Capricor Therapeutics’ Duchenne treatment is heading for an FDA rejection

Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall Street, and biotechnology. He is also a co-host of the weekly biotech podcast The Readout Loud and author of the newsletter Adam’s Biotech Scorecard. You can reach Adam on Signal at stataf.54.

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Capricor Therapeutics is trying to stave off the Food and Drug Administration’s near-certain rejection of its cell therapy for Duchenne muscular dystrophy by submitting results from an open-label extension of its failed Phase 3 study. 

It’s a stall tactic. Nothing more. The only option left for Capricor to secure approval of its treatment, called deramiocel, is to conduct an entirely new, randomized clinical trial.