Health and Healthcare
Does Catabasis Pharma Have a Chance After This DMD Study Flop?
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Catabasis Pharmaceuticals Inc. (NASDAQ: CATB) was absolutely devastated on Tuesday after the company announced top-line results from its late-stage study in muscular dystrophy. As a result, the company is evaluating what to do going forward.
In terms of the specifics, the results come from its Phase 3 PolarisDMD trial of edasalonexent in Duchenne muscular dystrophy (DMD). Unfortunately, the study did not meet the primary endpoint, and the secondary endpoint did not show any statistically significant improvements.
The primary endpoint was a change from baseline in the North Star Ambulatory Assessment over one year of edasalonexent compared to placebo. While the secondary endpoint was timed function tests (time to stand, 10-meter walk/run and four-stair climb).
Catabasis is stopping activities related to the development of edasalonexent, including the ongoing GalaxyDMD open-label extension trial.
On the plus side, edasalonexent was observed to be generally safe and well-tolerated in this trial, but this isn’t even close to enough without the efficacy. There were no treatment-related serious adverse events and no dose reductions.
Looking ahead, the firm plans to work with external advisors to explore and evaluate strategic options going forward.
The company plans to report its most recent quarterly numbers in November. The company had cash and cash equivalents totaling $52.9 million as of the end of September.
Even before Tuesday’s move, Catabasis Pharmaceuticals stock had underperformed the broad markets with a decline of about 9% year to date. In the past 52 weeks, the stock was up about 8%.
Catabasis Pharmaceuticals stock traded down about 69% to $1.67 on Tuesday, in a 52-week range of $1.63 to $8.59. The consensus price target is $33.00.
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