Edwards Lifesciences (NYSE: EW) today announced investments that reflect the company’s deep commitment to advancing patient care through structural heart innovation, addressing large unmet patient needs and supporting sustainable long-term growth.

Edwards has entered into an agreement to acquire JenaValve Technology, a pioneer in the transcatheter treatment of aortic regurgitation (AR), a deadly disease that impacts a significant and growing population and is largely untreated today. JenaValve presented positive results of its U.S. pivotal trial for the treatment of symptomatic, severe AR in high-risk patients late last year. As the pioneer in valve innovation for more than 60 years, Edwards believes it is uniquely positioned to lead this next frontier of aortic valve disease treatment. Edwards anticipates FDA approval of the JenaValve Trilogy Heart Valve System in late 2025, which will represent the first approved therapy for patients suffering from AR.

Building on an investment made in 2016, Edwards has exercised its option to acquire Endotronix, a leader in heart failure (HF) management solutions. Many structural heart patients Edwards serves today also suffer from HF with limited options. This acquisition will expand Edwards’ structural heart portfolio into a new therapeutic area to address the large unmet needs of patients suffering from HF. Last month, Endotronix received FDA approval for Cordella, an implantable pulmonary artery pressure sensor allowing early, targeted therapeutic intervention. A CMS national coverage determination is expected in early 2025.

“These acquisitions expand our opportunities to address the unmet needs of aortic regurgitation and heart failure patients around the world,” said Bernard Zovighian, Edwards’ CEO. “We are pleased to enter these structural heart therapeutic areas with innovation, world-class science and clinical evidence to provide access to life-saving technologies for patients around the world.”

Edwards anticipates these investments will strengthen its leadership in structural heart innovation and represent long-term growth opportunities. Edwards expects minimal revenue contribution from these acquisitions in 2025. The aggregate upfront purchase price for these strategic investments is approximately $1.2 billion. The acquisitions are subject to the satisfaction of certain closing conditions, including the receipt of required antitrust and foreign investment approvals.

About Edwards Lifesciences

Edwards Lifesciences is the global leader of patient-focused innovations for structural heart disease and critical care monitoring. We are driven by a passion for patients, dedicated to improving and enhancing lives through partnerships with clinicians and stakeholders across the global healthcare landscape. For more information, visit www.edwards.com and follow us on Facebook, Instagram, LinkedIn, X and YouTube.

This news release includes 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. We intend the forward-looking statements contained in this press release to be covered by the safe harbor provisions of such Acts. These forward-looking statements can sometimes be identified by the use of forward-looking words, such as “may,” “might,” “believe,” “will,” “expect,” “project,” “estimate,” “should,” “anticipate,” “plan,” “goal,” “continue,” “seek,” “intend,” “optimistic,” “aspire,” “confident” and other forms of these words and include, but are not limited to, statements made by Mr. Zovighian and statements regarding our expected continued performance of Edwards; performance of the Edwards, JenaValve or Endotronix technologies; product and therapy benefits; patient access and outcomes; size of treatable population; leading position; growth opportunities; unmet needs in structural heart, aortic regurgitation, and heart failure therapeutic areas; probability of approval by the FDA and in the anticipating timeline; probability of a positive NCD by the CMS and in the anticipated timeline; synergies between the technologies, business, and operations of each of JenaValve and Endotronix and Edwards’ technologies, products, portfolio, expertise, and operations; ability to leverage the technology or innovation from these acquisitions or cause or ensure accelerated access to life-saving technologies for patients or development of novel technologies as a result of these acquisitions; commitment to expand opportunities in structural heart innovation, address large unmet patient needs, and support sustainable long-term growth; objective to expand Edwards’ portfolio into new structural heart therapeutic areas; opportunities and revenue return on these acquisitions and their contribution to Edwards’ growth and performance, as well as the expectations on timing of such returns and contributions; therapy approval pipeline for patients suffering from AR; probability of the closing of the two acquisitions; other objectives and expectations; and other statements that are not historical facts. Forward-looking statements are based on estimates and assumptions made by management of the company and are believed to be reasonable, though they are inherently uncertain and difficult to predict. Our forward-looking statements speak only as of the date on which they are made, and we do not undertake any obligation to update any forward-looking statement to reflect events or circumstances after the date of the statement. Investors are cautioned not to unduly rely on such forward-looking statements.

Forward-looking statements involve risks and uncertainties that could cause actual results or experience to differ materially from that expressed or implied by the forward-looking statements. Factors that could cause actual results or experience to differ materially from that expressed or implied by the forward-looking statements include, but are not limited to: (i) Edwards may be unable to close the acquisitions of each of JenaValve and Endotronix, which may materially and adversely affect Edwards’ business and the price of Edwards’ common stock; (ii) the occurrence of any event, change or other circumstance that could cause Edwards to abandon the acquisitions of either or both of JenaValve and Endotronix; (iii) risks related to disruption of management’s attention from Edwards’ ongoing business operations; (iv) the effect of the announcement or the pendency of the acquisitions on Edwards’ relationships with its customers, operating results and business generally; (v) potential significant transaction costs associated with either or both acquisitions; (vi) the outcome of any legal proceedings or regulatory actions to the extent initiated against Edwards or others related to either or both acquisitions; (vii) the ability of Edwards to execute on its strategy and achieve its goals and other expectations after the closing of either or both acquisitions; (viii) legal, regulatory, tax and economic developments affecting Edwards’ business; (ix) the unpredictability and severity of catastrophic events, including, but not limited to, acts of terrorism, outbreak of war or hostilities or current or future pandemics or epidemics, as well as Edwards’ response to any of the aforementioned factors; and (x) other risks detailed in Edwards’ filings with the SEC, which may be found at edwards.com.

Edwards, Edwards Lifesciences, and the stylized E logo are trademarks of Edwards Lifesciences Corporation or its affiliates. All other trademarks are the property of their respective owners.

Contacts

Media Contact: Amy Hytowitz, 949-250-4009
Investor Contact: Mark Wilterding, 949-250-6826

enGene Holdings Inc. (Nasdaq: ENGN, “enGene” or the “Company”), a clinical-stage genetic medicines company whose non-viral, intravesical lead product candidate, EG-70, is in a pivotal study for BCG-unresponsive high-risk Non-Muscle Invasive Bladder Cancer (NMIBC), today announced that Ron Cooper has joined the Company as Chief Executive Officer and member of the Board of Directors, effective July 22, 2024. This transition follows a previously announced succession plan for Jason Hanson, who will remain in service to the Company as a strategic advisor.

“The Board of Directors and I would like to warmly welcome Ron Cooper, who brings significant discovery, development, and commercial launch expertise,” said Dr. Richard Glickman, Chairman of the Board of Directors. “Given his proven record of effectively developing and commercializing transformative therapeutics, Mr. Cooper is an excellent fit to lead enGene forward as the Company continues to advance EG-70 as an innovative and differentiated therapeutic option for patients with high-risk NMIBC.”

“The unmet need for patients with bladder cancer is significant,” said Mr. Cooper. “EG-70 has the potential to be the most practical treatment option for high-risk NMIBC based on a unique combination of efficacy, tolerability and ease of administration. Under Mr. Hanson’s leadership, the enGene team has developed an innovative platform to deliver genetic medicines through mucosal tissues, which I believe has underappreciated potential.”

Mr. Cooper most recently served as President and Chief Executive Officer of Albireo Pharma, a fully integrated global commercial biopharmaceutical company that Ipsen acquired in 2023. While at Albireo Pharma, Mr. Cooper took the company public, created a new corporate strategy to focus development on building a rare pediatric company while monetizing other pipeline assets, and guided Bylvay® through three Phase 3 programs, regulatory approvals, and a global commercial launch. Earlier in his career, Mr. Cooper spent nearly 30 years at Bristol-Myers Squibb (BMS) in roles of increasing responsibility in sales, marketing and general management, most recently serving as President, Europe. While at BMS, he played a leadership role in numerous successful product launches. Mr. Cooper is currently Chairman of the Board of Directors at C4 Therapeutics and serves on the Board of Generation Bio. He is a graduate of St. Francis Xavier University.

The Company also announced the promotion of Dr. Raj Pruthi to Chief Medical Officer, succeeding Dr. Richard Bryce. Dr. Pruthi, who joined enGene in April 2024, is a thought leader in the urological community with over 25 years of experience providing patient care and advancing the development of new therapeutics.

“On behalf of the Board of Directors, we would like to sincerely thank Mr. Hanson for his service to enGene,” said Dr. Glickman. “Mr. Hanson successfully led the Company’s evolution from an early-stage research organization to a well-capitalized, publicly traded, pivotal-stage company poised to make an impactful difference for patients living with NMIBC. We wish him all the best in his future endeavors.”

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) – a disease with a high clinical burden. EG-70 is being evaluated in the ongoing multi-cohort LEGEND Phase 2 study, which includes a registrational cohort studying EG-70 in Bacillus Calmette Guérin (BCG)-unresponsive patients with carcinoma in situ (Cis). 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.

Forward-Looking Statements

Certain 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 management teams’ 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 version 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 the potential of EG-70 and enGene’s proprietary DDX platform.

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” sections of the Company’s Annual Report on Form 10-K for the fiscal year ended October 31, 2023, our Quarterly Report on Form 10-Q for the fiscal quarter ended January 31, 2024 and our Quarterly Report on Form 10-Q for the fiscal quarter ended April 30, 2024 (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.

View source version on businesswire.com: https://www.businesswire.com/news/home/20240724237337/en/

For media: media@engene.com

For investors: investors@engene.com

Positive results including complete inhibition of cough response in preclinical models and a favorable safety profile in three clinical studies support the initiation of a Phase 2b clinical trial of taplucainium in 240 subjects in the second half of 2024.

Nocion Therapeutics, Inc., a clinical stage biopharmaceutical company developing novel small molecule charged sodium channel blockers called “nocions”, that selectively affect actively firing nociceptors for the treatment of serious conditions involving cough, itch, and pain, today announced the presentation of progress in the development of taplucainium, a novel, charged sodium channel blocker, as a potential treatment for chronic cough at the 13^th London International Cough Symposium. The presentation and poster will be given by Bernard Silverman, MD., Nocion’s Chief Medical Officer.

“We are excited with the strong progress that we have made developing taplucainium as a treatment for chronic cough with the ability to address this significant unmet medical need,” said Dr. Silverman. “We look forward to the start of our ASPIRE (Phase 2b) clinical trial of taplucainium in 240 chronic cough patients in the second half of 2024.”

ABOUT TAPLUCAINIUM
Taplucainium (formerly NTX-1175) is a proprietary molecule in the novel class of charged sodium channel blockers that allows for specific silencing of activated/inflamed nociceptors while having minimal local off-target effects or systemic exposure. Unlike other investigative cough therapies, such as P2X3-antagonists, TRPA1-antagonists and TRPM8-agonists, which target a specific large pore channel, taplucainium works downstream by selectively targeting the sodium channels in activated nociceptors which are the target of these large pore channels. Taplucainium is formulated into a dry powder for inhalation and, once inhaled, gains access to the pulmonary nociceptors through any open large pore channel including P2X, TRPV, TRPA and TRPM channels whereupon it inhibits the sodium channels responsible for initiating the pathological cough response. The broader mechanism of taplucainium has shown significant antitussive effects in preclinical models of cough. Combined with good preliminary safety and efficacy data from earlier stage clinical work, this forms the basis for its investigation not just in chronic cough but in other cough indications as well.

ABOUT NOCION
Nocion Therapeutics is a biopharmaceutical company developing novel small molecule charged sodium channel blockers called “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. Venture investors in Nocion include Arkin Bio Capital, Canaan Partners, F-Prime Capital, Lumira Ventures, Mass General Brigham Ventures, Mission BioCapital, Monograph Capital, Morningside and Osage University Partners. For more information, visit: www.nociontx.com.

Contacts

Stephanie Gillis
pr@nociontx.com

The Extroducer® Infusion Catheter System ® enables local delivery of XC001 to the heart without the need for surgery.

XC001 has achieved positive Phase 1/2 results in the EXACT Trial validating its transformative potential for treatment of refractory angina in patients who have exhausted available treatment options and have a debilitating quality-of-life.

XyloCor Therapeutics, Inc. (“XyloCor”), a clinical‑stage biopharmaceutical company developing novel gene therapies for cardiovascular disease, and SmartWise, a unit ofSmartCella Holding AB (“SmartCella”), have entered into a licensing agreement under which XyloCor has rights to the Extroducer® Infusion Catheter System ®, a first-in-class endovascular device designed to deliver advanced therapies directly into the heart [and hard-to-reach tissues] . XyloCor plans to deploy the Extroducer to support catheter-based endocardial delivery of its lead gene therapy candidate, XC001 (encoberminogene rezmadenovec), in future clinical studies and commercial use.

“This agreement with SmartCella will enable XyloCor to build upon its robust foundation of efficacy and safety data for XC001 by offering the potential for improved safety and ease of delivery without surgery via this novel catheter,” said Al Gianchetti, President and CEO of XyloCor. “Teaming up with SmartCella will help in our effort to optimize patient safety and tolerability while maintaining accurate delivery of XC001 to target areas in the heart for patients with refractory angina. It also opens up the potential to develop XC001 earlier in the coronary artery disease progression for even larger patient groups.”

XC001 is designed to reduce ischemic burden by creating new blood vessels in the heart through the local expression of multiple isoforms of vascular endothelial growth factor (VEGF). With the use of the Extroducer catheter, XyloCor can offer patients a better delivery option for local administration of XC001 directly to the heart, that is less invasive and eliminates potential risks associated with surgical administration.

“We welcome the Extroducer delivery of XC001 as it offers a more efficient method for gene therapy administration for patients with refractory angina,” said Timothy D. Henry MD, Interventional Cardiologist and Director of the Lindner Center, The Christ Hospital, Cincinnati, Ohio. “Preclinical models provide strong evidence that this approach will maintain, or even improve the efficacy when compared to surgical delivery and it should lower the risk of complications that may arise from surgical administration. I am looking forward to initiating the Phase 2b trial of XC001 in patients with refractory angina using this innovative administration approach.”

The recently published EXACT Phase 1/2 trial assessed the use of one-time gene therapy with XC001 as a new therapeutic approach in refractory angina – a debilitating and chronic condition that impacts over one million people in the United States and is growing in prevalence. In the EXACT trial, 42 patients with class II-IV angina were treated with XC001 directly administered to the heart following minimally invasive surgical access. The results demonstrated that treatment with XC001 can be safely administered and achieve durable clinical improvements of exercise duration, and angina frequency, due to a decrease in ischemic burden, as measured by Positron Emission Tomography (PET) imaging. Notably, six months after treatment 43% of patients had no chest pain with ordinary activities and 58% reported no angina episodes at 12-month clinical follow up. XC001 was well tolerated in the patient population and there were no serious adverse events related to the drug. The Phase 2b trial will be a randomized double-blinded study assessing the safety and efficacy of XC001 administered via the Extroducer® Delivery Catheter in coronary artery disease patients with refractory angina.

“The collaboration underscores the transformative potential of the Extroducer in delivering XC001 therapy for patients with refractory angina. A great example of a powerful combination of delivery system and drug therapy representing a substantial advancement in treatment options. The collaboration with such a distinguished partner as XyloCor marks a significant milestone for our global expansion efforts and will also enable us to further explore and harness the future capabilities of the Extroducer, ultimately expanding the benefits to a greater number of patients in need,“ said Niklas Prager, CEO of SmartCella.

Terms of the agreement include a global license to XyloCor for use of the Extroducer for the administration of XC001 and provide for SmartCella to supply catheters to XyloCor in clinical trials and commercial use in exchange for an upfront payment, clinical, regulatory and commercial milestones and a royalty on sales. Total deal value amounts to approximately USD 130 million and mid-single digit royalties.

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.

AboutExtroducer® Infusion Catheter System

The Extroducer® Infusion Catheter System is a first-in-class endovascular delivery device which enables direct-to-tissue drug delivery. The Extroducer® addresses a significant unmet need in the field of novel therapies, enabling targeted delivery of a wide range of modalities for solid tumor treatment, genetic disorders and tissue repair, to name but a few. Using standard equipment and routine interventional radiology approaches, the Extroducer provides access to hard-to-reach tissues by safely penetrating the vessel wall and delivering payload directly to the target location. SmartWise received U.S. Food and Drug Administration (FDA) clearance under 510(k) for the Extroducer® delivery catheter in June 2022.

About XyloCor

XyloCor Therapeutics, Inc. 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.

About SmartCella

SmartCella, founded in 2014, is an innovative biotechnology company based in Stockholm, Sweden. SmartCella’s vision is to combine first-in-class delivery platforms with cutting-edge cell and mRNA therapies to unleash the full potential of targeted therapies. The company has three main business units, Smartwise, SmartCella Solutions and ProCella. For more information, visit www.smartcella.com.

Contacts

Corporate and Investor Relations:
Brian Davis
XyloCor Therapeutics, Inc
brian.davis@xylocor.com
610-541-2056

Media Contact:
Mike Beyer
Sam Brown Inc. Healthcare Communications
mikebeyer@sambrown.com
312-961-2502

SmartCella Contact:
Niklas Prager, CEO, +46 768 117744; niklas.prager@smartcella.com

Oversubscribed financing led by strategic investor, with participation from new and existing investors

Endogenex, a clinical-stage medical device company dedicated to improving outcomes in individuals with type 2 diabetes, announced it has closed an oversubscribed Series C financing totaling $88 million. The new capital will be used to complete the pivotal ReCET Clinical Study, which has been granted an Investigational Device Exemption (IDE) by the U.S. Food and Drug Administration (FDA). A group of new investors, including Hatteras Venture Partners, Lumira Ventures, and Orlando Health Ventures, joined an undisclosed strategic lead investor and existing investors Intuitive Ventures, Longitude Capital, Mayo Clinic, and Santé Ventures in the Series C funding.

“We are excited to take this significant step forward in our mission to transform diabetes care,” said Stacey Pugh, CEO of Endogenex.  “This funding will enable us to complete our pivotal clinical study, bringing us closer to offering a groundbreaking solution for type 2 diabetes patients.  The therapeutic landscape in diabetes continues to evolve, especially around the earlier use of SGLT2i and GLP-1RA. However, there remains a considerable unmet need to address the underlying pathophysiology and progression of the disease.”

“We are very proud of the company’s progress, and this successful funding round is a testament to Endogenex’s innovative technology,” said Juliet Tammenoms Bakker, Endogenex Board Chair and Managing Director at Longitude Capital. “This milestone underscores our investors’ confidence in the company’s vision and the impact it can make in treating the epidemic of type 2 diabetes around the world.”

About the ReCET Clinical Study

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 received IDE approval in November 2023. The study will enroll up to 350 patients at clinical sites in the United States and Australia.

About the ReCET™ Procedure

ReCET is a novel, endoscopic outpatient procedure that targets the cellular pathology of the duodenum. This pathology may contribute to the development and progression of type 2 diabetes.

The ReCET System aims to initiate the body’s natural regenerative process by applying highly controlled, non-thermal pulsed electric fields to the mucosa and sub-mucosa duodenal tissue. This approach may help restore proper cellular signaling from the duodenum and improve metabolic function, including better control of blood glucose levels.

The ReCET System has been evaluated in feasibility clinical studies, such as REGENT-1 US, REGENT-1 Australia, and EMINENT in the Netherlands. These studies assessed the safety and efficacy of the treatment in adults with type 2 diabetes whose blood glucose levels were inadequately controlled despite using insulin and non-insulin medications. Preliminary outcomes from these studies have been presented at medical conferences globally.

The ReCET System has received FDA Breakthrough Device Designation for treating type 2 diabetes in adults inadequately controlled by glucose-lowering medications.

About Endogenex™

Endogenex, founded in partnership with Mayo Clinic, aims to revolutionize treatment options for individuals with type 2 diabetes (T2D). The company’s innovations focus on resetting the body’s metabolic signaling system by harnessing its natural regenerative capabilities to improve metabolic function. Through the development of the ReCET System and the novel application of precise, controlled, non-thermal pulsed electric fields, Endogenex is establishing a new era in T2D therapy, helping patients regain control of their blood glucose levels and slow disease progression.

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

Cordella, which consists of the now PMA-approved Cordella PA Sensor and commercial Cordella™ HF System, enables comprehensive heart failure management in the home using pulmonary artery (PA) pressure, a leading indicator of congestion, and non-invasive vital sign data to improve care decisions.

Backed by definitive clinical evidence from the PROACTIVE-HF pivotal trial, the company is planning a U.S. launch of Cordella this year.

Endotronix, Inc., a privately held company dedicated to advancing the treatment of heart failure (HF) at the intersection of digital health and medtech, today announced Premarket Approval (PMA) from the U.S Food and Drug Administration (FDA) of the company’s Cordella™ Pulmonary Artery (PA) Sensor System for the treatment of New York Heart Failure (NYHA) class III heart failure patients. The Cordella platform is the first and only PA pressure-guided platform to offer comprehensive patient management using daily PA pressure and vital signs from home to guide therapeutic management and improve patient outcomes.

“This approval is very exciting and has the potential to transform care for HF patients. Endotronix’s solution provides a more complete clinical picture of the patient, so providers are able to make informed remote care decisions between office visits,” stated Dr. Liviu Klein, 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. “PROACTIVE-HF demonstrated that with Cordella clinicians achieved more optimal and timely dosing of key HF medications, significantly improving outcomes. In addition, the easy-to-use platform engages patients to drive consistent daily habits and self-awareness of trends to support sustainable lifestyle changes.”

Cordella is a proactive HF management platform that delivers daily PA pressure and other vital data via an implantable sensor and user-friendly, non-invasive health tools, respectively, to a managing HF clinician for remote patient care. This information guides clinical decision-making and medication dosing while enhancing the adoption of guideline-directed medical therapy (GDMT) to reduce congestion and improve outcomes. Regulatory approval was based on the PROACTIVE-HF trial, which demonstrated a markedly low 0.159 rate of heart failure hospitalization and all-cause mortality at 6 months.

In addition, Cordella enables:

• Seated PA pressure measurements, preferred by most patients, with a handheld reader.
• Patient visibility into key health trends to support healthy lifestyle changes.
• Secure messaging on a tablet between the clinical team, patient, and caregiver to support remote care.
• Reimbursement for implantation and ongoing management through existing reimbursement pathways.

“At Endotronix, we firmly believe that innovation can drive patient care excellence in the home and ultimately change outcomes for patients with heart failure, one of the largest cost categories in healthcare. The FDA’s approval validates this foundational belief and is a major milestone for our company and the field of HF management,” commented Harry Rowland, CEO and co-founder of Endotronix. “With this approval, we will deliver proactive, comprehensive care that extends optimal HF therapeutic management to more patients, keeping them out of the hospital and living more fulfilling lives.”

Endotronix will launch Cordella in the U.S. later this year. The company also shared that they have submitted a dossier for CE Mark review and expect a decision on European market access in 2025.

About Endotronix
Endotronix innovates at the intersection of medtech and 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.

In the U.S., the Cordella PA Sensor System is Rx Only. CAUTION: Federal law restricts this device to sale by or on the order of a physician.
In Europe, the Cordella PA Sensor System is Exclusively for Clinical Investigation.
The Cordella HF System is commercially available in the U.S. and Europe.

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.

The Toronto-based biotech startup has the potential to reshape the way we approach pain relief.

Every 65 minutes and 22 seconds.

That’s how often someone dies from an opioid overdose in Canada. On average, it works out to about 22 people per day. By comparison, at the height of Covid-19, the death toll in 2020 was about 11 per day. All told, opioids—a class of prescription pain-relieving medications that include heroin, fentanyl, morphine, oxycontin, among others—have killed more than 42,000 Canadians since tracking began in 2016.

According to the OECD (Organization for Economic Cooperation and Development), Canada holds the dubious distinction of having the second highest rate of opioid–related deaths, just behind the U.S.

In the face of such brutal numbers, it’s clear that combatting the opioid epidemic requires an all-hands-on-deck approach, integrating everything from health care providers and emergency services to all levels of government. A pair of scientists-turned-startup-entrepreneurs, however, believe that the private sector, specifically their emerging biotech company, can play a critical part in solving this widespread and devastating issue.

Mike Cooke and Molly Shoichet are the co-founders of AmacaThera, a Toronto-based venture offering a novel way to provide pain relief without the use of opioids. Their breakthrough product, which is currently in clinical trials, was spun out of research led by Shoichet from her lab at the University of Toronto, where she’s a professor in the Department of Chemical Engineering and Applied Chemistry. Cooke, who hails from the U.K., worked in the lab studying stem cells while pursuing his PhD. In 2016, the two joined forces (she as chief science officer; he as CEO) with a vision, Cooke says, of “translating their academic research into real-world products” that have the potential to herald a promising avenue for long-term opioid reduction strategies.

The FedEx of therapeutic delivery

At the core of the company’s technology is its proprietary hydrogel platform. The AmacaGel, as it’s known, is a blend of hyaluronic acid (the same ingredient commonly used in anti-aging cosmetics), and methyl cellulose which, when combined with the hyaluronic acid, gives the material its gel consistency. When injected by syringe into a patient’s soft tissue, the gel acts as a kind of carrier system for existing drugs like an anesthetic, or other therapeutics that have been mixed with it, allowing their gradual release into the body.

Shoichet likens AmacaThera to FedEx, whereby it provides the protective packaging (the gel) for what’s inside (the drugs) and then determines where and when they need to be deployed. “We’re not changing the drugs, we’re changing the delivery method,” adds Cooke. “That’s why we call it a platform.”

How it works: a game-changer in pain management

To understand what sets AmacaThera’s technology apart as it relates to the opioid epidemic, is to understand why opioids are often prescribed in the first place. Anyone that’s undergone surgery—from major dental work to having a hip replaced—knows that the recovery period can be painful. During the procedure, an analgesic is typically administered to the area, but tends to wear off within hours, leaving most patients to rely on a prescription narcotic for relief. Studies have shown that this leads to millions of cases of opioid addiction each year.

In contrast, the AmacaGel platform’s controlled release mechanism would ensure a steady supply of opioid-free pain medication over three days, effectively numbing the surgical site for an extended duration, far beyond the 12 to 18 hour window of conventional local anesthetics. Cooke and Shoichet theorize this prolonged-release formulation not only obviates the need for frequent opioid administration, but also alleviates the intensity of postoperative pain, thereby safeguarding individuals from the perils of addiction and overdose. It’s against this backdrop that the company offers a lifeline in the battle against opioid misuse.

“It’s been so exciting to see our research invention of AmacaGel be the basis of AmacaThera and now to see it applied in people,” Shoichet says. “Our goal is to make products that will make a difference in people’s lives. With our first product, we aim to both alleviate pain and put a dent in the opioid crisis, thereby filling unmet medical and societal needs.”

“Our goal is to make products that will make a difference in people’s lives. With our first product, we aim to both alleviate pain and put a dent in the opioid crisis, thereby filling unmet medical and societal needs.”

Moreover, Cooke explains, its efficacy in controlling pain not only fosters optimal healing, but the downstream effect may be a boon to our struggling health system. “If you have that rapid pain control, you’ll get better recovery. You can get people out of the hospital quicker and by reducing their length of stay, you can increase hospital capacity, and serve more patients. These are huge drivers and we’re building the company with that mentality of efficacy and efficiency.”

Beyond the operating room: exploring diverse applications

While the company’s first formulation, AMT-143, is focused on post-surgical pain management, Cooke and Shoichet are betting that AmacaThera’s utility could extend far beyond the confines of the operating room. Consider, for example, the ability to deliver cancer-fighting treatment to otherwise difficult-to-reach areas of the body, like the lungs or the brain. “With this technology, you could inject chemotherapy right into the tumour,” Cooke explains. “Because it’s localized, you could have greater efficacy because more of the drug would infiltrate the tumour directly, so that you’re decreasing the systemic toxicity overall.”

That AmacaThera’s platform is compatible with other materials—small molecules, antibodies, stem cells, proteins, and peptides, to name a few—effectively means patient outcomes could be improved across multiple therapeutic areas. A wide swath of rigorous scientific research has come out of Shoichet’s lab (underscored by over 325 peer-reviewed publications) shows how the technology could be used to treat not just certain cancers, but blindness, spinal cord injuries, and strokes.

Further still, when one considers the possibility of extending the product to patients of the non-human variety (think: dogs and cats, but also farm animals), its versatility could make it a cornerstone in various medical disciplines. “People pay a lot of money to care for their pets,” Cooke points out, “so there are active discussions with animal health pharma companies looking at licensing this technology.”

In this landscape, AmacaThera emerges as a revolutionary solution, offering a paradigm shift in the range of patient treatment options available, while holding the promise of mitigating some of society’s biggest challenges. “I believe in this technology because I’ve seen it work with my own eyes,” says Cooke. “As we learn more about the product itself, the manufacturing, the usages, how it performs as we advance to human trials, and the more positive aspects we’re uncovering, the more exciting it gets.”

Given the potential market opportunity (surgery alone is performed on over 51 million Americans every year, with opioids the primary means of pain control), AmacaThera has, unsurprisingly, created alot of excitement among investors as well. Last November, the company announced it secured a $4 million Series A extension from investors, including Lumira Ventures and StandUp Ventures, both of which RBCx proudly backs as a Limited Partner.

RBCx also supports the young startup with its day-to-day banking needs. “We’ve been with RBCx since the very start and it just makes life a lot simpler and more efficient. The fewer headaches I have, the more time we can devote to focusing on the science,” Cooke says, adding that the opportunity to network at RBCx-hosted client events with our thought leaders is a “big deal…I see that you’ve hired a few people from Silicon Valley Bank, so it will be exciting to see what RBCx does in the future.”

And yet, despite banking support, deep-pocketed investors, a best-in-class product, and Shoichet’s own prior experience launching two other startups, the co-founders acknowledge that entrepreneurship has been anything but easy. “When you start on this journey, you don’t know how hard this is going to be; it takes a certain type of crazy to want to do this because raising capital has been by far the toughest part,” says Cooke.

“When you start on this entrepreneurial journey, you don’t know how hard this is going to be. It takes a certain type of crazy to want to do this because raising capital has been by far the toughest part.”

“The number one comment we’ve had from investors is congratulations on being so relentless. They’re amazed by the tenacity of this company and how it just keeps going,” says Cooke. “And that’s the key: we’re going to take this all the way to the finish line.”

To get there, AmacaThera is using the cash infusion to speed up testing and development of AMT-143, as well as advancing other formulations through their pipeline before going out to fundraise again later this year. And while they’re the first to acknowledge that Canada remains a tough economic environment for tech startups, the duo remain undaunted, driven by a profound commitment to their mission. “In 20 years, we envision a successful company with products that make lives better,” says Shoichet. “There’s nothing more exciting than working toward creating a better tomorrow with innovative ideas.”

“Getting the product in humans and moving it forward is such a huge privilege,” adds Cooke. “We’re privileged to have the investors, we’re privileged to have the team. I never want to lose sight of that. It would be great to have invented something going into potentially millions and millions of people. But, to be honest, I think if we’re able to improve even one person’s life, that would be a huge achievement.”

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 (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

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.

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

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 8^th, 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

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

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

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: 18^th 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: 18^th 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

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

• 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

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

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 18^th, 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, 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

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 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, 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

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, 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

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:

SOURCE Haemonetics Corporation

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.

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

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.

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 Diakines^TM, 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, 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

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.

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

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.

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

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 

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.

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, 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 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, 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.