Capricor Comments on Briefing Materials Ahead of July 29 FDA Advisory Committee Meeting for Deramiocel
Rhea-AI Summary
Capricor Therapeutics (NASDAQ: CAPR) reported an update ahead of the July 29 FDA Advisory Committee meeting, where an independent panel will discuss Capricor’s Biologics License Application (BLA) for Deramiocel, an investigational cell therapy for Duchenne muscular dystrophy (DMD).
According to Capricor, its efficacy results are governed by the final statistical analysis plan (SAP) version 3.0, finalized before unblinding. The company stated that post-hoc analyses in FDA briefing materials use SAP version 1.1, described as an unsigned, incomplete internal draft that became obsolete after adding cohort B and omitted FDA-requested content. Capricor reports that the Phase 3 HOPE-3 trial showed a statistically significant benefit on the primary endpoint, PUL 2.0, with supportive cardiac function data, and believes Deramiocel may offer a meaningful option for boys and young men with DMD. Selected advisory committee slides are being posted in the Investors section of Capricor’s website.
Positive
- Phase 3 HOPE-3 met primary endpoint with statistically significant benefit on PUL 2.0, according to Capricor
- Supportive cardiac function benefits reported from Deramiocel in the HOPE-3 study
- BLA for Deramiocel under FDA review with an advisory committee meeting scheduled for July 29
- Deramiocel in late-stage development for DMD, shown in clinical studies to preserve cardiac and skeletal muscle function
Negative
- None.
News Market Reaction – CAPR
In the Jul 27 session, CAPR declined 64.49%, reflecting a significant negative market reaction. Argus tracked a trough of -71.0% from its starting point during tracking. Our momentum scanner triggered 238 alerts that day, indicating exceptionally high trading interest and price volatility. Trading volume was exceptionally heavy at 15.7x the daily average, suggesting significant selling pressure.
Data tracked by StockTitan Argus on the day of publication.
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SAN DIEGO, July 27, 2026 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for rare diseases, today provided an update ahead of the Company's upcoming FDA Advisory Committee meeting, at which an independent advisory committee will discuss Capricor's Biologics License Application (BLA) for Deramiocel, an investigational cell therapy for the treatment of Duchenne muscular dystrophy (DMD), as part of the FDA's ongoing review of the application.
“Capricor has engaged fully and transparently with the FDA throughout the review process for our Biologics License Application for Deramiocel and has been responsive to every request from the FDA. Our results are governed by the final analysis plan, SAP version 3.0, which was finalized prior to unblinding. It is critical to understand that the post-hoc analyses in the FDA’s briefing materials rely on SAP version 1.1, an unsigned incomplete internal draft which became obsolete with the addition of cohort B and did not include content specifically requested by FDA. The Phase 3 HOPE-3 results demonstrate a statistically significant benefit on the primary endpoint, PUL 2.0, with supportive benefits in cardiac function, and we believe Deramiocel offers a meaningful treatment option for boys and young men living with Duchenne, who continue to face a significant unmet medical need. Ahead of the meeting, we have posted a portion of our slides to accurately frame the discussion for stakeholders on our website,” said Linda Marbán, Ph.D., Chief Executive Officer of Capricor Therapeutics.
These materials will be available in the Investors section of the Company’s website shortly.
About Capricor Therapeutics
Capricor Therapeutics (NASDAQ: CAPR) is a biotechnology company dedicated to advancing cell and exosome-based therapeutics for the treatment of rare diseases. Our lead product candidate, Deramiocel, is an allogeneic cardiac-derived cell therapy in late-stage development for Duchenne muscular dystrophy (DMD), shown in clinical studies to preserve cardiac and skeletal muscle function. Capricor is also advancing its proprietary StealthX™ exosome platform for the targeted delivery of oligonucleotides, proteins, and small-molecule therapeutics across a range of diseases. At Capricor, we are committed to delivering new therapies for patients with rare diseases. 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, revenue and reimbursement estimates, projected terms of definitive agreements, our financial position, our possible uses of existing cash and investment resources, and statements regarding our litigation with Nippon Shinyaku Co., Ltd. and NS Pharma, Inc., including the nature of the dispute, our expectations regarding any legal proceedings, and our ability to commercialize Deramiocel independent of our existing distribution agreement 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 and in our Quarterly Report on Form 10-Q for the quarter ended March 31, 2026, as filed with the Securities and Exchange Commission on May 13, 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.
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
