Prime Highlights
- Capricor shares surged 70% after the FDA agreed to review new data on Duchenne drug deramiocel.
- An FDA panel had earlier voted nine-to-three against the drug’s effectiveness data.
Key Facts
- Capricor Therapeutics is developing deramiocel for Duchenne muscular dystrophy-related heart disease.
- Capricor held $237.9 million in cash at the end of June.
Background
Capricor Therapeutics shares jumped 70% on Friday after the company said the US health regulator is willing to review new data on its experimental Duchenne muscular dystrophy drug. The FDA advisory panel’s nine-to-three vote against the medicine Deramiocel’s efficacy findings caused the stock to plummet last month.
The panel had echoed concerns raised by FDA staff that Capricor changed how it measured the trial’s main outcomes, and questioned whether enrolled patients actually had Duchenne-related cardiomyopathy, given their normal heart function at the start of the study.
Capricor Chief Executive Linda Marban said late on Thursday that the company will submit an amendment with new 24-month data focused on improving upper limb muscle function, the main goal of its late-stage study. She noted that some panel members had responded more positively to the treatment’s arm and hand function data than to its contested heart benefits.
Marban said the FDA is willing to review the new information and will push back its decision deadline once it receives the data. The agency’s original decision had been expected in the fourth week of August. She added that the company continues working closely with the FDA on a potential path forward.
An analyst at Cantor called the FDA’s willingness to review the new data a potentially positive surprise, noting the agency would have little reason to extend its deadline if it had already ruled against approval. Capricor shares traded at $7.16 in the morning, still sharply below the $19.70 level seen before last month’s setbacks.
Analysts at Roth Capital said the FDA is likely to treat the resubmission as a major amendment, extending the review timeline by about three months. Capricor, which held $237.9 million in cash at the end of June, said it is pacing commercial launch spending while awaiting regulatory clarity.
