Capricor Therapeutics NASDAQ: CAPR executives said the company is in active discussions with the U.S. Food and Drug Administration as it works toward a potential approval decision for deramiocel, its allogeneic cardiac-derived cell therapy for Duchenne muscular dystrophy.
Speaking at a Goldman Sachs biotech event, Chief Executive Officer Linda Marbán said Capricor’s regulatory process over the past year had been “tumultuous,” citing a canceled advisory committee meeting, a complete response letter and subsequent discussions with the FDA. Marbán said the company met with the agency in a Type A meeting and confirmed that data from its HOPE-3 Phase 3 pivotal trial would be sufficient for review.
The FDA, according to Marbán, declined to accept left ventricular ejection fraction as the primary efficacy endpoint and asked the company to retain Performance of the Upper Limb 2.0 as the primary endpoint. Capricor has since submitted the HOPE-3 data to reopen its biologics license application. The company’s PDUFA date is Aug. 22, 2026.
“We’re very much in active conversations with the FDA now regarding information requests, regarding clinical opportunities, confirming all of the CMC data which we had previously presented, and all going very smoothly,” Marbán said. She added that labeling discussions are expected to move into “a more serious mode” in the coming weeks.
HOPE-3 Data and Safety Profile
Marbán said Capricor has conducted five clinical trials showing attenuation of skeletal muscle dysfunction as measured by Performance of the Upper Limb assessments. In HOPE-3, the company used Performance of the Upper Limb 2.0 as the primary efficacy endpoint and reported statistically significant results, including an absolute 1.2-point change. Marbán said the FDA had previously stated that a 1-point change would be considered clinically meaningful.
She said the trial also met its key secondary endpoint of ejection fraction, positioning deramiocel as a therapy that has shown effects in both skeletal and cardiac muscle function in Duchenne muscular dystrophy. Three-year data from the HOPE-2 open-label extension and HOPE-3 data are under review for publication, and Capricor plans to present five-year HOPE-2 open-label extension data at the Parent Project Muscular Dystrophy meeting.
Marbán described deramiocel as a quarterly infusion of 150 million cells that can be delivered through a butterfly needle over about an hour. She said most patients tolerate the therapy well, though early development included cases of anaphylaxis and some patients still experience hypersensitivity that is managed with steroids, antihistamines and sometimes acetaminophen.
Company Plans Independent Launch Amid NS Pharma Litigation
Marbán also addressed Capricor’s recently filed legal action against NS Pharma. She said the original agreement included a transfer price structure that Capricor later concluded would not work from a reimbursement and pricing standpoint because it could set the average sale price below a level the company considered appropriate for deramiocel.
According to Marbán, both Capricor and NS Pharma acknowledged the structure was problematic, but negotiations over a new arrangement did not produce an agreement. She said NS Pharma supported a private-label distributor model, which Capricor opposed because it would “turn them into a contract manufacturer.”
Marbán said Capricor now plans to launch deramiocel independently of Nippon Shinyaku or NS Pharma. She said she does not expect the lawsuit to affect regulatory timing, noting that the FDA focuses on safety and efficacy rather than sales, marketing or distribution issues.
Commercial Preparation and Pricing
Capricor recently hired Mike Moyer as chief commercial officer. Marbán said Moyer has rare disease and Duchenne muscular dystrophy experience, including at Sarepta during the launch of ELEVIDYS. She said the company is building its commercial organization around market access, patient services and reimbursement strategies.
Marbán said more than 100 patients are currently on open-label extension therapy and could potentially transition to commercial product after launch. She also said payers are interested in deramiocel because of its cardiac data in Duchenne muscular dystrophy.
In response to a pricing question, an executive identified in the transcript as Operator said Capricor is targeting a price “at or above” approved exon-skipping therapies. The executive noted deramiocel would be a chronic therapy administered four times per year.
Marbán said Capricor has focused first on U.S. approval because its trials were conducted in the United States and the company is U.S.-based. She said Capricor is also engaging with the European Medicines Agency and believes the HOPE-3 trial may be sufficient for EMA approval, though it would likely seek a European partner.
Manufacturing, Pipeline and Cash Position
Capricor has maintained manufacturing as a core capability for deramiocel, Marbán said. The company has built a small commercial manufacturing facility at its Torrey Pines location that she said can support an initial launch for about 200 to 250 patients annually. Additional clean rooms are expected to come online sequentially in 2027, with capacity potentially reaching about 2,000 to 2,500 patients by the end of that year.
Beyond Duchenne muscular dystrophy, Marbán said Capricor is evaluating deramiocel in other diseases involving inflammation and fibrosis with skeletal and cardiac implications, including Becker muscular dystrophy, limb-girdle muscular dystrophy, FSHD and rare cardiomyopathies.
She also discussed Capricor’s StealthX exosome platform. Marbán said the company discovered that exosomes mediate the benefit of deramiocel and is evaluating exosome-based technologies for biodistribution, targeting and potential therapeutic indications. She said an exosome-based vaccine program has been funded and operated by the National Institute of Allergy and Infectious Diseases.
On funding, an executive identified in the transcript as Operator said Capricor had $278 million in cash at the end of the first quarter. The executive said the company is investing in CMC expansion, commercial development and its pipeline. At approval, Capricor expects to be eligible for a priority review voucher, which the executive said the company would look to sell and monetize.
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.
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