Capricor Therapeutics Announces Late-Breaking Presentation at 2026 MDA Clinical and Scientific Conference
MWN-AI** Summary
Capricor Therapeutics, a biotech firm focused on innovative therapies for rare diseases, has announced that results from its Phase 3 HOPE-3 clinical trial of Deramiocel for treating Duchenne muscular dystrophy (DMD) will be presented as a late-breaking oral session at the 2026 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference. The event is scheduled for March 8–11, 2026, in Orlando, Florida.
Linda Marbán, CEO of Capricor, highlighted the significance of the HOPE-3 trial being selected for late-breaking presentation, emphasizing the growing clinical evidence supporting Deramiocel’s potential to benefit patients with DMD. The company has recently submitted a clinical study report to the U.S. Food and Drug Administration (FDA) to support its ongoing Biologics License Application (BLA) review, which is pivotal in their efforts to bring Deramiocel to market.
The presentation, titled "Confirmation of Musculoskeletal and Cardiac Benefit in DMD from Deramiocel," will be led by Professor Craig McDonald, the trial's National Principal Investigator. The HOPE-3 trial involved a total of 106 non-ambulatory and ambulatory boys with DMD, receiving either Deramiocel or a placebo.
Duchenne muscular dystrophy is a severe genetic disorder characterized by progressive muscle degeneration, predominantly affecting boys, and lacks effective treatment options. Deramiocel is formulated from allogeneic cardiosphere-derived cells (CDCs), which have shown promise in enhancing muscle function through immunomodulatory and anti-fibrotic effects.
Capricor's innovative approach and regulatory designations, including Orphan Drug and Regenerative Medicine Advanced Therapy status, reinforce the organization’s commitment to pioneering treatments for DMD. The upcoming conference presentation represents an important step toward advancing the therapeutic landscape for this debilitating condition.
MWN-AI** Analysis
Capricor Therapeutics (NASDAQ: CAPR) is poised for significant momentum following the selection of its Phase 3 HOPE-3 study results on Deramiocel for Duchenne muscular dystrophy (DMD) for a late-breaking presentation at the upcoming MDA Clinical and Scientific Conference in March 2026. This recognition underscores the strength of the clinical evidence supporting Deramiocel, a cell-based therapy currently under review by the FDA.
Investors should view this announcement as a crucial inflection point. The late-breaking presentation not only reflects a potential breakthrough in therapy for a rare, life-threatening condition but also elevates Capricor's visibility within the biotech community and among potential investors and partners.
With the clinical study report already submitted to the FDA as part of the Biologics License Application (BLA) review process, any positive data shared at the MDA conference could support a favorable regulatory decision. Given that DMD currently lacks effective treatment options, Deramiocel's path toward approval could align with significant unmet medical needs, further enhancing its market potential.
Moreover, potential regulatory designations such as Orphan Drug Designation and Regenerative Medicine Advanced Therapy (RMAT) status position Capricor favorably for expedited review processes and market exclusivity benefits. Investors should monitor developments closely as these designations can significantly impact sentiment and stock performance.
However, caution is advised. The biotech sector can be volatile, particularly regarding clinical trial outcomes and regulatory approvals. Potential investors should remain attuned to upcoming presentations and announcements that will provide critical insights into Deramiocel's efficacy and safety profile.
In summary, Capricor has an opportunity to capitalize on this presentation, potentially fueling stock growth. Aligning optimistic expectations with strategic risk management could create value for investors looking at Capricor in the upcoming months.
**MWN-AI Summary and Analysis is based on asking OpenAI to summarize and analyze this news release.
- 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**
How do the Phase 3 HOPE-3 results for Deramiocel support the prospects of Capricor Therapeutics Inc. (CAPR) in the DMD market, and what specific musculoskeletal and cardiac benefits were observed in the study?
What are the key regulatory milestones that Capricor Therapeutics Inc. (CAPR) aims to achieve following the submission of the HOPE-3 clinical study report to the FDA?
Considering the potential PDUFA target action date, how does Capricor Therapeutics Inc. (CAPR) plan to expedite the approval process for Deramiocel, and what implications could this have for patients with DMD?
Given the unique therapeutic approach of allogeneic cell therapy, how does Capricor Therapeutics Inc. (CAPR) differentiate Deramiocel from existing treatments for Duchenne muscular dystrophy, and what future studies are planned?
**MWN-AI FAQ is based on asking OpenAI questions about Capricor Therapeutics Inc. (NASDAQ: CAPR).
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