Capricor Therapeutics (Capricor (CAPR)) had a rough Thursday. The company's stock nose-dived after an FDA advisory committee voted against its investigational cell therapy Deramiocel for Duchenne muscular dystrophy (DMD). The vote was 9-3, with the panel concluding that the company hadn't provided sufficient evidence that the drug actually works.
This wasn't a total surprise. Earlier in the week, FDA staff released a briefing document that was pretty skeptical of the data. They noted that the results from the pivotal Phase 3 HOPE-3 trial, even when combined with earlier HOPE-2 data, didn't add up to "substantial evidence of effectiveness." The staff wrote that HOPE-3 showed "small and variable changes" in upper limb function and cardiac imaging that "did not reach statistical significance and are, thus, difficult to interpret." Ouch.
The FDA has a PDUFA target action date of August 22 for Deramiocel, so this advisory committee vote is a big deal. While the FDA doesn't have to follow the panel's recommendation, it often does. The odds just got a lot longer for Capricor.
The Company's Counterargument: 'But The Lancet Published It!'
Capricor isn't going down without a fight. On Wednesday, just a day before the panel vote, The Lancet—a prestigious peer-reviewed medical journal—published the full results from the HOPE-3 trial. The company was quick to highlight that the manuscript went through independent expert peer review, which they see as external validation of their trial design, statistical methods, and findings.
The results are based on the company's prespecified Statistical Analysis Plan version 3.0 (SAP 3.0). And they do look impressive on paper: a 54% slowing of upper limb disease progression, with a p-value of 0.03. That's statistically significant, and in a disease where functional decline is usually relentless, it sounds like a big deal.
Dr. Craig McDonald, a professor at UC Davis Health and the lead author of the publication, called the results "a landmark moment for the Duchenne community." He emphasized that HOPE-3 is the first Phase 3 trial to show a significant functional benefit in a largely non-ambulatory DMD population, and that the concurrent cardiac benefits support a consistent treatment effect across muscle types.
But here's the rub: the FDA panel wasn't convinced. The agency's concern seems to be that the company changed its analysis plan after the trial was over—a classic red flag in drug development. Post hoc analyses can be useful for generating hypotheses, but they're not the same as pre-specified endpoints. The panel apparently felt that the data, even with the Lancet publication, didn't overcome that skepticism.
What This Means for Capricor
For now, the market has spoken. Capricor shares were down 52.36% at $3.13 at the time of publication, hitting a new 52-week low. That's a brutal haircut for a company that was hoping for a regulatory win in a few weeks.
The next big date is August 22, when the FDA makes its final decision. The agency could still approve Deramiocel despite the panel's vote, but it's an uphill climb. Alternatively, the FDA could ask for more data or another trial, which would mean more years of development and more cash burn.
For investors, this is a reminder that peer-reviewed publications and regulatory approval are two very different things. The Lancet may have validated the science, but the FDA panel is looking for something else: convincing evidence that the drug works in a way that meets the agency's standards. And right now, they're not seeing it.
