Catabasis Rocked by DMD Trial Failure, Will Scrap Study and Explore Strategic Options – BioSpace

Shares of Catabasis Pharmaceuticals have plunged more than 64% in premarket trading following Mondays announcement the companys last-stage Duchenne muscular dystrophy treatment failed to meet the trials primary and secondary endpoints. The company will scrap the program and explore strategic options.

On Monday, Boston-based Catabasis said its Phase III PolarisDMD trial of edasalonexent in Duchenne muscular dystrophy (DMD) did not meet the primary endpoint, which was a change from baseline in the North Star Ambulatory Assessment (NSAA) over one year of edasalonexent in comparison to placebo. The study also failed to meet its secondary endpoint of timed function tests. Those tests included time to stand, a 10-meter walk/run and a four-stair climb. With the failed study, Catabasis said it is ceasing activities related to the development of edasalonexent including the ongoing GalaxyDMD open-label extension trial. As a result, Catabasis will work with its advisers to explore its options moving ahead.

Chief Executive Officer Jill C. Milne expressed her disappointment in the trial results. She said the entire Catabasis team has tirelessly worked to find a treatment for DMD. Although the Phase III PolarisDMD study failed, Milne said she hopes the data from the study can be used to benefit ongoing and future research into DMD.

Edasalonexent is an investigational oral small molecule designed to inhibit NF-kB, a key driver of skeletal and cardiac muscle disease progression, which was seen as a potential foundational therapy for all patients affected by DMD, regardless of their underlying mutation. Duchenne muscular dystrophy causes a progressive loss of muscle strength attributable to a loss of a protein called dystrophin, which normally protects muscle fibers from breaking down. Approximately 15,000 U.S. patients are affected with Duchenne, with a total of about 300,000 patients worldwide. Currently, there are two DMD treatments that have been approved by the U.S. Food and Drug Administration. Sarepta Therapeutics Exondys 51 was approved in 2016 for DMD patients amenable to skipping exon 51. Last year, Sarepta secured another FDA approval for Vyondys 53, which was green lit for patients amenable to skipping exon 53.

The Catabasis Phase III trial was designed to evaluate the safety and efficacy of edasalonexent in boys ages 4 to 7 years. It included 131 boys with any mutation type who was not being treated with steroids. While the asset failed to meet its endpoints, Catabasis said edasalonexent was well-tolerated and there were no treatment-related serious adverse events and no dose reductions during the study. Data from the PolarisDMD trial will be further analyzed and are expected to be presented at an upcoming scientific conference and published.

Pat Furlong, CEO of the advocacy group Parent Project Muscular Dystrophy, called the results of the study disheartening. Like Milne, Furlong expressed the hope the data collected over the course of the PolarisDMD study will add to the knowledge base of DMD to, one day, produce a foundational, long-term therapy for this disease.

Catabasis will provide an update on its financials in November. As of Sept. 30, Catabasis had cash and cash equivalents of approximately $52.9 million.

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Catabasis Rocked by DMD Trial Failure, Will Scrap Study and Explore Strategic Options - BioSpace