Key Takeaways
- Shares of CAPR jumped more than 100% during premarket hours Friday following the company’s announcement of an amended BLA submission for Deramiocel
- Last month, an FDA advisory committee rejected Deramiocel for treating DMD-associated cardiomyopathy by a 9-3 vote
- The biotech company is now targeting an upper limb skeletal muscle indication, backed by 24-month extension study results
- FDA officials have agreed to review the amended application and will adjust the PDUFA deadline accordingly
- The company faces a securities fraud class action lawsuit with a September 28, 2026 deadline for lead plaintiff applications
Shares of Capricor Therapeutics (CAPR) experienced a dramatic surge in Friday’s premarket session, soaring over 100% following the company’s disclosure that it intends to file an amended Biologics License Application for Deramiocel, its flagship therapeutic candidate.
Capricor Therapeutics, Inc., CAPR
This announcement follows a challenging period for the biotech firm. On July 27, 2026, CAPR experienced a devastating 64.5% plunge, tumbling from $19.70 to $7.00 per share. The collapse occurred after the FDA published briefing materials highlighting issues with modifications to Capricor’s statistical analysis methodology prior to a scheduled advisory committee evaluation.
Just 48 hours later, the situation worsened when the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee delivered a negative 9-3 vote against endorsing Deramiocel for treating cardiomyopathy associated with Duchenne muscular dystrophy. The stock plummeted an additional 36%, sliding from $6.57 to $4.19.
The FDA is currently evaluating the BLA with a scheduled PDUFA action deadline of August 22.
During Capricor’s second quarter 2026 earnings conference call, CEO Linda Marbán offered perspective on the advisory committee outcome. She emphasized that cardiomyopathy represented a secondary measure in the Phase 3 HOPE-3 trial rather than the study’s main objective.
“The Advisory Committee was not asked to vote on whether they believe the data on the HOPE-3 primary efficacy endpoint could support approval of the product, nor whether the overall benefit-risk profile of Deramiocel was favorable,” Marbán said.
Strategic Shift to Alternative Indication
Capricor has strategically repositioned its regulatory approach to seek approval for treating upper limb skeletal muscle dysfunction, which directly corresponds to the HOPE-3 trial’s primary efficacy measure. The amended BLA will incorporate 24-month data from the open-label extension phase along with supplementary analyses derived from the existing clinical evidence.
FDA representatives have indicated their willingness to evaluate the amended submission and will adjust the PDUFA target date following receipt of the modified application.
Class Action Litigation Underway
Despite the positive stock momentum, Capricor is navigating legal challenges. Law firm Bleichmar Fonti & Auld has initiated a securities fraud class action against the company and select senior management members.
The complaint contends that Capricor disseminated misleading information regarding Deramiocel and the reliability of clinical trial data underpinning the BLA submission. Additionally, the lawsuit claims the company concealed modifications to the predetermined statistical analysis framework implemented without FDA consent prior to BLA resubmission.
The legal action has been filed in the U.S. District Court for the Southern District of California. Affected shareholders have until September 28, 2026 to petition for lead plaintiff status.
While the existing PDUFA deadline stands at August 22, the forthcoming amended submission is anticipated to result in an extended review timeline.
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