Capricor Therapeutics (NASDAQ:CAPR) said its biologics license application for deramiocel remains under U.S. Food and Drug Administration review, while the company prepares to submit an amendment seeking a refined indication centered on upper limb skeletal muscle function in Duchenne muscular dystrophy, or DMD.
The FDA’s current target action date for the application is Aug. 22. However, Chief Executive Officer Linda Marbán said the agency has indicated it is willing to review the planned amendment and extend the PDUFA action date after receiving it. Capricor is finalizing the timing of its submission, which will include 24-month open-label extension data from the HOPE-3 study as well as additional analyses of its existing data package.
Company Pursues Upper Limb Indication
Marbán said the advisory committee’s voting question focused narrowly on cardiomyopathy, while the HOPE-3 study’s primary endpoint measured upper limb function. The company’s original requested indication in its BLA, dating to 2024, was for DMD cardiomyopathy.
According to Marbán, the study’s measure of cardiomyopathy was a key secondary endpoint and evaluated changes in ejection fraction across the overall DMD study population rather than solely among patients with established cardiomyopathy. HOPE-3 was designed and powered to assess a skeletal functional endpoint involving upper limb function, she said.
“We are continuing to work closely with FDA on a potential path forward for deramiocel focused on an upper limb skeletal muscle indication reflected in the primary efficacy endpoint of HOPE-3,” Marbán said.
Capricor said the HOPE-3 primary endpoint was unaffected by a statistical-model issue identified during peer review and discussions with the FDA and The Lancet. The company reverted to Statistical Analysis Plan version 3.0, which had been established before unblinding and included an interaction term for age and baseline variables.
Under the pre-specified model published in The Lancet, the treatment difference for left ventricular ejection fraction among all patients was 1.8 percentage points, with a P value of 0.09. Capricor had previously reported a 2.4-percentage-point difference with a P value of 0.04. Marbán said the result in the pre-specified cardiomyopathy subgroup was unchanged, showing a 2.8-percentage-point treatment difference with a P value of 0.02.
For the primary upper limb endpoint, Capricor said deramiocel demonstrated a statistically significant slowing of disease progression measured by PUL 2.0. The company reported a 4.55% mean difference in favor of deramiocel, with a P value of 0.029, corresponding to a 1.2-point absolute change in total PUL 2.0.
Manufacturing and Commercial Plans Being Slowed
Capricor said it will continue commercial-readiness activities at a slower pace pending greater regulatory clarity. Its San Diego GMP manufacturing facility is operational and positioned to support an initial commercial launch if deramiocel is approved, according to Marbán.
The company is continuing expansion work on the facility’s second floor, with full validation and FDA approval of the expanded space estimated in 2027. Capricor also appointed Michael Maurer as chief commercial officer. Marbán said Maurer has DMD and rare-disease commercial experience and has been building the launch organization with the company’s market-access leadership.
The FDA also conducted a Bioresearch Monitoring Inspection in July and issued a Form 483 with one observation. Capricor said it submitted its response and is awaiting agency feedback.
NS Pharma Dispute Moves Toward Arbitration
Capricor withdrew, without prejudice, its motion for a preliminary injunction in its dispute with NS Pharma. The state court had been scheduled to hear that motion on Aug. 10, ahead of the then-expected PDUFA date.
Marbán said the company concluded that pursuing arbitration after the FDA decision would provide a more complete regulatory record for both parties. Capricor expects arbitration to begin this fall.
The company continues to seek rescission of its U.S. distribution agreement with NS Pharma, maintaining that the agreement’s pricing structure is fundamentally flawed and could impede patient access, Marbán said.
Second-Quarter Financial Results
Chief Financial Officer A.J. Bergmann said Capricor held approximately $237.9 million in cash equivalents and marketable securities as of June 30. The company reported no revenue for either the second quarter of 2026 or the same period in 2025.
- Total operating expenses were approximately $42.9 million in the second quarter, compared with $27.7 million a year earlier.
- Second-quarter net loss was approximately $40.7 million, or $0.70 per share, compared with a loss of $25.9 million, or $0.57 per share, in the prior-year period.
- Net loss for the first six months of 2026 was approximately $74.7 million, compared with $50.3 million in the prior-year period.
- Capricor’s accumulated deficit stood at approximately $379.6 million as of June 30.
Bergmann attributed higher expenses primarily to clinical, regulatory, manufacturing and commercial-infrastructure investments for the company’s DMD program. Capricor said it has flexibility in deploying capital for the remainder of the year and is pacing certain commercial expenditures as its regulatory timeline develops.
Marbán added that pipeline work not directly related to deramiocel is on hold pending regulatory clarity. The company has initiated regulatory engagement in Europe and Japan, while potential expansion into younger DMD patients and Becker muscular dystrophy will be staged according to the U.S. regulatory path for deramiocel.
About Capricor Therapeutics (NASDAQ:CAPR)
Capricor Therapeutics, Inc is a clinical-stage biotechnology company focused on the development of cell and exosome-based therapeutics for cardiovascular and rare diseases. Headquartered in Beverly Hills, California, the company leverages proprietary cardiosphere-derived cell (CDC) technology to address conditions characterized by inflammation, fibrosis, and tissue degeneration. Since its founding, Capricor has advanced its lead candidate through multiple clinical trials and has built a pipeline that spans both cell therapy and extracellular vesicle (exosome) platforms.
The company’s leading product candidate, CAP-1002, comprises allogeneic CDCs and is being evaluated in indications such as Duchenne muscular dystrophy (DMD) and COVID-19-related heart injury.
