Capricor soars on potential FDA review extension, filing amendments

News Drug Development Capricor soars on potential FDA review extension, filing amendments August 14, 2026 | 4 min read | Gabrielle Masson Twitter LinkedIn Facebook Email Print Capricor Therapeutics’ CEO Linda Marbán believes the FDA is willing to work with the biotech as the Aug. 22 deadline approaches for its Duchenne muscular dystrophy cell therapy deramiocel, with the biotech’s stock climbing 68% on the update. Capricor Therapeutics is working with the FDA as the approval deadline for the Duchenne muscular dystrophy cell therapy deramiocel looms, and the biotech’s CEO Linda Marbán says the agency is receptive to open-label extension data being added to the current submission.Marbán’s comments arrived during Capricor’s second quarter earnings call on Thursday, as investors wait to hear if the FDA will approve deramiocel after a contentious advisory committee meeting last month.

The CEO seemed to indicate that, despite the committee ultimately recommending against the therapy, the FDA remains open to an alternative regulatory path.“Following discussions with the agency subsequent to our advisory committee meeting, we plan to submit an amendment to our BLA that includes the 24-month open label extension data from the HOPE-3 study along with additional analyses on the existing data package in order to support a refined indication focused on the primary endpoint,” she said on a Thursday earnings call.“FDA has indicated it is willing to review this amendment and upon receipt to extend the PDUFA action date accordingly,” Marbán continued. “We are finalizing the timing of that submission and will provide an update as appropriate.”The Q2 call, held after market close on Thursday, sent Capricor’s shares soaring 68% to $6.95 on Friday morning.As of publication, the FDA has not responded to BioSpace’s request for comment on Marbán’s statements. The current PDUFA date for deramiocel is Aug.

22. That date was issued following a July 2025 rejection of the asset and subsequent resubmission by Capricor. “Because that review is ongoing, there is a limit to what I can say about our interactions with the agency,” Marbán said on the call.Nevertheless, she expressed optimism about deramiocel’s future.

“We are continuing to work closely with FDA on a potential path forward for deramiocel.”A key point of contention at the July adcomm was the appropriate indication for which deramiocel could be granted approval, specifically in relation to patients with cardiomyopathy.In the Phase 3 study of 106 boys and men with Duchenne muscular dystrophy (DMD), the primary endpoint was improving scores measuring upper limb performance using an aggregate DMD-specific tool that grades 22 functional activities across the shoulder, elbow and wrist. Cardiac function was a secondary endpoint.According to Capricor, the trial met both endpoints with statistical significance. But the FDA disagrees, arguing that the therapy did not meet HOPE-3’s prespecified primary and secondary efficacy endpoints, “showing no statistically significant difference between deramiocel and placebo at 12 months,” according to documents released ahead of the July 29 advisory committee meeting.

Capricor and the FDA have disagreed over the specific statistical analysis plan used to determine the impact of the investigational therapeutic.The agency used a draft version of that plan, rather than what Capricor intended to be the final version to determine efficacy, the company has said. During the adcomm FDA advisors called the data around the secondary endpoint “very fragile.” Ultimately, the committee voted 9-3 against approval, a decision based on the cell therapy’s efficacy in DMD cardiomyopathy.Now, the full dataset under the pre-specified model has been published in The Lancet, with the measure of the left ventricular ejection fraction in all patients no longer statistically significant.But Marbán argues that the FDA and the adcomm’s focus on cardiomyopathy is flawed, given that it was a secondary endpoint and measured change in ejection fraction across the full DMD population rather than in patients with established cardiomyopathy, which is the population the proposed indication addresses. FDA FDA advisers vote against approval of Capricor’s DMD therapy in chaotic adcomm meeting During a perplexing advisory committee meeting in which FDA reviewers focused largely on the Phase 3 HOPE-3 trial’s secondary endpoint of cardiac function, experts pointed to “very fragile” data.

The committee voted 9-3 against approval of deramiocel. July 29, 2026 · 8 min read · Heather McKenzie Read more “Let me be clear that the HOPE-3 primary endpoint was unaffected, and its significance stands both statistically and clinically,” Marbán said on the call. Halting all other pipeline projects As deramiocel’s fate hangs in the balance, Capricor has decided to put all other pipeline work on hold until the company receives “further regulatory clarity.”That includes StealthX, an exosome-based vaccine that was in a Phase 1 trial for COVID-19 that was funded and run by the U.S.

Department of Health and Human Services and concluded in June. The program is designed as an alternative to mRNA vaccines, with Capricor believing its vaccine platform holds potential across a variety of indications, including rare diseases.“The StealthX platform is advancing several therapeutic programs, with a focus on muscle-targeted delivery of siRNA, proteins and small molecules,” Capricor said this May in its first quarter earnings release.The biotech also has another DMD candidate made from cardiosphere-derived cell exosomes, the same approach underlying deramiocel. The unnamed candidate is in preclinical development, according to Capricor’s online pipeline.As of publication, the biotech has not responded to BioSpace’s questions regarding the pipeline changes.Deramiocel expansion trials into younger patients and Becker muscular dystrophy also hinge on the ongoing regulatory review of the asset, the biotech said.Capricor has also launched regulatory discussions for deramiocel in Europe and Japan, and has operational manufacturing in the U.S.

to support potential commercialization, according to the company release. “Capricor is advancing commercial readiness at a slower pace pending regulatory clarity.” Regulatory Capricor CEO won’t rule out legal action against FDA after negative adcomm After an FDA advisory committee voted 9-3 on Wednesday against recommending Capricor Therapeutics’ Duchenne muscular dystrophy cardiomyopathy drug for approval, stakeholders allege an “ulterior motive” and “biased intentions.” July 31, 2026 · 6 min read · Heather McKenzie Read more As of June 30, Capricor had $237.9 million on hand, reflecting a net loss of $40.7 million for the second quarter, or 70 cents per share. That’s up from a loss of $25.9 million for the same period last year.

The biotech currently has zero revenue stream.Capricor believes its cash runway can stretch “for at least the next twelve months,” and expects to provide longer-term financial guidance “following greater regulatory clarity,” the company said. Twitter LinkedIn Facebook Email Print Cell therapy Adcomms Duchenne muscular dystrophy Rare diseases Capricor Therapeutics Gabrielle Masson Gabrielle is a senior editor at BioSpace. You can reach her at gabrielle.masson@biospace.com.