Capricor Therapeutics Announces Late-Breaking Presentation of HOPE-3 Phase 3 Results at the American Academy of Neurology 2026 Annual Meeting
Rhea-AI Summary
Capricor Therapeutics (NASDAQ: CAPR) presented Phase 3 HOPE-3 results for Deramiocel in Duchenne muscular dystrophy at AAN 2026, reporting a statistically and clinically meaningful benefit on the upper-limb primary endpoint (PUL v2.0) and supportive Duchenne Video Assessment (DVA) findings.
The company has a BLA under FDA review with a PDUFA target action date of August 22, 2026 and an exclusive U.S./Japan commercialization agreement with Nippon Shinyaku.
Positive
- HOPE-3 Phase 3 showed statistically and clinically meaningful PUL v2.0 benefit
- Duchenne Video Assessment (DVA) showed slowed decline in self-feeding function
- BLA under FDA review with PDUFA target date of August 22, 2026
- Exclusive U.S. and Japan commercialization agreement with Nippon Shinyaku
Negative
- Regulatory uncertainty: approval depends on FDA review and potential inspections
- Deramiocel remains investigational and is not approved for commercial use
News Market Reaction – CAPR
In the Apr 22 session, CAPR declined 0.96%, reflecting a mild negative market reaction.
Data tracked by StockTitan Argus on the day of publication.
Key Figures
Previous Clinical trial Reports
| Date | Event | Sentiment | 24h Move | Catalyst |
|---|---|---|---|---|
| Jan 20 | Regulatory HOPE-3 update | Positive | -0.6% | FDA requested full HOPE-3 CSR but no new studies, supporting BLA review. |
| Dec 16 | HOPE-3 webinar | Positive | +0.5% | Community webinar to review positive Phase 3 HOPE-3 topline results. |
| Dec 03 | Phase 3 topline data | Positive | +371.1% | HOPE-3 met primary PUL v2.0 and key cardiac LVEF endpoints with significance. |
| Sep 09 | CRL response update | Negative | +2.0% | Company responded to public posting of FDA Complete Response Letter for BLA. |
| Aug 18 | Exosome vaccine trial | Neutral | -3.3% | First subjects dosed in Phase 1 trial of StealthX exosome-based vaccine. |
24h Move is the share-price change in the day after each event; other market factors may also have contributed.
Clinical and regulatory HOPE-3 updates have produced volatile reactions, including a 371.07% spike on positive Phase 3 topline data and smaller, sometimes negative moves on subsequent regulatory updates.
Over the past year, CAPR’s key milestones have centered on Deramiocel’s HOPE-3 program. Positive Phase 3 topline data on Dec 3, 2025 drove a 371.07% move, followed by a community webinar and subsequent FDA interactions about the Complete Response Letter and full HOPE-3 clinical study report. Additional clinical and regulatory updates in 2026 have focused on resuming BLA review and setting a new PDUFA date, underscoring a trajectory from pivotal data readout to late-stage regulatory review.
Key Terms
performance of the upper limb (pul v2.0) medical
duchenne video assessment medical
orphan drug designation regulatory
regenerative medicine advanced therapy (rmat) regulatory
advanced therapy medicinal product (atmp) regulatory
rare pediatric disease designation regulatory
biologics license application (bla) regulatory
pdufa target action date regulatory
AI-generated analysis. How Rhea-AI works. Not financial advice.
SAN DIEGO, April 22, 2026 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for rare diseases, today announced the presentation of data from its Phase 3 HOPE-3 clinical trial of Deramiocel in Duchenne muscular dystrophy (DMD) at the American Academy of Neurology (AAN) 2026 Annual Meeting in Chicago, Illinois. The data were presented by Dr. Aravindhan Veerapandiyan, Associate Professor and Director of the Comprehensive Neuromuscular Program at Arkansas Children's Hospital, during the Late-Breaking Science 2 session on Tuesday, April 21, 2026.
AAN Presentation Highlights
"No therapeutic has ever delivered a statistically and clinically meaningful difference in the upper limb functional primary endpoint of Performance of the Upper Limb (PUL v2.0)," said Dr. Aravindhan Veerapandiyan, Associate Professor and Director of the Comprehensive Neuromuscular Program at Arkansas Children's Hospital. "These findings were further supported by an alternative measure of upper limb function, specifically video recordings of tasks performed at home (the Duchenne Video Assessment, or DVA), which showed meaningful slowing of disease progression in the ability to self-feed, a function central to patient independence. These data reinforce the potential of Deramiocel to make a meaningful difference in the lives of those living with Duchenne."
"Bringing these results to AAN reflects the significant momentum behind Deramiocel and our deep commitment to the Duchenne community," said Linda Marbán, Ph.D., Chief Executive Officer of Capricor. "The HOPE-3 data tell a compelling story of preserved function, slowed decline, and real-world impact on patients' daily lives, evidence we believe positions Deramiocel as a potentially transformative therapy for Duchenne. With our BLA currently under FDA review and a PDUFA target action date of August 22, 2026, we remain on track and focused on bringing this therapy to the patients who need it as quickly as possible."
Data from this presentation were released under AAN embargo on April 21, 2026 at 4:45 p.m. ET.
Additional information about the 2026 AAN Annual Meeting is available at www.aan.com. The Company's presentation will be made available in the Investors section of Capricor's website shortly.
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 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, 2025, as filed with the Securities and Exchange Commission on March 17, 2026. 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:
Caitlin Kasunich
KCSA Strategic Communications
ckasunich@kcsa.com
212.896.1241
Capricor Company Contact:
AJ Bergmann, Chief Financial Officer
abergmann@capricor.com
858.727.1755
