Capricor Therapeutics Publishes Peer-Reviewed Study in Biomedicines Describing the Mechanism of Action and Potency Assay for its Investigational Cell Therapy, Deramiocel

Rhea-AI Summary

Capricor Therapeutics (NASDAQ: CAPR) published a peer-reviewed Biomedicines study describing a novel in‑vitro potency assay for its lead cell therapy, Deramiocel.

The paper reports that cardiosphere‑derived cells (CDCs) and their exosomes suppressed collagen I and III gene expression in primary human fibroblasts, supporting an anti‑fibrotic mechanism. Results were consistent across more than 100 Deramiocel manufacturing lots. The assay was validated to assess lot‑to‑lot bioactivity and potency under quality‑controlled manufacturing conditions.

Deramiocel is in late‑stage development for Duchenne muscular dystrophy; topline Phase 3 HOPE‑3 data are expected in the mid‑fourth quarter of 2025, and Capricor plans to resubmit its BLA after responding to a CRL.

Positive

• Assay validated across more than 100 manufacturing lots
• Anti‑fibrotic mechanism shown via collagen I/III suppression in human fibroblasts
• Assay supports lot‑to‑lot bioactivity and potency under quality control

Negative

• Topline Phase 3 HOPE‑3 data pending mid‑fourth quarter 2025
• BLA resubmission required following a previously issued CRL

Insights

Translational cell therapy expert

Peer-reviewed validation of Deramiocel's anti-fibrotic potency and a lot-level assay strengthens development and regulatory positioning ahead of Phase 3 topline data.

Deramiocel shows a plausible anti-fibrotic mechanism: cardiosphere-derived cells (CDCs) suppressed collagen I and collagen III gene expression in primary human fibroblasts via secreted exosomes and soluble factors. The reported consistency across more than one hundred manufacturing lots and alignment with in vivo pharmacodynamic signals and over four years of clinical observations supply concrete, measurable evidence of biological activity.

The validated in vitro potency assay directly addresses a core regulatory and manufacturing need: demonstrating reproducible lot-to-lot bioactivity under quality-controlled conditions. This reduces a key uncertainty regulators review in late-stage cell therapy programs and supports comparability claims when scaling or making process changes. Remaining dependencies include the upcoming Phase 3 HOPE-3 topline readout expected in mid-fourth quarter of 2025 and the planned BLA resubmission after management reviews the CRL response.

Watch for the HOPE-3 topline results and any regulator commentary tied to the assay when the company submits its BLA response; those items form the near-term milestones that will most affect regulatory decisions and commercial-readiness within the next several months.

–Publication highlights Deramiocel’s anti-fibrotic activity and describes a validated potency assay supporting quality control and product consistency in late-stage development– 

SAN DIEGO, Nov. 03, 2025 (GLOBE NEWSWIRE) -- Capricor Therapeutics, Inc. (NASDAQ: CAPR), a biotechnology company developing cell and exosome-based therapies for rare diseases, today announced the publication in Biomedicines, an international peer-reviewed journal, describing a novel in-vitro potency assay designed to characterize the anti-fibrotic mechanism of action of the Capricor’s lead investigational product, Deramiocel, which is currently in late-stage development for the treatment of Duchenne muscular dystrophy (DMD).

The publication, titled “A Novel In Vitro Potency Assay Demonstrating the Anti-Fibrotic Mechanism of Action of CDCs in Deramiocel,” showed results that further elucidate the biological activity of cardiosphere-derived cells (CDCs), the active component of Deramiocel. The study found that CDCs, through their secreted exosomes and soluble factors, suppressed collagen I and III gene expression in primary human fibroblasts in vitro, supporting their potential to modulate fibrotic processes associated with disease progression.

Findings were consistent across more than one hundred Deramiocel manufacturing lots, demonstrating the anti-fibrotic mechanism of action, which aligns with in vivo pharmacodynamic studies and more than four years of clinical data demonstrating stabilization of cardiac function in DMD patients. The assay was developed and validated as a tool to help assess Deramiocel’s lot-to-lot bioactivity and potency under quality-controlled manufacturing conditions, enhancing Capricor’s ability to ensure product consistency and quality as development progresses.

“This publication contributes to the growing body of evidence describing how cardiosphere-derived cells may modulate fibrotic and inflammatory pathways that underlie the progression of Duchenne muscular dystrophy,” said Linda Marbán, Ph.D., Chief Executive Officer of Capricor. “By establishing a reproducible and quantitative assay, we have strengthened our understanding of Deramiocel’s biological profile and enhanced our ability to ensure product consistency as our Duchenne program advances.”

Deramiocel is currently being evaluated in the Phase 3 HOPE-3 trial for the treatment of DMD with topline data expected to be available in the mid-fourth quarter of 2025. Following receipt of these data, Capricor expects to resubmit its Biologics License Application (BLA) with its response to the CRL.

To complement this publication, Capricor has released a short educational video describing the mechanism of action of Deramiocel, available here.

About Deramiocel

Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs), a rare population of cardiac cells that have been shown in preclinical and clinical studies to exert potent immunomodulatory and anti-fibrotic actions in the preservation of cardiac and skeletal muscle function in muscular dystrophies such as DMD. CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile to adopt a healing rather than pro-inflammatory phenotype. CDCs have been investigated in more than 250 peer-reviewed scientific publications and administered to over 250 human subjects across multiple clinical trials.

Deramiocel has received Orphan Drug Designation for the treatment of Duchenne Muscular Dystrophy (DMD) from both the U.S. FDA and the European Medicines Agency (EMA). In addition, it has been granted Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S., Advanced Therapy Medicinal Product (ATMP) designation in Europe, and Rare Pediatric Disease Designation from the FDA, which may qualify Capricor for a Priority Review Voucher upon approval.

About the HOPE-3 Phase 3 Trial

HOPE-3 is a Phase 3, multi-center, randomized, double-blind, placebo-controlled clinical trial consisting of two cohorts evaluating the safety and efficacy of Deramiocel in participants with DMD. Non-ambulatory and ambulatory boys who meet eligibility criteria are randomly assigned to receive either Deramiocel or placebo every 3 months for a total of four doses during the first 12 months of the trial. A total of 105 eligible subjects have been enrolled in the dual-cohort trial. For more information, please visit ClinicalTrials.gov (NCT05126758).

About Capricor Therapeutics

Capricor Therapeutics (NASDAQ: CAPR) is a biotechnology company dedicated to advancing transformative cell and exosome-based therapeutics to redefine the treatment landscape for rare diseases. At the forefront of our innovation is our lead product candidate, Deramiocel, an allogeneic cardiac-derived cell therapy that is currently in late-stage clinical development for the treatment of Duchenne muscular dystrophy (DMD). Extensive preclinical and clinical data have demonstrated Deramiocel’s potent immunomodulatory and anti-fibrotic effects in helping to preserve cardiac and skeletal muscle function in DMD. Capricor is also leveraging the power of its exosome technology, using its proprietary StealthX™ platform in preclinical development focused on vaccinology and the targeted delivery of oligonucleotides, proteins, and small-molecule therapeutics, with the potential to treat and prevent a wide range of diseases. At Capricor, we are committed to pushing the boundaries of possibility and forging a path toward transformative treatments for those in need. For more information, visit capricor.com, and follow Capricor on Facebook, Instagram and X.

Cautionary Note Regarding Forward-Looking Statements

Statements in this press release regarding the efficacy, safety, and intended utilization of Capricor’s product candidates; the initiation, conduct, size, timing and results of clinical trials; the pace of enrollment of clinical trials; plans regarding regulatory filings, future research and clinical trials; regulatory developments involving products, including future interactions with regulatory authorities and the ability to obtain regulatory approvals or otherwise bring products to market; manufacturing capabilities; dates for regulatory meetings; the potential that required regulatory inspections may be delayed or not be successful which would delay or prevent product approval; the ability to achieve product milestones and to receive milestone payments from commercial partners; and any other statements about Capricor’s management team’s future expectations, beliefs, goals, plans or prospects constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact (including statements containing the words “believes,” “plans,” “could,” “anticipates,” “expects,” “estimates,” “should,” “target,” “will,” “would” and similar expressions) should also be considered to be forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward-looking statements. More information about these and other risks that may impact Capricor’s business is set forth in Capricor’s Annual Report on Form 10-K for the year ended December 31, 2024, as filed with the Securities and Exchange Commission on March 26, 2025, and in our Quarterly Report on Form 10-Q for the quarter ended June 30, 2025, as filed with the Securities and Exchange Commission on August 11, 2025. All forward-looking statements in this press release are based on information available to Capricor as of the date hereof, and Capricor assumes no obligation to update these forward-looking statements.

Capricor has entered into an agreement for the exclusive commercialization and distribution of Deramiocel for DMD in the United States and Japan with Nippon Shinyaku Co., Ltd. (U.S. subsidiary: NS Pharma, Inc.), subject to regulatory approval. Deramiocel and the StealthX™ vaccine are investigational candidates and have not been approved for commercial use in any indication.

For more information, please contact:

Capricor Media Contact:
Raquel Cona
KCSA Strategic Communications
rcona@kcsa.com
212.896.1204

Capricor Company Contact:
AJ Bergmann, Chief Financial Officer
abergmann@capricor.com
858.727.1755

FAQ

What did Capricor announce about Deramiocel on November 3, 2025 (CAPR)?

Capricor published a Biomedicines study describing a validated in‑vitro potency assay and an anti‑fibrotic mechanism for Deramiocel.

How many Deramiocel manufacturing lots showed consistent assay results in the study?

The study reported consistent results across more than 100 manufacturing lots.

What mechanism of action did the Deramiocel study demonstrate for CAPR's CDCs?

CDCs and their exosomes suppressed collagen I and III gene expression in primary human fibroblasts, indicating anti‑fibrotic activity.

When does Capricor expect topline Phase 3 HOPE‑3 results for Deramiocel (CAPR)?

Topline HOPE‑3 data are expected in the mid‑fourth quarter of 2025.

Does the validated potency assay affect Deramiocel manufacturing quality for CAPR?

Yes — the assay was validated to assess lot‑to‑lot bioactivity and help ensure product consistency under quality control.

What regulatory step will Capricor take after receiving HOPE‑3 topline data (CAPR)?

Capricor expects to resubmit its BLA with a response to the existing CRL after topline results are received.