STAT+: FDA expert committee reviews Capricor Therapeutics’ Duchenne drug

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.Welcome to STAT’s live blog coverage of the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee meeting, which on Wednesday will be reviewing deramiocel, a cell therapy for Duchenne muscular dystrophy developed by Capricor Therapeutics. The stakes are rather high. Capricor has positioned deramiocel as one of the only treatment options for teenagers and young men with Duchenne who have already lost their ability to walk. In a Phase 3 study, deramiocel preserved upper-arm function and staved off the heart failure most patients eventually experience, Capricor said last December, achieving the primary and secondary goals of the trial with statistical significance. The FDA, in a harshly critical review posted publicly on Monday, disagreed with the company’s conclusions. In its own analysis, deramiocel failed to separate from placebo on the upper-limb and cardiac function endpoints, agency reviewers concluded. Furthermore, Capricor made numerous changes to the deramiocel study’s statistical analysis plan after it was completed, further muddying the outcome, the FDA said. STAT+ Exclusive Story Already have an account? Log in This article is exclusive to STAT+ subscribers Unlock this article — plus daily coverage and analysis of the biotech sector — by subscribing to STAT+. Already have an account? Log in View All Plans To read the rest of this story subscribe to STAT+. Subscribe