Celtaxsys Inc. Receives $5M Research Award to Advance Once Daily, Oral Anti-Inflammatory Medicine into Phase 2 CF Patient Lung Function Preservation Trial

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ATLANTA, June 2, 2015 /PRNewswire/ — Celtaxsys, a clinicalstage drug development company focused on advancing care for patients sufferingfrom inflammatory diseases, including those with rare and orphan inflammatorydiseases, announced today that it has received a development award for $5million from Cystic Fibrosis Foundation Therapeutics, Inc. (“CFFT”),the non-profit drug discovery and development affiliate of the Cystic FibrosisFoundation The development award will help support a Phase 2 clinical trial ofthe Company’s lead development candidate, a once daily, oral anti-inflammatorydrug CTX-4430 in adults with cystic fibrosis (“CF”). CTX-4430 is aselective inhibitor of Leukotriene A4 Hydrolase that targets re-balancing of apatient’s over activated inflammatory response without increasingsusceptibility to infection. CTX-4430 has been granted orphan status for CF inboth the US and the EU.

 “We are grateful for the support of CFFT and the manyleading researchers in CF who have helped us design a trial to assess thebenefits of chronic anti-inflammatory therapy in CF patients. Our Phase 1CTX-4430 studies, including the study in CF patients, demonstrated thatCTX-4430 is well tolerated. Importantly, in only two weeks in CF patients,CTX-4430 demonstrated positive results on well-established CF biomarkers,including encouraging reductions in sputum neutrophils, one of the primary”bad actors” in the CF lung that clogs the airways of CF patients. Inaddition, CTX-4430 demonstrated a statistically significant reduction versusplacebo in neutrophil elastase which has been shown to predict the onset ofbronchiectasis and which can degrade CFTR and other protective proteins,”said Greg Duncan, Chief Executive Officer of Celtaxsys, Inc.

 “The Celtaxsys team is delighted by CFFT’s decision tohelp fund this study and believe CTX-4430 could represent an important newmedicine for people with cystic fibrosis, either alone or in conjunction withother therapies, irrespective of the patient-specific CF gene mutation. Webelieve CTX-4430’s ability to reduce neutrophil elastase in the airways of CFpatients has the potential to boost the effect of other important existingtreatment regimens, including CFTR modulators and airway clearancetherapies,” added Eric Springman, PhD, Chief Scientific Officer atCeltaxsys.

Principal investigators for the trial in the U.S. and EU,respectively include: Steven M. Rowe, MD, MSPH, Professor in the Division ofPulmonary, Allergy and Critical Care Medicine and Director of the Center forCFTR Detection at the University of Alabama at Birmingham School of Medicine,and Professor Stuart Elborn, CBE, Dean School of Medicine, Dentistry andBiomedical Sciences and Professor of Respiratory Medicine at Queens UniversityBelfast and Consultant Physician in the Adult CF Centre at Belfast CityHospital. Each have played central roles in helping to design this potentiallyground breaking study. “These two world-class thought leaders spearheadedimportant innovations in CFTR research and are now advancing efforts towardsanti-inflammatory research, one of the next frontiers in CF research asidentified by the CFFT,” commented Sanjeev Ahuja, MD, Chief MedicalOfficer at Celtaxsys.

Celtaxsys will submit its Phase 2 clinical trial protocolfor the treatment of CF with CTX-4430 to the U.S. Food and Drug Administration(“FDA”) this quarter and anticipates beginning this study in thesecond half of 2015, pending FDA approval of the protocol.

About Cystic Fibrosis: Cystic fibrosis (CF) is alife-threatening disease that affects the lung and digestive system and impactsabout 70,000 patients worldwide. CF is caused by mutations in the Cystic FibrosisTransmembrane conductance Regulator (CFTR) gene leading to abnormal CFTRprotein functioning, the result of which causes the body to accumulateexcessive levels of unusually thick mucus in the lungs. This excessive stickymucus in the lungs is a site for infections that can require hospitalization.In the pancreas and GI tract CFTR protein dysfunction results in malabsorptionof nutrients and sometimes intestinal blockage. Respiratory distress in CF,defined as acute difficulty in breathing, infection and/or hospitalization, ismost commonly related to lung infection and inflammation induced lung tissuedamage attributable to an overwhelming and dysfunctional response byderegulated neutrophils. Treatment of this lung inflammation is, therefore,thought to be key to improving CF patients’ lung health and well-being. Learn more about CF.

 About CTX-4430: CTX-4430 is a once-daily oral drug candidatecurrently undergoing clinical trials for inflammatory diseases. It is a novelsmall molecule inhibitor of Leukotriene A4 Hydrolase (LTAH4), the key enzyme inthe production of the potent inflammatory mediator Leukotriene B4 (LTB4). LTA4Hand LTB4 have been strongly implicated in the pathogenesis of many diseasesinvolving inflammation, including cystic fibrosis.

 About Celtaxsys: Celtaxsys is aprivately-held drug discovery and development company focused on advancingmedicine to treat patients suffering from serious inflammatory diseases. Thecompany is building a sustainable pipeline of first-in-class immuno-modulators,the most advanced of which is CTX-4430. Our follow-on molecules enable newintellectual property and exhibit differentiated properties that enableoptimization for alternate routes of administration. For more information,visit www.celtaxsys.com.