Capricor Therapeutics Provides Regulatory Update on Deramiocel BLA Following FDA Review of HOPE-3 Topline Data
Rhea-AI Summary
Capricor (NASDAQ: CAPR) said the FDA has requested the full HOPE-3 clinical study report (CSR) as part of its Biologics License Application (BLA) review for Deramiocel and did not ask for additional clinical studies or new patient data. The company is preparing the HOPE-3 CSR and plans to submit the requested materials in February 2026 to address the Complete Response Letter (CRL) received in July 2025, with the goal of supporting continued review and assignment of a new PDUFA target action date.
HOPE-3 randomized 106 subjects across ambulatory and non-ambulatory cohorts; Deramiocel holds multiple regulatory designations including Orphan, RMAT, ATMP, and Rare Pediatric Disease.
Positive
- Plan to submit HOPE-3 CSR to FDA in February 2026
- FDA did not request additional clinical studies or new patient data
- HOPE-3 randomized 106 subjects across two cohorts
- Deramiocel holds Orphan, RMAT, ATMP, and Rare Pediatric Disease designations
Negative
- Company received a Complete Response Letter (CRL) in July 2025
- BLA remains under review pending CSR submission and a new PDUFA date
News Market Reaction
On the day this news was published, CAPR declined 0.62%, reflecting a mild negative market reaction. Argus tracked a peak move of +35.8% during that session. Argus tracked a trough of -8.7% from its starting point during tracking. Our momentum scanner triggered 3 alerts that day, indicating moderate trading interest and price volatility. This price movement removed approximately $8M from the company's valuation, bringing the market cap to $1.26B at that time.
Data tracked by StockTitan Argus on the day of publication.
Key Figures
Market Reality Check
Peers on Argus
CAPR gained 1.29% pre-news while close peers were mixed: CADL +7.56%, ALEC +2.22%, LCTX +2.41%, OMER -1.4%, FHTX -3.99%, supporting a stock-specific narrative for this regulatory update.
Historical Context
| Date | Event | Sentiment | Move | Catalyst |
|---|---|---|---|---|
| Dec 16 | Webinar on HOPE-3 | Positive | +0.5% | Community webinar to review positive Phase 3 HOPE-3 topline results. |
| Dec 05 | Offering priced | Negative | +5.4% | Pricing of $150M common stock offering under existing shelf registration. |
| Dec 04 | Offering proposed | Negative | -14.9% | Announcement of proposed underwritten offering of common stock. |
| Dec 03 | HOPE-3 topline data | Positive | +371.1% | Pivotal Phase 3 HOPE-3 met primary and key secondary endpoints in DMD. |
| Nov 24 | Exosome platform data | Positive | -19.3% | New preclinical data on scalable exosome loading framework at AAEV 2025. |
Clinical trial and regulatory milestones have often triggered strong reactions, with major positive HOPE-3 data driving the largest move, while scientific platform updates have occasionally seen negative or muted responses.
Over the last six months, CAPR has progressed Deramiocel through key regulatory and clinical milestones. A June 17, 2025 update highlighted Orphan Drug Designation for Becker muscular dystrophy and ongoing DMD regulatory progress. The Dec 3, 2025 HOPE-3 topline release produced a large positive price reaction, followed by a smaller move around the Dec 16, 2025 webinar. December also included a proposed and then priced equity offering. Today’s regulatory update fits into this ongoing effort to resolve the July 2025 CRL using HOPE-3 data.
Regulatory & Risk Context
An effective S-3 shelf filed on Sep 10, 2025 authorizes up to $300,000,000 of securities, plus up to $150,000,000 of common stock under an equity distribution agreement, enabling flexible future capital raises that could be dilutive if utilized.
Market Pulse Summary
This announcement details the FDA’s request for the full HOPE-3 clinical study report and Capricor’s plan to update the Deramiocel BLA in February 2026, aiming to address the July 2025 CRL. It follows prior positive HOPE-3 topline data and multiple regulatory designations. Investors may watch for the completeness of the CSR submission, any new PDUFA date assignment, and how future use of the up to $300,000,000 shelf and related $150,000,000 equity program affects funding and dilution.
Key Terms
biologics license application regulatory
complete response letter regulatory
clinical study report regulatory
prescription drug user fee act regulatory
regenerative medicine advanced therapy regulatory
advanced therapy medicinal product regulatory
rare pediatric disease designation regulatory
extracellular vesicles medical
AI-generated analysis. Not financial advice.
- FDA has requested the HOPE-3 clinical study report (CSR) as part of the BLA review process
- Company expects to submit updates to the BLA in February 2026 to support continued FDA review
SAN DIEGO, Jan. 20, 2026 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today provided a regulatory update regarding its Biologics License Application (BLA) for Deramiocel, the Company’s investigational first-in-class cell therapy for the treatment of Duchenne muscular dystrophy (DMD).
As previously disclosed, the Company provided topline results from its Phase 3 HOPE-3 clinical study to the U.S. Food and Drug Administration (FDA) in late 2025. Following its review of these data, the FDA has formally requested the full HOPE-3 clinical study report (CSR) and supporting data to address the Complete Response Letter (CRL). The FDA did not request any additional clinical studies or new patient data as part of this request.
Preparation of the HOPE-3 CSR is well underway, and the Company plans to submit the requested materials to the FDA in February 2026. The Company expects that this submission will address the items outlined in the CRL and support continued review of the BLA, including the assignment of a new Prescription Drug User Fee Act (PDUFA) target action date.
“We are actively engaging with the FDA in order to facilitate an efficient review of the HOPE-3 data that directly address the issues raised in the CRL we received in July 2025. We were pleased that the FDA requested the HOPE-3 clinical study report, as this is an expected and appropriate next step following their initial review of the topline data,” said Linda Marbán, Ph.D., Chief Executive Officer of Capricor. “The HOPE-3 results demonstrated statistically significant and clinically meaningful improvements in both skeletal muscle and cardiac function—key drivers of disease progression and long-term outcomes in Duchenne. These findings build on more than a decade of consistent clinical evidence and reinforce our confidence in Deramiocel’s potential. Our near-term priority is to address the FDA’s request and continue working collaboratively so that patients with late-stage DMD, who currently have very limited treatment options, may gain access to Deramiocel as soon as possible.”
About Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD) is a severe, X-linked genetic disorder characterized by progressive muscle degeneration affecting the skeletal, respiratory, and cardiac muscles. It is caused by the absence of functional dystrophin, a key structural protein in muscle cells. DMD affects approximately 15,000 individuals in the United States and primarily impacts boys. Over time, deterioration of the heart muscle leads to cardiomyopathy and heart failure, which is the leading cause of death in DMD. There is no cure, and treatment options remain limited.
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 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, multicenter, 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 were 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 106 eligible subjects were randomized 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 September 30, 2025, as filed with the Securities and Exchange Commission on November 10, 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 (CAPR) announce about the Deramiocel BLA on January 20, 2026?
Did the FDA ask Capricor to run more clinical trials for Deramiocel (CAPR)?
How many subjects were randomized in the HOPE-3 Phase 3 trial mentioned by Capricor (CAPR)?
When does Capricor expect to submit the HOPE-3 clinical study report to the FDA for Deramiocel (CAPR)?
What regulatory designations does Deramiocel (CAPR) have that could affect approval and incentives?
