Edasalonexent

A few months after finally admitting defeat on their failed Duchenne MD program, Catabasis Pharmaceuticals is heading in a new direction with the purchase of Quellis Biosciences and their lead antibody for hereditary angioedema. Catabasis shared the news on Friday that they’re buying out Quellis, a rare disease-focused biotech that was unveiled over a year ago by Chris Garabedian and Perceptive Advisors’ PXV Fund. With it, Catabasis is snapping up QLS-215, a monoclonal antibody inhibitor of plasma kallikrein. The candidate is currently in preclinical development — but won’t be for long, according to Catabasis. In addition to the buyout, the Boston-based biotech closed a $110 million private placement to fund the completion of IND-enabling, Phase Ia and Phase Ib/II studies. Catabasis and Quellis are keeping the terms of their deal under wraps. But after the acquisition and private placement, Catabasis expects to have a cash runway through 2023. The company plans on filing an IND application for QLS-215 in the first half of 2022, and reading out initial Phase I results by the end of 2022. If all goes well, they hope to launch a Phase Ib/II trial in 2023, with initial results by the end of that year. Back in October, Catabasis acknowledged a final failure for its Duchenne MD drug edasalonexent. Three years earlier, CEO Jill Milne and the crew insisted they found reason for hope in data from a failed study, and that it justified a try in Phase III. After the primary, change in baseline on the North Star Ambulatory Assessment, and the secondary on timed function tests both came up short of statistical significance, Catabasis killed the effort — leaving them with one other drug in the pipeline, a preclinical program on autophagy. Drug hunters and developers working in neurological diseases will now have a new place to turn to for insights. The Gladstone-UCSF Center for Neurovascular Brain Immunology is launching with a $2.5 million donation and neuroimmunologist Katerina Akassoglou as the founding director. “Historically, neurological diseases have been classified as being only degenerative, inflammatory, or vascular,” she said in a statement . “But we now know, given recent insights from clinical research and the failure of many clinical trials, that this classification cannot explain how diseases start and progress, nor has it been able to identify the best potential drug candidates.” By integrating basic and clinical research on vascular biology, immunology and neuroscience, scientists at the new center aim to shed light on overlapping mechanisms behind diseases like multiple sclerosis, Alzheimer’s disease and traumatic brain injury — and have implications extending as far as neonatal brain injury, epilepsy, infectious diseases and psychiatric disorders. Add it all together, Akassoglou said, and their approaches in imaging and drug discovery may just “transform biomedicine.” To uncover the role of vascular abnormalities in the brain, they will bring together basic and clinical experts and develop new molecular diagnostics, while testing new therapies and biomarkers.

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, announced today that it has named Andrew A. Komjathy as Chief Commercial Officer. Mr. Komjathy brings to Catabasis more than 30 years of experience in commercial operations and sales including extensive experience in rare diseases. “Andrew brings valuable rare disease commercial experience including building high performing commercial teams and strategies for successful product launches,” said Jill C. Milne, Ph.D., Chief Executive Officer of Catabasis. “We are looking forward to leveraging his expertise as we prepare for the potential commercialization of edasalonexent for the treatment of Duchenne muscular dystrophy.” “Catabasis is in an exciting period progressing its Phase 3 trial of edasalonexent and preparing to potentially bring edasalonexent to market,” said Mr. Komjathy. “I am proud to be part of a team that puts patients first and thrilled to be joining their efforts to make a new treatment option available to the Duchenne community.” Mr. Komjathy brings deep rare disease and commercial experience, most recently from his role as Vice President, Commercial Sales at Alkermes, Inc. He has also held senior level commercial operating positions in the U.S. Multiple Sclerosis Business Unit at Genzyme, in the North American and Asia-Pacific regions for Shire Human Genetic Therapies, as Shire Global Franchise Leader for Fabry/Gaucher in Switzerland, and at Biogen. Mr. Komjathy holds an MBA from New York University Stern School of Business and his BS in Business Administration from Bucknell University. About Edasalonexent Edasalonexent (CAT-1004) is an investigational oral small molecule designed to inhibit NF-kB that is being developed as a potential foundational therapy for all patients affected by DMD, regardless of their underlying mutation. In DMD the loss of dystrophin leads to chronic activation of NF-kB, which is a key driver of skeletal and cardiac muscle disease progression. We are currently enrolling our global Phase 3 PolarisDMD trial to evaluate the efficacy and safety of edasalonexent for registration purposes. Edasalonexent is also being dosed in the open-label extension trial GalaxyDMD. In our MoveDMD Phase 2 trial and open-label extension, we observed that edasalonexent preserved muscle function and substantially slowed disease progression compared to rates of change in a control period, and significantly improved biomarkers of muscle health and inflammation. The FDA has granted orphan drug, fast track, and rare pediatric disease designations and the European Commission has granted orphan medicinal product designation to edasalonexent for the treatment of DMD. For a summary of clinical results, please visit . About Catabasis At Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families. Our lead program is edasalonexent, an NF-kB inhibitor in development for the treatment of Duchenne muscular dystrophy. The global Phase 3 PolarisDMD trial is currently enrolling boys affected by Duchenne. For more information on edasalonexent and our Phase 3 PolarisDMD trial, please visit . Forward Looking Statements Any statements in this press release about future expectations, plans and prospects for the Company, including statements about future clinical trial plans including, among other things, statements about the Company’s global Phase 3 PolarisDMD trial in DMD to evaluate the efficacy and safety of edasalonexent for registration purposes, including the anticipated timing for top-line results, potential timing for the filing of an NDA, and other statements containing the words “believes,” “anticipates,” “plans,” “expects,” “may” and similar expressions, constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development of the Company’s product candidates; whether interim results from a preclinical or clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products; availability of funding sufficient for the Company’s foreseeable and unforeseeable operating expenses and capital expenditure requirements; other matters that could affect the availability or commercial potential of the Company’s product candidates; and general economic and market conditions and other factors discussed in the “Risk Factors” section of the Company’s Quarterly Report on Form 10-Q for the year ended June 30, 2019, which is on the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. In addition, the forward-looking statements included in this press release represent the Company’s views as of the date of this press release. The Company anticipates that subsequent events and developments will cause the Company’s views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Company’s views as of any date subsequent to the date of this release.