Capricor Therapeutics to Present Long-Term Data from HOPE-2 Open Label Extension Study at 2024 World Muscle Society Congress

Rhea-AI Summary

Capricor Therapeutics (NASDAQ: CAPR) announced it will present three-year safety and efficacy results from its HOPE-2 open-label extension (OLE) study for deramiocel, its lead asset for treating Duchenne muscular dystrophy (DMD). The data will be presented in a late-breaking poster at the 29th Annual Congress of the World Muscle Society (WMS 2024) in Prague, Czechia, from October 8-12, 2024.

The presentation, titled "Multi-modal benefits of deramiocel (CAP-1002) in late-stage patients with DMD: a new treatment approach to target skeletal and cardiac muscle pathogenesis (HOPE 2-OLE trial: 36-month data)," will be led by Dr. Craig McDonald from UC Davis. It will take place on October 11, 2024, from 9:45-10:45 a.m. EDT.

Linda Marbán, Ph.D., CEO of Capricor, stated that these results support the company's recent decision to begin filing a Biologics License Application (BLA) for potential approval of deramiocel to treat patients with DMD-cardiomyopathy.

SAN DIEGO, Oct. 04, 2024 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, announced today that the Company will present the three-year safety and efficacy results from its HOPE-2 open-label extension (OLE) study with lead asset deramiocel for treating Duchenne muscular dystrophy (DMD). The data will highlight the long-term, multi-modal benefits of deramiocel in a late-breaking poster presentation at the 29^th Annual Congress of the World Muscle Society (WMS 2024), taking place October 8-12, 2024, in Prague, Czechia.

“We look forward to sharing the latest updates from our HOPE-2 OLE trial at this year’s World Muscle Society Congress,” said Linda Marbán, Ph.D., Chief Executive Officer of Capricor. “The results of this study are foundational to our recent announcement to commence the filing of a Biologics License Application (BLA) for potential approval of deramiocel to treat patients with DMD-cardiomyopathy and continue to support the long-term impact of deramiocel for the treatment of DMD."

Late Breaking Poster
Title:	Multi-modal benefits of deramiocel (CAP-1002) in late-stage patients with DMD: a new treatment approach to target skeletal and cardiac muscle pathogenesis (HOPE 2-OLE trial: 36-month data)
Lead Author:	Dr. Craig McDonald (UC Davis)
Details:	721LBP, Session 4, October 11, 2024 (9:45-10:45 a.m. EDT (15:45-16:45 CEST)

A copy of the poster presentation will be added to the publications section of the Capricor website following the presentation. The full WMS 2024 program is available at https://www.wms2024.com/page/programme.

About Capricor Therapeutics

Capricor Therapeutics, Inc. (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 (CAP-1002), an allogeneic cardiac-derived cell therapy. Extensive preclinical and clinical studies have shown deramiocel to demonstrate immunomodulatory, antifibrotic, and regenerative actions specifically tailored for dystrophinopathies and heart disease. Deramiocel is currently advancing through Phase 3 clinical development for the treatment of Duchenne muscular dystrophy. Capricor is also harnessing the power of its exosome technology, using its proprietary StealthX™ platform in preclinical development focused on the areas of vaccinology, targeted delivery of oligonucleotides, proteins and small molecule therapeutics to potentially treat and prevent a diverse array of diseases. At Capricor, we stand 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 Twitter.

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 discovery efforts and clinical trials; the pace of enrollment of clinical trials; plans regarding regulatory filings, future research and clinical trials; regulatory developments involving products, including the ability to obtain regulatory approvals or otherwise bring products to market; manufacturing capabilities; dates for regulatory meetings; statements about our financial outlook; the ability to achieve product milestones and to receive milestone payments from commercial partners; plans regarding current and future collaborative activities and the ownership of commercial rights; potential future agreements; scope, duration, validity and enforceability of intellectual property rights; future revenue streams and projections; expectations with respect to the expected use of proceeds from the recently completed offerings and the anticipated effects of the offerings; 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, 2023, as filed with the Securities and Exchange Commission on March 11, 2024, and in our Quarterly Report on Form 10-Q for the quarter ended June 30, 2024, as filed with the Securities and Exchange Commission on August 8, 2024. 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 (CAP-1002) for DMD in the United States and Japan with Nippon Shinyaku Co., Ltd. (U.S. subsidiary: NS Pharma, Inc.), subject to regulatory approval. Deramiocel is an Investigational New Drug and is not approved for any indications. None of Capricor’s exosome-based candidates have been approved for clinical investigation.

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 will Capricor Therapeutics present at the 2024 World Muscle Society Congress?

Capricor Therapeutics (CAPR) will present three-year safety and efficacy results from its HOPE-2 open-label extension study on deramiocel for treating Duchenne muscular dystrophy (DMD) at the 2024 World Muscle Society Congress.

When and where will the 29th Annual Congress of the World Muscle Society (WMS 2024) take place?

The 29th Annual Congress of the World Muscle Society (WMS 2024) will take place from October 8-12, 2024, in Prague, Czechia.

What is the title of Capricor's late-breaking poster presentation at WMS 2024?

The title of Capricor's late-breaking poster presentation is "Multi-modal benefits of deramiocel (CAP-1002) in late-stage patients with DMD: a new treatment approach to target skeletal and cardiac muscle pathogenesis (HOPE 2-OLE trial: 36-month data)".

Who is the lead author of Capricor's presentation at WMS 2024?

The lead author of Capricor's presentation at WMS 2024 is Dr. Craig McDonald from UC Davis.

What recent announcement did Capricor make regarding deramiocel?

Capricor recently announced the commencement of filing a Biologics License Application (BLA) for potential approval of deramiocel to treat patients with DMD-cardiomyopathy.