Some advisers viewed the upper-limb data more favorably, fueling hopes for a narrower approval focused on that benefit.
- Panelists said Capricor had not shown convincing evidence that Deramiocel treats DMD-related cardiomyopathy, calling the data “very fragile.”
- FDA reviewers questioned Capricor’s Phase 3 HOPE-3 analysis and said later statistical changes weakened confidence in the results.
- Capricor pushed back, saying the FDA relied on an unfinished draft rather than the final analysis plan.
Shares of Capricor Therapeutics, Inc. (CAPR) are on track for a record weekly selloff after an Food and Drug Administration (FDA) advisory panel voted 9-3 against Deramiocel, even as retail investors continue to bet that the Duchenne muscular dystrophy (DMD) therapy could still secure some form of approval.
CAPR stock has plunged 67% so far this week after falling 6% on Wednesday to mark its third straight session in the red.
FDA Panel Questions Deramiocel Data
The FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee concluded on Wednesday that Capricor had not shown substantial evidence that Deramiocel effectively treats cardiomyopathy in DMD. Several panelists said the data was not convincing enough.
“The word of the day seems to be fragile. The results were very fragile,” committee member Janet Turk Wittes said, adding that she did not see enough evidence of benefit to vote yes. Another panelist said the evidence was “really not that compelling,” despite the severe unmet need in DMD. The vote is advisory, not a final FDA decision. The agency is expected to rule on Capricor’s resubmitted application by Aug.22.
A major point of contention was how Capricor’s Phase 3 Hope-3 results should be analyzed. FDA reviewers argued that under an earlier statistical plan, the study did not show a statistically significant benefit over placebo at 12 months. They also questioned later changes to the analysis, saying they weakened confidence in the findings. Capricor strongly disputed that interpretation, saying that the agency relied on an unfinished draft rather than the final analysis plan completed before the study was unblinded.
CEO Linda Marban compared it to a professor “grading your term paper on an early draft you have never even submitted.” The company has also said the changes were made as its discussions with the FDA evolved, while regulators argued the later plan was not formally agreed upon.
Upper-Limb Data Could Support Narrower Approval
The panel vote focused specifically on Deramiocel’s effectiveness in DMD-related cardiomyopathy. This matters since Capricor is seeking approval for upper-limb impairment and/or cardiomyopathy in DMD patients ages nine and older.
Some advisers were somewhat more receptive to the upper-limb results. Committee member Christopher Coffey called the data “a little more favorable,” though he added that he remained unconvinced the therapy works. This distinction is now fueling speculation that the FDA could still consider a narrower approval centered on upper-limb function, although the agency has not signaled that such an outcome is planned.
As the panel meeting began, Capricor also announced that full Hope-3 results had been published in The Lancet. The company said Deramiocel slowed upper-limb decline by 54% versus placebo and delivered a “clinically meaningful cardiac benefit,” arguing that peer review provided outside validation of the trial and its methodology.
How Do Retail Traders Feel About CAPR?
On Stocktwits, CAPR’s retail sentiment hit a record 97/100, firmly in “extremely bullish” territory, as 24-hour message volume surged 258%. Several traders argued that the meeting became overly focused on statistical technicalities and an older analysis plan rather than the totality of the clinical evidence.
View this Stocktwits post
One trader predicted “conditional approval coming in spite of the 3-9 vote,” while another said the meeting had gone “sideways on statistical details.”
View this Stocktwits post
For updates and corrections, email newsroom[at]stocktwits[dot]com.<
