By Christy Santhosh
July 30 (Reuters) – Shares of Capricor Therapeutics fell nearly 60% in premarket 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.
“Based on the FDA negative stance on the trial, we expect it will likely issue another Complete Response Letter and not approve deramiocel,” said Piper Sandler analyst Edward Tenthoff.
Capricor shares have 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.
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.
While the FDA is not required to follow the advice of its outside experts, it often considers their recommendations when making decisions. The agency is expected to decide on the therapy by August 22.
(Reporting by Christy Santhosh in Bengaluru; Editing by Leroy Leo)
