Fusion’s proprietary Fast-Clear™ linker technology platform with antibodies from AstraZeneca’s oncology portfolio, as well as exploration of potential combination strategies involving existing assets in their respective portfolios. Fusion will be operationally responsible for the Phase 1 study, while AstraZeneca will be responsible for subsequent clinical development. The companies will share costs equally through clinical development.

    About FPI-2068

    [225Ac]-FPI-2068 (FPI-2068) is a targeted alpha therapy (TAT) designed to deliver actinium-225 to various solid tumors that express EGFR and cMET. EGFR and cMET are validated cancer targets that are co-expressed in multiple tumor types, including head and neck squamous cell carcinoma, non-small cell lung cancer, colorectal cancer, and pancreatic ductal adenocarcinoma. FPI-2068 will be evaluated in a Phase 1 study.

    About Fusion

    Fusion Pharmaceuticals is a clinical-stage oncology company focused on developing next-generation radiopharmaceuticals as precision medicines. Fusion connects alpha particle emitting isotopes to various targeting molecules to selectively deliver the alpha emitting payloads to tumors. Fusion’s clinical portfolio includes: FPI-2265 targeting prostate specific membrane antigen (PSMA) for metastatic castration resistant prostate cancer currently in a Phase 2 trial; FPI-1434 targeting insulin-like growth factor 1 receptor currently in a Phase 1 trial; FPI-1966, targeting the fibroblast growth factor receptor 3 (FGFR3), currently in a Phase 1 trial; and FPI-2059, a small molecule targeting neurotensin receptor 1 (NTSR1), currently in a Phase 1 trial. In addition to a robust proprietary pipeline, Fusion has a collaboration with AstraZeneca to jointly develop novel targeted alpha therapies (TATs) and combination programs between Fusion’s TATs and AstraZeneca’s DNA Damage Response Inhibitors (DDRis) and immuno-oncology agents. Fusion has also entered into a collaboration with Merck to evaluate FPI-1434 in combination with Merck’s KEYTRUDA® (pembrolizumab) in patients with solid tumors expressing IGF-1R. To support Fusion’s growing pipeline of TATs, the company has signed strategic actinium supply agreements with TRIUMF, Niowave, Inc. and BWXT Medical.

    Forward-Looking Statements

    This press release contains “forward-looking statements” for purposes of the safe harbor provisions of The Private Securities Litigation Reform Act of 1995, including but not limited to the statements regarding Fusion Pharmaceuticals Inc.’s (the “Company”) future business and financial performance. For this purpose, any statements contained herein that are not statements of historical fact may be deemed forward-looking statements. Without limiting the foregoing, the words “expect,” “plans,” “anticipates,” “intends,” “will,” and similar expressions are also intended to identify forward-looking statements, as are expressed or implied statements with respect to the Company’s potential drug candidates, including any expressed or implied statements regarding the successful development of its product candidates. Actual results may differ materially from those indicated by such forward-looking statements as a result of risks and uncertainties, including but not limited to the following: there can be no guarantees that the Company will advance FPI-2068 in the clinic, through the regulatory process or to commercialization; management’s expectations could be affected by unexpected patient recruitment delays or regulatory actions or delays; uncertainties relating to, or unsuccessful results of, clinical trials; the Company’s ability to obtain additional funding required to conduct its business activities; and changes in the Company’s business plan or objectives. Such forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause actual results to be materially different from any future results, performance or achievements expressed or implied by such statements. These and other risks which may impact management’s expectations are described in greater detail under the heading “Risk Factors” in the Company’s annual report on Form 10-K for the year ended December 31, 2022, as filed with the SEC and in any subsequent periodic or current report that the Company files with the SEC. All forward-looking statements reflect the Company’s estimates only as of the date of this release (unless another date is indicated) and should not be relied upon as reflecting the Company’s views, expectations or beliefs at any date subsequent to the date of this release. While Fusion may elect to update these forward-looking statements at some point in the future, it specifically disclaims any obligation to do so, even if the Company’s estimates change.

    Investors and others should note that Fusion communicates with its investors and the public using the Fusion website, www.fusionpharma.com, including, but not limited to, company disclosures, investor presentations, SEC filings, and press releases. The information that Fusion posts on this website could be deemed to be material information. As a result, Fusion encourages investors, media and others interested to review the information that Fusion posts there on a regular basis.

    Contact:
    Amanda Cray
    Senior Director of Investor Relations & Corporate Communications
    (617) 967-0207
    cray@fusionpharma.com

    SOURCE Fusion Pharmaceuticals

    Biotheryx Announces Research Collaboration and License Agreement with Incyte for Discovery of Targeted Protein Degraders for Novel Oncology Targets

    April 10, 2023 / Portfolio News

    Biotheryx, Inc., a biopharmaceutical company discovering and developing a portfolio of first-in-class protein degraders with a focus on validated targets in cancer and inflammatory disease, today announced it has entered into a Research Collaboration and License agreement with Incyte to discover and develop targeted protein degraders for novel oncology targets.

    “We are pleased to embark on this collaboration with Incyte to identify targeted protein degraders for novel oncology targets. Biotheryx and Incyte share a commitment to finding new, transformative treatment options for people living with cancer,” said Philippe Drouet, President and Chief Executive Officer of Biotheryx. “Our PRODEGY platform is designed to increase efficiency in degrader discovery and design, enabling the development of therapies for previously undruggable targets. We look forward to leveraging this differentiated approach in our collaboration with Incyte and in the continued advancement of our pipeline of first-in-class, next generation bifunctional degraders and molecular glues for the treatment of cancers and inflammatory disease.”

    Under the terms of the agreement, Biotheryx will utilize its distinctive PRODEGY platform to identify and initially develop molecular glue degraders for multiple historically undruggable oncology targets. For the initial target, Biotheryx will receive a technology access fee of $7 million plus up to an additional $6 million in potential research and development funding from Incyte for costs associated with the collaboration. Biotheryx is also eligible to receive potential future regulatory and commercial milestones of up to $347 million plus tiered single-digit royalties on global net product sales for the initial target. Incyte will be solely responsible for further development and commercialization of any molecular glue degraders discovered by Biotheryx’s PRODEGY platform. Additionally, under the terms of the agreement, the collaboration can be expanded under the same financial terms. Further financial terms of the deal were not disclosed.

    “As we work to transform the oncology treatment landscape, Incyte is harnessing breakthrough science that may offer patients with unmet needs new treatment options,” said Dashyant Dhanak, Ph.D., Executive Vice President and Chief Scientific Officer of Incyte. “The Biotheryx team has significant expertise in targeted protein degradation, one of the most promising modalities in oncology, and we look forward to collaborating to develop therapies that can help improve patient lives.”

    About Biotheryx, Inc.

    Biotheryx is a biopharmaceutical company discovering and developing a portfolio of first-in-class protein degraders, including bifunctional degraders and molecular glues. Our initial focus is on deploying the differentiated potential of degraders towards validated targets in cancer and inflammatory disease. Members of our founding and scientific teams previously developed the first U.S. Food and Drug Administration (FDA) approved modulators of Cereblon, the most widely validated E3 ligase involved in protein degradation, and we have applied our expertise in Cereblon binding to build our proprietary PRODEGY platform. Our pipeline of bifunctional degraders includes the first-ever degraders of SOS1 for pan-KRAS mutant cancers, CDK4/6 for solid tumors and PDE4 for inflammatory diseases. Our pipeline of molecular glues includes BTX-1188, a rationally designed, dual-targeting molecular glue degrader of GSPT1 and IKZF1/3 for acute myeloid leukemia and solid tumors. For more information, please visit www.biotheryx.com and engage with us on LinkedIn.

    SOURCE Biotheryx, Inc.

    Deka Biosciences Announces First-in-Human Dose in Phase 1 Clinical Trial of DK210 (EGFR)

    April 6, 2023 / Portfolio News

    Deka Biosciences (“Deka”), a clinical-stage biotechnology company focused on developing novel cytokine therapies to treat cancer and inflammatory diseases, announced today that the first subject has been dosed in a Phase 1 clinical trial of DK210 (EGFR) at NEXT Oncology in Fairfax, Virginia. The Phase 1, first-in-human, multicenter clinical study seeks to characterize the safety, potential efficacy and evaluate possible biomarkers of response to DK210 (EGFR) in patients with advanced solid cancer(s) who are expressing epidermal growth factor receptors (EGFR) (NCT05704985).

    “We are beyond thrilled to begin the clinical trial of DK210 (EGFR), marking our first program to enter clinical development,” said John Mumm, Ph.D., President and CEO of Deka. “Importantly, we anticipate that the results of the study will confirm the clinical safety, pharmacokinetics, exploratory efficacy and correlative biomarker responses to our first DiakineTM, establishing a solid foundation for the expanded use of this treatment in cancer patients.”

    “This novel agent holds significant potential for patients and we are excited to be at the forefront of this groundbreaking platform that places great emphasis on patient comfort and convenience via a self-administered treatment,” shared Dr. Spira, CEO and Clinical Director at NEXT Oncology in Virginia.

    Groundbreaking platform that places great emphasis on patient comfort and convenience via a self-administered treatment

    DK210 (EGFR) is the first of several experimental therapeutics developed as part of Deka’s platform of molecules, which combines the cytokines full strength IL-2 and a high affinity IL-10. While IL-2 is known to be toxic, when coupled with IL-10, toxicity is not only reduced but its potency is increased, thereby creating a more tolerable and effective treatment for patients. It is the first of several experimental therapeutics in Deka’s platform of molecules which are being developed to treat both cancer and inflammatory diseases. These therapeutics, known as DiakinesTM, involve coupling two cytokines together onto a single chain variable fragment (scFv) targeting system to enhance their precision in targeting specific tissues. Furthermore, the scFv scaffold used in the DiakineTM platform also improves efficacy, safety and manufacturability of each treatment.

    About Deka Biosciences

    Deka Biosciences is a biotech company led by entrepreneur Dr. John Mumm, who is backed by a team of experienced academic, biopharma and CDMO innovators with expertise in drug discovery, product development, characterization, testing and clinical development. Deka has developed disease specific Diakines™ designed to maximize patient benefits through improved pharmacokinetics / pharmacodynamics (PK/PD) function by the targeted accumulation of dual and complimentary cytokines into affected tissues. Through developing a better understanding of each patient’s immune response to different Diakines™, Deka hopes to maximize the impact of its Diakines™ by building specific targeted therapies for everyone.

    SOURCE Deka Biosciences

    OpSens Announces 1,000th SavvyWire Procedure

    April 5, 2023 / Portfolio News

    OpSens Inc. (“OpSens” or the “Company”) (TSX: OPS) (OTCQX: OPSSF), a medical device cardiology-focused company delivering innovative solutions based on its proprietary optical technology, announced a significant milestone for the Company as the SavvyWire has been successfully used by physicians in 1,000 procedures. The SavvyWire is OpSens’ sensor-guided solution for transcatheter aortic valve replacement procedures, TAVR or TAVI, to treat aortic valve stenosis.

    “The successful use of the SavvyWire in 1,000 patients marks an important milestone towards full-scale commercialization of our innovative device. The early commercialization phase is progressing steadily and the impressive number of procedures completed demonstrates the acceptance and relevance of the SavvyWire within the structural heart market” said Louis Laflamme, President and Chief Executive Officer of OpSens.

    “We are gaining positive traction in the U.S. and Canada with our proprietary 3-in-1 solution for TAVR  procedures. Our sales team is operating at a high level as they educate cardiologists on the benefits of the SavvyWire to expand the number of accounts and drive adoption. Additionally, signing a GPO agreement last month with one of the largest group purchasing organizations in the United States and SavvyWire being featured in two live case broadcasts during the CRT conference is expected to drive continuous growth going forward,” Mr. Laflamme concluded.

    The SavvyWire is the first and only Sensor-Guided solution for TAVR procedures to treat aortic valve stenosis. It uniquely provides a 3-in-1 solution for stable aortic valve delivery and positioning, continuous accurate hemodynamic measurement during the procedure, and reliable left ventricular pacing without the need for adjunct devices or venous access.

    About OpSens Inc. (www.OpSens.com or www.OpSensmedical.com)

    OpSens focuses mainly on cardiology. The Company offers an advanced optical-based pressure guidewire that aims at improving the clinical outcome of patients with coronary artery disease. Its flagship product, the OptoWire, is a second-generation fiber optic pressure guidewire designed to provide the lowest drift in the industry and excellent lesions access. The OptoWire has been used in the diagnosis and treatment of more than 200,000 patients in more than 30 countries. It is approved for sale in the United States, the European Union, the United Kingdom, Japan and Canada.

    OpSens has recently received FDA clearance and Health Canada approval to commercialize the SavvyWire for transcatheter aortic valve replacement procedures (TAVR). This unique guidewire is a 3-in-1 solution for stable aortic valve delivery and positioning, continuous accurate hemodynamic measurement during the procedure, and reliable left ventricular pacing without the need for adjunct devices or venous access.

    OpSens’ SavvyWire is on trend with a minimalist approach to TAVR and advances the procedure, allowing patients to leave the hospital earlier, sometimes the same day.

    The TAVR procedure is growing rapidly globally, driven by the aging population and recent studies that demonstrate its benefits for a broader array of patients. The global TAVR market is currently estimated at over 200,000 procedures and is expected to reach 400,000 in 2027.

    OpSens is also involved in industrial activities in developing, manufacturing, and installing innovative fiber optic sensing solutions for critical applications.

    SOURCE OpSens Inc.

    For further information: Louis Laflamme, CPA, President and Chief Executive Officer, 418.781.0333; John Hannigan, FCA, Chief Financial Officer, 418.781.0333

    HistoSonics Announces First Ever Kidney Tumor Treatment Using Histotripsy

    April 5, 2023 / Portfolio News

    HistoSonics, (www.histosonics.com), the developer of a non-invasive platform and novel sonic beam therapy called histotripsy, announced today the first kidney patient has been treated using the Company’s histotripsy platform. The procedure was performed in Leeds, UK, by Professor Tze Min Wah, Senior Consultant Radiologist and Clinical Lead for Interventional Oncology Program at Leeds Teaching Hospitals NHS Trust, UK. This procedure marks the initial treatment in the HistoSonics sponsored “CAIN” Trial which is a Phase I prospective, multi-center study designed to evaluate the safety and technical success of the Company’s histotripsy system in targeting and destroying primary solid renal tumors, completely non-invasively and without the need for incisions or needles. The trial is named in honor of Charles Cain PhD, former Chair of Biomedical Engineering at the University of Michigan, co-inventor of histotripsy, and co-founder of HistoSonics, who passed away in March of 2020.

    “This first treatment is a significant milestone for the Company as it represents expansion into our second active clinical application (after liver) and supports our mission to deliver histotripsy to patients who may potentially benefit from its unique capabilities,” commented Mike Blue, President, and CEO of HistoSonics. Blue added, “Our goal is to enable physicians to precisely target and destroy kidney tumors with our novel, noninvasive solution, avoiding the morbidity and complications seen with current invasive surgery or ablative techniques.”

    Current kidney therapies such as partial nephrectomy and thermal ablation are invasive and exhibit complications from bleeding and infection that non-invasive histotripsy may avoid. While surgical intervention is the “gold standard” in removing kidney tumors, a non-invasive approach with histotripsy provides the potential to destroy targeted tissue without damaging non-targeted kidney tissue. Additionally, histotripsy’s purely mechanical mechanism of cellular destruction could preserve function of the kidney’s urine collecting system and eliminate certain complications seen with existing invasive approaches.

    Example of HistoSonics technology targeting kidney tissue to be destroyed in a non-invasive histotripsy procedure.

    Professor Tze Min Wah commented, “I was delighted to lead the clinical team in carrying out this world’s first kidney tumor treatment using histotripsy and a real privilege to have the trust of the patient and their family in translating this innovative technology into our clinic. The CAIN Trial represents a significant milestone for treatment of solid renal tumors with histotripsy as a needle-less technology and is a paradigm shift from this point onwards.”

    HistoSonics’ image guided sonic beam therapy system uses advanced imaging and proprietary sensing technology to deliver non-invasive, personalized treatments with precision and control. The science of histotripsy uses focused sound energy to produce controlled acoustic cavitation that mechanically destroys and liquifies targeted tissue at sub-cellular levels. The company believes that the novel mechanism of action of their proprietary technology provides significant advantages to patients, including the ability of the treatment site to recover and resorb quickly. Uniquely, the HistoSonics’ platform also provides physicians the ability to monitor the destruction of tissue under continuous real-time visualization and control, unlike any modality that exists today.

    HistoSonics histotripsy systems are not currently available for sale. Any use is limited to clinical investigations. The HistoSonics Edison™ System is pending FDA review for a destruction of liver tissue indication. The CAIN Trial is expected to support a future expansion of the indication to include the destruction of kidney tissue.

    About HistoSonics

    HistoSonics is a privately held medical device company developing a non-invasive platform and proprietary sonic beam therapy utilizing the science of histotripsy, a novel mechanism of action that uses focused ultrasound to mechanically destroy and liquify unwanted tissue and tumors. The company is currently focused on the continued development of its Edison™ Platform, global clinical studies, and new strategic projects including future clinical applications and platforms. HistoSonics has offices in Ann Arbor, Michigan and Minneapolis, MN.

    For more information please visit: www.histosonics.com/

    SOURCE HistoSonics, Inc.

    Endotronix Announces Enrollment Completion of PROACTIVE-HF Pivotal Trial

    April 5, 2023 / Portfolio News

    Endotronix, Inc., a digital health and medical technology company dedicated to advancing the treatment of heart failure (HF), today announced its PROACTIVE-HF pivotal study successfully completed enrollment. Designed to evaluate the safety and efficacy of the Cordella Pulmonary Artery (PA) Sensor, the data will support the pre-market approval (PMA) application for market access in the U.S.

    “Building upon a strong foundation of evidence in favor of PA pressure-guided therapy, the PROACTIVE-HF trial is investigating a more comprehensive clinical picture for both clinicians and patients,” commented Liviu Klein, MD, Section Chief of Advanced Heart Failure, Mechanical Circulatory Support, Pulmonary Hypertension, and Heart Transplant at the University of California San Francisco and national principal investigator of the PROACTIVE-HF trial. “Cordella provides daily PA pressure and vital sign data to help clarify patient’s clinical status. Additionally, we are seeing high levels of engagement and lifestyle changes as a result of patients having visibility to their own data. We look forward to sharing the study results in the coming months.”

    Notably the first global investigational device exemption (IDE) study for PA pressure-guided HF management, PROACTIVE-HF enrolled over 450 patients across the U.S., Ireland, and Belgium. Primary endpoints for the single-arm, multi-center trial include mortality and HF hospitalizations as well as device safety. The company expects to report primary endpoint data from the trial in the first half of 2024.

    “I would like to congratulate all our clinical partners on their tremendous effort to complete enrollment for PROACTIVE-HF. Their hard work and dedication are key to improving outcomes and expanding patient access, and we appreciate their continued commitment to this trial,” stated Harry Rowland, CEO and co-founder of Endotronix.

    Endotronix completed enrollment in the PROACTIVE-HF clinical trial and successfully implanted over 450 Cordella PA Sensors in preparation for their regulatory submission.

    About Endotronix

    Endotronix innovates at the intersection of Medtech & Digital Health to improve care for people living with heart failure (HF).  The comprehensive Cordella solution enables proactive, data-driven HF management that engages patients, reduces and prevents congestion, and improves outcomes. The Cordella PA Sensor is an implantable pulmonary artery (PA) pressure sensor which directly measures the leading indicator of congestion, allowing early, targeted therapy. The Cordella HF System is a patient health management platform, which combines comprehensive vital sign data from non-invasive devices to support patient-clinician engagement and care decisions. Combining trended insights, the versatile and scalable Cordella enhances current clinical practice and supports guideline-based care across the entire HF continuum. Learn more at www.endotronix.com.

    The Cordella™ PA Pressure Sensor System is an investigational device and is not currently approved for clinical use in any geography.

    CAUTION – Investigational Device. Limited by Federal (or United States) Law to Investigational Use. Exclusively for Clinical Investigation.

    The Cordella HF System, without the sensor, is available for commercial use in the U.S. and E.U.

    Cautionary Statement Regarding Forward-Looking Statements

    This press release may contain predictions, estimates, or other information that might be considered forward-looking statements. Such forward-looking statements are not a guarantee of future performance.

    Congruence Therapeutics Announces Close of Series A Extension, Bringing Total Round to Over US$65 Million

    March 6, 2023 / Portfolio News

    Congruence Therapeutics, a biotechnology company working at the interface of computational and experimental drug discovery to design novel small molecules for diseases of protein misfolding, announced today the close of an extension to its Series A financing, bringing the total amount raised to over US$65 million. This extension was led by new investor BDC Capital’s Thrive Venture Fund, with participation from current investors Amplitude Ventures, Fonds de solidarité FTQ, OrbiMed, Investissement Quebec (IQ), SilverArc, and others.

    “This financing provides further evidence of the emerging role that technology must play to drive innovation in biotechnology. We are proud to be pioneers in tech-enabled drug discovery with the support of high-quality investors who share our commitment to developing computational tools that will re-invent the way that drugs are discovered,” said Dr. Clarissa Desjardins, CEO of Congruence. “These additional proceeds will allow us to accelerate our pace, advancing our Revenir™ platform and our pipeline of first-in-class and best-in-class programs for devastating genetic, rare and neurological diseases toward the clinic.”

    Michelle Scarborough, Managing Partner, Thrive Venture Fund at BDC Capital added, “With an incredible team led by Clarissa and a first-of-its-kind discovery platform, Congruence will quickly emerge as a leading biotechnology company who is well-positioned to disrupt traditional drug discovery and deliver real benefits to patients. We are proud to lead this investment round and look forward to supporting Congruence through its next phase of growth.”

    In February 2022, Congruence announced the close of its Series A financing of US$50 million, led by Amplitude Ventures and Fonds de solidarité FTQ, with participation from Lumira Ventures, Investissement Quebec, OrbiMed Advisors, Driehaus Capital Management, and others. Congruence Therapeutics was founded in 2021 by Clarissa Desjardins, Ph.D., previously the founder and chief executive officer of Clementia Pharmaceuticals, a company developing therapies for rare pediatric bone diseases and sold to Ipsen in early 2019 for US$1.3 billion. Clementia’s lead product, Sohonos (palovarotene), was recently approved in Canada and is under review at the FDA as the first treatment for Fibrodysplasia Ossificans Progressiva, a debilitating and progressive rare bone disorder. At Congruence, Dr. Desjardins has put into place a team of ‘drug hunters’ with experience in rare disease strategy and clinical development, computational chemistry, medicinal chemistry, and business development.

    About Congruence Therapeutics

    Congruence Therapeutics is a drug discovery company that is uniquely harnessing the power of innovative fields such as protein dynamics, biophysics, machine learning, AI, and computational chemistry to advance drug discovery. Our ground-breaking discovery engine, RevenirTM, captures the biophysical features of functional proteins and their pathogenic counterparts in order to discover functional allosteric and cryptic pockets which can lead to small molecule hits at unprecedented speed. 

    For more information, please visit www.congruencetx.com.

    Company Contact
    Tanya Borsuk
    EVP of Corporate and Business Development
    tborsuk@congruencetx.com

    Media Contact
    Amy Conrad
    Juniper Point
    amy@juniper-point.com 
    858-366-3243

    Rare Disease Day 2023

    February 28, 2023 / Lumira News

    Today, February 28th, 2023, is Rare Disease Day. Celebrated internationally, the day is an opportunity to raise awareness and advocate for rare diseases as a human rights priority and work towards a more inclusive society. Over 300 million people globally are living with rare diseases: reflecting 3.5-5.9% of the total population. Additionally, over 6000 rare diseases are characterized by a broad diversity of disorders and symptoms that vary not only from disease to disease but also from patients suffering from the same disease. Established in 2008, the campaign has a long-term goal of achieving equitable access to diagnosis, treatment, health and social care, and opportunity for people affected by rare diseases.

    At Lumira Ventures, we are proud to support the discovery, development, and commercialization of innovative medical products with the potential to provide greater health outcomes for patients suffering from rare diseases. In partnership with Angelini Pharma, the Angelini Lumira Biosciences Fund (ALBF) was established to invest in and help build best- and first-in-class treatments that resolve the many unmet medical needs of those with rare diseases. Since the inception of The Fund, we have invested in two rare disease-focused companies (GeneEp and Congruence Therapeutics) that are paving the way in their unique areas to create impactful therapeutic solutions for patient lives. We are excited about the progress being made by our portfolio companies and look forward to continuing our support for the very best impact-driven companies, focused on bringing meaningful changes to the lives of patients with rare diseases.

    Reza’s Story: Thirteen-year-old Reza lives in Iran with his family, and he is also living with a rare disease called Cystinosis.
    www.rarediseaseday.org

    Spotlight Story: Congruence Therapeutics, Accelerating the Understanding of and Course of Action for Treating Rare Diseases.

    Congruence Therapeutics is a pre-clinical drug discovery company on a mission to advance the knowledge of rare diseases and discover treatments that help to improve the quality of life for those affected. Harnessing the power of protein dynamics, biophysics, machine learning, and computational chemistry, the company aims to discover and design novel small molecules for the real-time treatment of rare diseases of protein misfolding. A condition where there is currently no treatment.

    The company’s lead program, Revenir™, is a purpose-built computational drug discovery engine that captures the biophysical changes caused by mutations in proteins. The platform examines the surface features and numerous biophysical descriptors of both the mutated and wild-type proteins. Through building this unique understanding of the defect, the platform then applies real-time ligand-based corrections to predict hits for small molecule treatment with the power to drive a resolution of the disease state. Following the identification of a promising small molecule compound, effective treatments can be performed to potentially resolve the disease.

    Along with an important and powerful mission behind Congruence’s technology, the company is led and founded by a notably successful entrepreneur, dedicated to creating this future of more significant treatment options and outcomes. Dr. Clarissa Desjardins is determined to use her experience and success to create real rare disease-focused solutions to improve patient lives. “When researching rare diseases, the outcome is very important for those affected and their families,” she explains. “In collaboration with the medical community, we are developing new treatments and not just another version of an existing treatment; it’s going to make a real difference.”

    Congruence’s pipeline consists of both best- and first-in-class programs that demonstrate the potential to address significant unmet medical needs in numerous high-value indications across rare disease categories. The company’s lead indication is genetic metabolic diseases, with additional developments in rare genetic and neurological diseases, cancer, and solid as well as hematologic tumors.

    As Congruence continues to make promising progress with its program development, the company is well-positioned to create a life-changing platform that significantly contributes to the knowledge and development of treatments for various rare diseases of protein folding.

    2023 Lumira Venture Innovation Program (VIP): Fellow and Summer Analyst Applications Open

    February 8, 2023 / Lumira News

    Looking to make a career in life sciences innovation? Our Venture Innovation Program (VIP) is accepting applications for exceptional students across Canada who want to gain exposure to life sciences venture capital and start-ups, and are committed to pushing the future of life sciences innovation forward. By working hand in hand with seasoned industry professionals from all dimensions of the life sciences ecosystem, you will gain first-hand knowledge and insights into the challenges, opportunities, processes, and people shaping the future of healthcare innovation in Canada and around the globe.

    We are currently accepting applications for two different roles:

    Venture Fellow

    The Lumira VIP Fellow Position is a 6-12 month program for Ph.D., MD, and MBA students who are motivated build careers in life sciences innovation. The program is designed to be completed part-time alongside your academic program and to offer you the opportunity to participate in the full lifecycle of life sciences venture innovation and investment via different engagements across science, medicine, business, strategy, and operations. The program can be completed remotely from anywhere in Canada, with periodic opportunities to interact in-person at one of our Canadian offices. 

    Application Deadline: March 31st, 2023

    Summer Analyst

    The Summer Analyst will join Lumira’s Toronto office on-site from May-August. In this position you will work closely with Lumira’s Toronto based investment team to learn all aspects of the venture business, with a particular focus on financial modeling, business diligence and proactive market analysis. This position is open to exceptional business-stream upper year undergraduates, MBAs, and Masters students with a strong financial analysis skill set. Education or work experience in the life sciences would also be an asset.

    Application Deadline: Sunday, March 5th, 2023

    Frequently Asked Questions

    Who is Lumira Ventures?

    Lumira Ventures is an impact investor that has consistently delivered first quartile financial returns to investors in its funds, while also delivering medical innovations that impact patient lives and healthcare ecosystems globally. Our portfolio companies have brought more than 50 biomedical innovations to the market impacting the lives of over 1 billion patients, and generating over $70 billion of cumulative revenue.

    Why did Lumira Ventures launch the LV VIP?

    From the seed investment all the way through to growth stage investment, we have partnered with entrepreneurs in Canada and throughout North America to develop and commercialize truly transformative products that address unmet patient needs and improve the lives of patients worldwide. As Canada is at the foundation of Lumira’s business, we feel privileged to be an active builder of Canada’s biotech and life sciences ecosystem. As a function, we recognize the growing need to identify and support the development of next generation of leaders and innovators within Canada’s high-growth life sciences ecosystem.

    Am I eligible to apply for LV VIP?

    Venture Fellow:

    • Canadian citizen or Permanent Resident of Canada
    • Student in the final years of a PhD, MBA or MD program or recent graduates engaged in post-doctoral research or residency programs, with a strong background in the life sciences.

    Summer Analyst:

    • Canadian citizen or Permanent Resident of Canada
    • Business-stream upper year undergraduate, MBA, and/or masters students with a strong financial analysis skill set, and track record of academic excellence.
    • Background in the life sciences would be an asset.

    How much of a time commitment is ideal and is the program paid?

    Venture Fellow: To get the true benefit of the program, the candidates are expected to participate for at least 6 months, and commit 10-15h per week. Participants must be available to join some meetings during business hours, the timing of which can be flexible. Participants will also be entitled to a monthly stipend.

    Summer Analyst: To ensure the greatest experience and engagement for our Summer Analyst, candidates are expected to be working with our team full-time and in-person at our Toronto office. The position is a paid experience.

    Where does the program take place?

    Venture Fellow: Candidates can work remotely from anywhere in Canada, and will have opportunities for in-person interactions with the Lumira team if logistics allow.

    Summer Analysts: Candidates will work full-time and in-person from the Lumira Ventures Toronto office.

    VIP Alumni Experiences

    XyloCor Therapeutics Reports Positive Topline Safety and Efficacy Results from Phase 2 EXACT Clinical Trial of XC001 Novel Gene Therapy for Refractory Angina

    January 26, 2023 / Portfolio News

    No serious adverse events related to drug product were reported
    Patients demonstrated improvements in exercise capacity and reductions in episodes of chest pain
    Cardiac imaging results provide mechanistic evidence supporting the therapeutic potential of XC001 in cardiovascular disease

    XyloCor Therapeutics, a clinical-stage biopharmaceutical company developing novel gene therapies for cardiovascular disease, today announced completion of the Phase 2 portion of its Phase 1/2 clinical trial (EXACT) designed to assess the safety and preliminary evidence of efficacy of lead gene therapy candidate XC001 (encoberminogene rezmadenovec) in patients with refractory angina.  EXACT met both safety and efficacy objectives. There were no safety issues related to drug product or unexpected serious adverse events related to XC001 administration.  Six-month data from 28 patients in the Phase 2 portion of the study showed improvements in several key efficacy measures, including reduction in ischemic burden.

    “We are excited to see EXACT completing its 6-month endpoint.  The trial met all of its safety and exploratory objectives, showing intriguing benefits in these needy patients across a variety of objective and subjective measures,” said Thomas Povsic, M.D., Ph.D., Professor of Medicine, Duke University School of Medicine and National Principal Investigator for the EXACT study. “The strong range of mechanistic evidence demonstrate that administration of XC001 is a scientifically-sound approach for achieving a biological effect that has the potential to improve patients’ quality of life.”

    XC001 is a one-time gene therapy designed to reduce ischemic burden by creating new blood vessels in  the heart. In the Phase 2 portion of the EXACT trial, evidence of the drug’s mechanism of action was demonstrated by the reduction of ischemic burden measured by cardiac positron emission tomography (PET) imaging.  The reduction in ischemic burden was accompanied by an improvement in total exercise duration, an important measure of exercise capacity. Prior to treatment, almost all subjects had marked limitations on ordinary physical activity. Six months after treatment, nearly half of all subjects were able to conduct ordinary physical activity without causing angina. The data from the Phase 2 EXACT study are potentially meaningful for patients with refractory angina, which includes more than one million people in the United States, who have no treatment options.

    “We are excited to share this positive topline data from the Phase 2 portion of the EXACT trial, reinforcing our confidence in XC001 as a novel therapeutic approach with the potential to address the significant unmet medical needs of people with refractory angina,” said Al Gianchetti, President and CEO of XyloCor. “We now look forward to pursuing key upcoming milestones in XC001’s continued development, including finalizing our pivotal trial design through our ongoing discussions with the FDA and other regulatory authorities.”

    About XC001

    XC001 is designed to promote new blood vessels in the heart that will bypass diseased blood vessels and improve blood flow. By restoring blood flow, chest pain associated with refractory angina may decrease, potentially improving patients’ quality of life by enabling them to engage in daily physical activities that would otherwise cause pain. XC001 is designed to avoid toxicity issues observed with other gene therapies through a strategy of one-time, local administration. This approach allows XC001 to achieve higher gene expression in the heart while minimizing systemic vector circulation and associated side effects.

    About the EXACT Study

    The recently completed Epicardial Delivery of XC001 Gene Therapy for Refractory Angina Coronary Treatment (EXACT) clinical trial was a Phase 1/2 multicenter, open-label, single-arm trial. Twelve subjects (n=3 per dose cohort) who have refractory angina were enrolled into four ascending dose groups, followed by an expansion phase of the trial in which additional subjects were enrolled at the highest tolerated dose (1 x 1011 vp, the highest tested dose). The investigational gene therapy is administered directly to the heart muscle through a mini-thoracotomy by a cardiac surgeon.

    About Chronic Refractory Angina

    In the United States, coronary artery disease is a leading cause of death and disability. Chronic angina pectoris occurs when the heart muscle does not receive sufficient oxygen resulting in chest pain. This is usually due to atherosclerotic plaques that block the coronary arteries. Refractory angina is a growing problem that occurs in patients with chronic angina who are symptomatic despite optimal medical therapy and are no longer eligible for mechanical interventions like percutaneous coronary intervention (PCI) and coronary artery bypass grafting (CABG). These patients currently have no treatment options and are frequently highly symptomatic, which severely impacts their quality of life, and may exacerbate comorbidities and cause further deterioration of their health status. Refractory angina results in significant consumption of healthcare resources, including visits to the emergency department as a result of patients’ chest pain.

    About XyloCor

    XyloCor Therapeutics is a private, clinical-stage biopharmaceutical company developing potential best-in-class gene therapies to transform outcomes for patients with cardiovascular disease. The Company’s lead product candidate, XC001, is in clinical development to investigate use for patients with refractory angina for whom there are no treatment options. XyloCor has a second preclinical investigational product, XC002, in discovery stage, being developed for the treatment of patients with cardiac tissue damage from heart attacks. The company, which was co-founded by Ronald Crystal, M.D., and Todd Rosengart, M.D., has an exclusive license from Cornell University. For more information, visit www.xylocor.com.

    Corporate and Investor Relations:

    Brian Davis, XyloCor Therapeutics

    brian.davis@xylocor.com

    610-541-2056

    Media Contact:

    Mike Beyer, Sam Brown Inc. Healthcare Communications

    mikebeyer@sambrown.com

    312-961-2502

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