Capricor Therapeutics Q4 Earnings Call Highlights
by Tristan Rich · The Markets DailyCapricor Therapeutics (NASDAQ:CAPR) executives used the company’s fourth-quarter and full-year 2025 earnings call to outline progress toward a potential U.S. approval of deramiocel for Duchenne muscular dystrophy (DMD), highlight additional HOPE-3 clinical data presented at a medical meeting, and review a strengthened cash position following financings completed in late 2025.
FDA accepts BLA resubmission; PDUFA date set for August 22, 2026
Chief Executive Officer Linda Marbán said the U.S. Food and Drug Administration has informed the company that its response to a prior complete response letter (CRL) is complete and that the agency has accepted Capricor’s previously submitted Biologics License Application (BLA) for review. The FDA assigned a Prescription Drug User Fee Act (PDUFA) target action date of August 22, 2026.
Marbán described the development as a “significant regulatory milestone” and said the BLA seeks full approval for deramiocel. She noted that detailed label discussions have not yet occurred, but said Capricor’s internal goal is to position the therapy to treat as many eligible patients as possible, consistent with clinical data the company says show stabilization in both skeletal and cardiac muscle function.
On the call, Marbán also said the FDA classified the filing as a Class 2 resubmission and that, at this stage, the agency has not identified potential review issues in its communication to the company—an update Capricor characterized as encouraging.
HOPE-3 results highlighted; new data presented at MDA meeting
Marbán said the full HOPE-3 dataset—submitted to the FDA as part of Capricor’s CRL response and included in the clinical study report—will serve as the foundation for potential approval and commercial launch planning.
Capricor described HOPE-3 as a pivotal Phase 3, multi-center, randomized, double-blind, placebo-controlled study evaluating deramiocel in DMD cardiomyopathy. According to management, the study enrolled 106 patients and met its primary endpoint on the Performance of the Upper Limb (PUL), along with all Type I error-controlled secondary endpoints. The company also emphasized left ventricular ejection fraction (LVEF) as a key secondary endpoint, stating HOPE-3 showed a “91% slowing of disease progression” in evaluable patients regardless of cardiac disease status, with statistical significance. Management added that results were stronger in patients with a diagnosis of cardiomyopathy, citing a P-value of 0.01.
In addition to the previously reported top-line data, Marbán said Capricor presented additional HOPE-3 findings in a late-breaking oral presentation at the 2026 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference. Highlights discussed on the call included:
- Duchenne Video Assessment (DVA) results in a home-based setting using the “Eat Ten Bites” task, which Marbán said showed a statistically significant improvement. She described the DVA as a validated and published patient-reported outcome approach that can complement clinic-based measures and support payer discussions.
- Cardiac fibrosis imaging comparing treated versus placebo patients, assessed via MRI with gadolinium, which management said showed a significant reduction in fibrosis in deramiocel-treated patients.
During Q&A, Marbán said the DVA dataset included about 50 patients in each group (deramiocel and placebo). She also explained that late gadolinium enhancement (LGE) fibrosis measurement was added for cohort B only, citing earlier concerns during cohort A design about gadolinium safety in young children; she said that view later changed and that cohort B participants also needed certain kidney function levels, contributing to a smaller sample size for the endpoint.
Capricor said the full HOPE-3 dataset has been submitted for publication in a major peer-reviewed academic journal, though management did not provide a publication timeline.
Safety profile, cohort questions, and label expectations
Marbán emphasized deramiocel’s safety, stating that Capricor has completed more than 800 intravenous infusions across multiple clinical studies and continues to see a consistent safety profile. She added that some participants in the HOPE-2 open-label extension (OLE) have received continuous infusions for up to five years and that there are over 100 patients across Capricor’s open-label extension studies.
Analysts asked about two cohorts within HOPE-3 and manufacturing comparability. Marbán said the FDA has not raised cohort questions since around 2024, when Capricor filed a cardiomyopathy-focused BLA based on HOPE-2, HOPE-2 OLE, and natural history data. She said FDA agreed cohort A and cohort B could be considered as one trial because of non-clinical comparability, and she added that cohort B alone was statistically significant on both PUL and ejection fraction.
On labeling, Marbán said Capricor did not reach detailed label discussions before the CRL was issued. She said the company believes the data support labeling that addresses both skeletal muscle aspects of DMD and cardiomyopathy, but acknowledged the FDA will ultimately determine the label. She also said she does not expect a conditional approval, arguing the HOPE-3 dataset is a randomized, double-blind, placebo-controlled trial that met primary and key secondary endpoints.
Manufacturing, commercial readiness, and potential priority review voucher
Capricor said its in-house GMP manufacturing facility in San Diego completed an FDA pre-license inspection (PLI) connected to the BLA review process last year. Management said Form 483 observations were addressed and the facility is operational for a potential initial commercial launch.
Marbán said the current facility can meet demand of approximately 250 patients per year. Capricor also plans to expand to a second floor at the same site, adding about six additional clean rooms. At full capacity, the expansion is expected to support approximately 2,500 patients per year, or roughly 10,000 doses annually, with the company projecting the expanded facility could support commercial manufacturing in late 2027.
In response to questions, Marbán said Capricor built the new clean rooms within the same building to reduce regulatory requirements and expects process performance qualification runs and another inspection. She also cited public comments by FDA leadership about potentially reducing the number of PPQ runs, though she did not say any change has been implemented.
Commercially, Marbán said Capricor is hiring and preparing across patient support, market access, reimbursement, and physician education. She also said Capricor expects open-label extension patients to transition to commercial drug after approval and is focused on market access to enable a smooth transition.
Capricor also said it expects to be eligible for a priority review voucher upon approval of deramiocel, which management said could be transferred and monetized as a potential source of non-dilutive capital, though the company did not provide estimates for timing or proceeds.
Pipeline updates and 2025 financial results
Beyond deramiocel, management discussed its StealthX engineered exosome platform and a Phase 1 COVID vaccine study under Project NextGen with the National Institute of Allergy and Infectious Diseases (NIAID). Capricor said preliminary results indicate the vaccine has been well-tolerated with a favorable safety profile across doses tested, but limited neutralization was observed at tested dose levels, which management suggested may reflect prior vaccination or infection in trial participants. Final results, including cellular response data, are expected in the second quarter of 2026, and Capricor said NIAID has requested exploration of higher doses and potential use of adjuvants.
Chief Financial Officer AJ Bergmann reported that cash, cash equivalents, and marketable securities totaled approximately $318.1 million as of December 31, 2025, up from about $151.5 million a year earlier. He said Capricor completed a public offering in December 2025 with net proceeds of approximately $162 million, and also drew about $75 million under its at-the-market program in December 2025.
Revenue was $0 for both the fourth quarter and full year 2025, compared with approximately $11.1 million in the fourth quarter of 2024 and $22.3 million for full-year 2024. Bergmann said prior-year revenue primarily reflected ratable recognition of upfront and developmental milestone payments under a U.S. distribution agreement with Nippon Shinyaku, which was fully recognized as of December 31, 2024.
Total operating expenses rose to approximately $29.2 million in the fourth quarter of 2025 (from $18.8 million a year earlier) and about $108.1 million for full-year 2025 (from $64.8 million in 2024), driven by investment in clinical, regulatory, manufacturing, and infrastructure activities for the Duchenne program. Net loss was approximately $30.2 million in the fourth quarter and $105 million for the year, compared with net losses of $7.1 million and $40.5 million, respectively, in 2024.
Both Marbán and Bergmann said Capricor believes its current capital is sufficient to fund operations into the fourth quarter of 2027, excluding potential additional sources such as product revenue, milestone payments, or priority review voucher monetization.
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.