Capricor Stock Tumbles After FDA Panel Rejects Evidence for Duchenne Cell Therapy - Capricor Therapeutics

Capricor Therapeutics Inc.

(NASDAQ:CAPR) stock nose-dived on Thursday after the FDA Advisory Committee voted against its Deramiocel, an investigational cell therapy for Duchenne muscular dystrophy (DMD).

The agency assigned a PDUFA target action date of August 22.

FDA Advisory Panel Questions Deramiocel Effectiveness Reuters reported that the FDA advisory panel voted 9-3 that the company failed to provide sufficient evidence supporting Deramiocel’s effectiveness, echoing FDA staff concerns over post hoc changes to key analyses after the late-stage trial concluded.

In the FDA briefing document released on Monday, the staff noted that the submitted data from Study HOPE-3, considered together with the results from Study HOPE-2, does not provide substantial evidence of effectiveness for deramiocel in DMD.