STAT+: Capricor Therapeutics’ Duchenne treatment is heading for an FDA rejection

Publicly, the company maintains confidence in its drug. It’s not clear why Molly Ferguson/STAT 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. This story first appeared in Adam’s Biotech Scorecard, a subscriber-only newsletter. STAT+ subscribers can sign up here to get it delivered to their inbox. 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. STAT+ Exclusive Story Already have an account? Log in This article is exclusive to STAT+ subscribers Unlock this article — plus in-depth analysis, newsletters, premium events, and news alerts. Already have an account? Log in View All Plans To read the rest of this story subscribe to STAT+. Subscribe 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.