Capricor Therapeutics Announces Late-Breaking Presentation at 2026 MDA Clinical and Scientific Conference
Rhea-AI Summary
Capricor Therapeutics (NASDAQ: CAPR) announced that Phase 3 HOPE-3 results for Deramiocel in Duchenne muscular dystrophy were selected for a late-breaking oral presentation at the 2026 MDA Clinical and Scientific Conference on March 11, 2026 at 2:45 p.m. ET.
The company submitted the HOPE-3 clinical study report to the U.S. FDA to address items in a prior Complete Response Letter and to support the ongoing Biologics License Application review, including possible 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
- Phase 3 HOPE-3 selected as a late-breaking MDA presentation
- HOPE-3 clinical study report submitted to the U.S. FDA
- HOPE-3 randomized 106 subjects in dual-cohort trial
- Deramiocel holds Orphan, RMAT, ATMP and Rare Pediatric Disease designations
Negative
- FDA issued a prior Complete Response Letter that prompted CSR submission
- Deramiocel remains investigational; BLA under review with no approval yet
Key Figures
Market Reality Check
Peers on Argus
CAPR was at $27.87, down 2.24% pre-news. Peers were mixed: CADL (-1.58%), OMER (-4.35%), LCTX (-1.67%) were weaker, while ALEC (+5.81%) and FHTX (+0.35%) gained, suggesting stock-specific factors rather than a uniform biotech move.
Historical Context
| Date | Event | Sentiment | Move | Catalyst |
|---|---|---|---|---|
| Jan 20 | Regulatory CSR update | Positive | -0.6% | FDA requested full HOPE-3 CSR to continue BLA review and new PDUFA date. |
| Dec 16 | Clinical data webinar | Positive | +0.5% | Webinar to review positive Phase 3 HOPE-3 topline results with DMD community. |
| Dec 16 | Offering priced | Negative | +5.4% | Priced $150M common stock offering at $25 per share with underwriter option. |
| Dec 04 | Offering proposed | Negative | -14.9% | Announced proposed underwritten offering of common stock under S-3 shelf. |
| Dec 03 | Phase 3 topline data | Positive | +371.1% | HOPE-3 met primary and key secondary endpoints with significant efficacy and safety. |
Recent news shows mostly aligned reactions: major positive HOPE-3 data and one offering aligned with large moves, while a follow-on offering and a regulatory CSR update saw mild or opposite responses.
Over the last few months, CAPR has moved through key HOPE-3 and financing milestones. On Dec 3, 2025, positive Phase 3 HOPE-3 topline results with statistically significant musculoskeletal and cardiac endpoints drove a 371.07% move. Subsequent financing activity in early December, including a proposed and then priced equity offering, produced a -14.87% decline followed by a 5.43% gain. Later, a December webinar reiterated positive HOPE-3 data, and a Jan 20, 2026 regulatory update on the FDA-requested HOPE-3 CSR corresponded to a modest -0.62% move.
Regulatory & Risk Context
An effective S-3 shelf filed on 2025-09-10 authorizes up to $300,000,000 of securities and, separately, up to $150,000,000 of common stock via an equity distribution agreement. As of the provided data, reported usage count is 0, so the disclosed shelf capacity remains available for potential future financings.
Market Pulse Summary
This announcement highlights increased scientific and regulatory visibility for Deramiocel, with Phase 3 HOPE-3 data selected as a late-breaking presentation and the clinical study report submitted to address the FDA’s CRL within the ongoing BLA review. Investors may watch for any new PDUFA target action date and details emerging from the March 11, 2026 presentation. The multiple designations (Orphan, RMAT, ATMP, Rare Pediatric Disease) and prior pivotal HOPE-3 results frame this as a key step in the approval pathway.
Key Terms
biologics license application (bla) regulatory
complete response letter (crl) regulatory
prescription drug user fee act (pdufa) regulatory
orphan drug designation regulatory
regenerative medicine advanced therapy (rmat) regulatory
advanced therapy medicinal product (atmp) regulatory
rare pediatric disease designation regulatory
AI-generated analysis. Not financial advice.
- Late-breaking presentation at MDA to feature Phase 3 HOPE-3 results supporting Deramiocel in Duchenne muscular dystrophy
- HOPE-3 clinical study report (CSR) submitted to the U.S. Food and Drug Administration (FDA) in support of the ongoing BLA review
SAN DIEGO, Feb. 24, 2026 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced that results from its Phase 3 HOPE-3 clinical study of Deramiocel in Duchenne muscular dystrophy (DMD) have been selected for a late-breaking oral presentation at the 2026 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, taking place March 8–11, 2026, in Orlando, Florida.
“The selection of HOPE-3 as a late-breaking presentation at the MDA Conference recognizes the strength and growing body of clinical evidence supporting Deramiocel and its potential impact for patients living with Duchenne,” said Linda Marbán, Ph.D., Chief Executive Officer of Capricor. “We look forward to sharing these Phase 3 results with the DMD community while continuing to advance our regulatory efforts, including the recent submission of the HOPE-3 clinical study report to the FDA as part of our ongoing BLA review process. We remain focused on working toward a potential approval decision and on bringing this therapy to patients as efficiently as possible.”
MDA Presentation Details
| Date and Time: | March 11, 2026, 2:45 p.m. ET |
| Presentation Title: | Confirmation of Musculoskeletal and Cardiac Benefit in DMD from Deramiocel, an Allogeneic Cell Therapy, in the Phase 3 HOPE-3 Study |
| Presenter: | Craig McDonald, M.D. (Professor of Physical Medicine & Rehabilitation and Pediatrics at UC Davis Health and National Principal Investigator of the HOPE-3 trial) |
| Location: | Florida 4 |
The CSR submission was requested by the FDA following prior regulatory interactions and is intended to address items outlined in the Complete Response Letter (CRL) and support the ongoing review of the Company’s Biologics License Application (BLA) for Deramiocel in Duchenne muscular dystrophy, including the potential assignment by the FDA of a new Prescription Drug User Fee Act (PDUFA) target action date.
For more details on the MDA conference, click here. To view the conference agenda, click here.
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 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 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 Deramiocel at the MDA conference on March 11, 2026?
Has Capricor (CAPR) submitted HOPE-3 data to the FDA and why does it matter?
How many patients were randomized in the HOPE-3 Phase 3 trial reported by Capricor (CAPR)?
What regulatory designations does Deramiocel have, per Capricor (CAPR)?
Does the HOPE-3 announcement mean Deramiocel is approved for Duchenne (CAPR)?
