Capricor Therapeutics (NASDAQ:CAPR) said its Duchenne muscular dystrophy therapy candidate deramiocel remains under active FDA review, while the company is preparing to commercialize the product itself amid a legal dispute with distribution partner NS Pharma and parent company Nippon Shinyaku.
On the company’s first-quarter 2026 earnings call, Chief Executive Officer Linda Marbán said Capricor is at “a truly defining moment” as it advances deramiocel toward a potential approval, builds commercial infrastructure and pursues litigation seeking to unwind its U.S. distribution agreement.
The company’s biologics license application for deramiocel has a PDUFA target action date of Aug. 22, 2026. Marbán said the FDA has accepted Capricor’s response to a July 2025 complete response letter as complete and classified the resubmission as Class 2, resuming full review of the application. She said Capricor has responded to “a significant number” of FDA information requests and expects labeling discussions “in the very near future.”
Deramiocel Review Advances Toward August FDA Decision
Marbán said Capricor’s response to the complete response letter was based on results from the HOPE-3 Phase 3 trial, which she described as “one of the most compelling data sets generated for DMD to date.” HOPE-3 enrolled 106 patients in a multicenter, randomized, double-blind, placebo-controlled study.
According to Marbán, the trial met its primary endpoint, the Performance of the Upper Limb version 2.0, with an approximately 54% reduction in disease severity at 12 months and a P value of 0.029. She also said all type 1 error-controlled secondary endpoints reached statistical significance.
Because the current BLA is for cardiomyopathy, Marbán highlighted left ventricular ejection fraction as a key secondary endpoint. She said deramiocel showed an approximately 91% slowing of disease progression in all evaluable patients. In a subset of patients with a confirmed diagnosis of cardiomyopathy, she said the LVEF effect showed an approximately 120% slowing of disease and achieved a P value of 0.01.
Marbán also pointed to additional HOPE-3 data presented at medical meetings, including the Muscular Dystrophy Association Clinical and Scientific Conference, the American Academy of Neurology and the American Society of Gene & Cell Therapy. She said a home-based caregiver-captured video assessment of upper limb function showed statistically significant improvement in treated patients versus placebo, and cardiac MRI data showed significant stabilization in the progression of cardiac fibrosis.
On safety, Marbán said Capricor has completed more than 800 intravenous infusions across multiple clinical studies and that deramiocel continues to show a “consistent and well-tolerated safety profile.” She said some participants in the HOPE-2 open-label extension have received continuous infusions for up to five years, with approximately 90 patients in collective open-label extension studies.
The company also expects to be eligible for a priority review voucher upon potential approval, which Marbán said could be sold and represent a source of non-dilutive capital.
Capricor Seeks to Rescind NS Pharma Agreement
A major focus of the call was Capricor’s May 7 lawsuit against Nippon Shinyaku and its U.S. subsidiary, NS Pharma. Marbán said Capricor filed suit seeking rescission of its U.S. distribution agreement through an expedited process and requested a preliminary injunction that would allow Capricor to distribute deramiocel to patients on its own or through other distributors, pending FDA approval.
Marbán said the dispute centers on pricing. She said the pricing structure in the commercialization and distribution agreement contains “a fundamental flaw” that would make it “economically impossible” to deliver deramiocel to patients covered by Medicare, Medicaid or private insurance. She said providers could face reimbursement shortfalls “per dose” and characterized the issue as a structural barrier to patient access rather than a commercial preference.
Marbán said Capricor attempted to resolve the issue with NS Pharma, beginning discussions in March 2025, but said NS Pharma refused to address the structure on acceptable terms. She also said NS Pharma sought terms that would require Capricor to cede control of the product, regulatory relationships and commercial identity, which she called unacceptable.
“The FDA review process and the PDUFA date are unaffected by this lawsuit,” Marbán said.
In response to analyst questions, Marbán said Capricor’s current plan is to launch deramiocel itself, though timing of the litigation is dependent on the court. She said Capricor is not expecting milestone payments from NS Pharma at this time and may repay the $50 million it has already received if rescission requires it.
Commercial and Manufacturing Plans Accelerate
Marbán said Capricor has been hiring and building infrastructure to support commercialization, with a focus on patient access, physician education, payer readiness and distribution services. She said the company has a senior vice president of market access with rare disease experience and has secured a chief commercial officer expected to join in the coming weeks.
Capricor’s in-house GMP manufacturing facility in San Diego completed its FDA pre-license inspection last year, Marbán said, adding that all Form 483 observations were addressed. The facility is operational and positioned to support an initial commercial launch for approximately 200 to 250 patients per year, which she later clarified equates to about 1,000 doses annually.
The company is also expanding to a second floor at the same facility with additional clean rooms. At full capacity, Marbán said the expansion could support treatment of approximately 2,000 to 2,500 patients per year, or roughly 10,000 doses annually. She said her goal is to have the expanded facility fully validated and approved by the FDA in the first half of 2027.
Because deramiocel is an ultra-cold chain product that must be labeled before freezing, Marbán said Capricor is working with the FDA on a temporary label solution that would allow the company to begin producing commercial doses. She said Capricor’s planning indicates it will have sufficient product to support a launch if approved.
Pipeline Updates Focus on Exosomes and Broader Neuromuscular Use
Marbán said Capricor has historically focused most of its resources on deramiocel, but continues to explore exosome-based therapeutic programs. She said the company is moving away from vaccine development “at this time” and will focus on therapeutics using CDC-based exosomes and its StealthX technology.
She said Capricor’s exosome-based vaccine work with the National Institute of Allergy and Infectious Diseases moved more slowly than originally planned amid instability in vaccine development, but noted that the program achieved clinical-grade vaccine exosome manufacturing, FDA-approved CMC for large-scale manufacturing and evidence that an exosome-based vaccine is safe.
For deramiocel lifecycle management, Marbán said Capricor’s first target will likely be younger DMD patients. The company also plans to begin discussing a clinical pathway for Becker muscular dystrophy with the FDA after the DMD PDUFA date. She said Capricor expects to meet with the EMA and PMDA later this year as it explores deramiocel outside the United States.
First-Quarter Financial Results
Chief Financial Officer Anthony J. Bergmann said Capricor had approximately $279 million in cash, cash equivalents and marketable securities as of March 31, 2026. He said the company believes its current capital is sufficient to fund anticipated operating expenses and capital expenditures into the fourth quarter of 2027, excluding potential product revenue or priority review voucher monetization.
The company recognized no revenue in the first quarter of 2026 or the first quarter of 2025. Total operating expenses were approximately $36.8 million in the first quarter of 2026, compared with approximately $25 million a year earlier. Bergmann said the increase was primarily driven by investment in clinical, regulatory and manufacturing activities, along with infrastructure spending supporting the Duchenne program.
Capricor reported a first-quarter net loss of approximately $33.9 million, or $0.59 per share, compared with a net loss of approximately $24.4 million, or $0.53 per share, in the prior-year period.
Bergmann said Capricor’s expense profile reflects investment across three priority areas: regulatory and clinical activities supporting the BLA, manufacturing capacity expansion and commercial infrastructure build-out.
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.
This instant news alert was generated by narrative science technology and financial data from MarketBeat in order to provide readers with the fastest reporting and unbiased coverage. Please send any questions or comments about this story to contact@marketbeat.com.
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