FDA says Capricor Therapeutics’ Duchenne muscular dystrophy cell therapy deramiocel flunked its own pivotal trial. In briefing documents posted ahead of Wednesday’s advisory committee vote, the agency wrote that the HOPE-3 study “did not meet its pre-specified primary and secondary efficacy endpoints,” finding no statistically significant difference between deramiocel and placebo at 12 months. That directly contradicts what Capricor told investors in December: that the Phase 3 trial hit both endpoints.
The dispute turns on paperwork. FDA reviewers say Capricor generated at least two revised versions of its statistical analysis plan after the trial’s blinded portion ended, changing how the primary and secondary endpoints were defined and how missing data got handled. Reviewers also flagged that 42% of deramiocel patients had hypersensitivity reactions versus 15% on placebo, a gap wide enough that patients could guess which arm they were in despite blinding.
Capricor isn’t backing down. In a statement Monday, CEO Linda Marbán said the agency built its case on “SAP version 1.1,” an unsigned draft she called obsolete, while the company’s real numbers run on “SAP version 3.0,” finalized before unblinding. She told BioSpace the FDA is “refusing to meet with us.” Shares dropped more than 65%, to $6.53, on the news.
This isn’t Capricor’s first stumble. FDA rejected the original application last July after canceling a planned adcomm without warning the company first. A second rejection, with the agency publicly branding the data set statistically hollow before the vote even happens, would be a far harder story for Capricor to spin. The adcomm meets Wednesday; FDA’s own action date is August 22.
Sarah Chen