By Christy Santhosh
July 30 (Reuters) – Shares of Capricor Therapeutics fell 50% in morning trading on Thursday after the U.S. FDA’s panel of experts voted against the effectiveness data of its cell therapy for a heart condition related to Duchenne muscular dystrophy.
Late on Wednesday, the panel voted nine-to-three against deramiocel, aligning with concerns of the FDA’s staff that the company had not provided sufficient evidence of the therapy’s effectiveness, while questioning changes to key analyses after its late-stage study was completed.
Capricor shares had already fallen more than 66% through its last close since the Food and Drug Administration staff raised concerns about the company’s lead therapy candidate in their briefing documents on Monday. The company had a market value of about $380 million as of Wednesday’s close.
Panel members who voted against deramiocel cited unconvincing cardiac-benefit evidence, missing data, and uncertainty over whether the study’s heart-function measure reflected a meaningful patient benefit.
They said a larger, longer trial with different end goals may have provided clearer evidence of whether the therapy preserves function.
The FDA relied on an outdated, unsigned analysis plan, Capricor CEO Linda Marbán said, arguing that it “pigeonholed the whole meeting into a discussion of which stat plan was right, which imputation model was right and how the data should be looked at for one or two patients.”
Marbán said that focus ignored the totality of the evidence and the impact on people with DMD and their families.
Cantor Fitzgerald analyst Kristen Kluska said the FDA staff hosting the meeting seemed “extremely negative during the entire conversation and were going into this meeting with the mindset to reject the application”.
Last year, the FDA had declined to approve the therapy and sought additional data after saying the treatment did not meet efficacy requirements.
“We remain focused on working with the FDA toward potential approval” ahead of the action date, Capricor said in a statement.
The agency is expected to issue its decision by August 22.
(Reporting by Christy Santhosh in Bengaluru; Editing by Leroy Leo)
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