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Capricor Therapeutics Announces Late-Breaking Presentations at 28th Annual Congress of the World Muscle Society

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Capricor Therapeutics announces positive data supporting CAP-1002's efficacy in treating Duchenne Muscular Dystrophy and potential of StealthX™ technology for ASO delivery
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  • Data from the 24-month open label extension of the HOPE-2 clinical trial shows that CAP-1002 attenuates disease progression in patients with Duchenne Muscular Dystrophy by approximately 64%
  • CAP-1002 demonstrates clinically meaningful improvements in cardiac function
  • Capricor's StealthX™ technology shows promise in enhancing the delivery of ASO therapies to muscle tissues
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-24-Month Open Label Extension Data from HOPE-2 Study Supports CAP-1002’s Sustained Efficacy and Safety in Treating Duchenne Muscular Dystrophy-

-Company’s StealthX™ Technology Shows Promise of Exosome-Based Delivery of ASOs-

SAN DIEGO, Oct. 10, 2023 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company focused on the development of transformative cell and exosome-based therapeutics for the treatment and prevention of muscular and other select diseases, today announced data from two late-breaking posters presented at this year’s 28th International Annual Congress of the World Muscle Society (WMS) which took place in Charleston, South Carolina, from October 3-7, 2023. Presentations included data from the two-year open label extension (OLE) of the HOPE-2 clinical trial highlighting the efficacy of Capricor’s lead asset, CAP-1002 to treat patients with Duchenne muscular dystrophy (DMD), and preclinical data showing the potential of the Company’s proprietary StealthX™ exosome platform to enhance the delivery of antisense oligonucleotides (ASOs).

Late-Breaking Poster: Capricor’s 24-Month HOPE-2 Results Show CAP-1002’s Long-Term Safety and Efficacy in Patients with Late-Stage DMD

Data from the Company’s HOPE-2 OLE study measured by the Performance of the Upper Limb (PUL 2.0) showed a mean PUL 2.0 decline after 24-months of treatment with CAP-1002 was 2.8 points versus a 7.7-point decline on average observed over 24-months in the placebo patient group (delta change=4.9 points, p=0.021). The average rate of decline in CAP-1002 treated patients showed an attenuation of disease progression by approximately 64%. Additionally, CAP-1002 revealed clinically meaningful improvements in ameliorating cardiac function. The data showed that cardiac function, as measured by left ventricular ejection fraction (LVEF%) by MRI at the 24-month timepoint, improved in 67% of patients, compared to a steady decline in a comparable natural history population.

Late-Breaking Poster: StealthX™ Platform for Enhanced Exosome-Based ASO Delivery

One of the predominant strategies for treating DMD has been through the employment of antisense oligonucleotides to exclude exons resulting in DMD proteins with partially restored function. The main challenge of antisense drugs is to improve their delivery to target tissues. To overcome this challenge, a muscle-targeting moiety was engineered on the surface of the exosomes using Capricor’s StealthX™ technology. The study results showed the presence of exosomes loaded with a labeled ASO in the lower limbs of mice 24 hours post-injection. Notably, the exosomes carrying the muscle-targeting moiety were not detected in any other tissues except for the expected clearance pathways (kidney and liver). This early data suggests that Capricor's StealthX™ technology holds promise for enhancing the delivery of ASO therapies to muscle tissues, potentially leading to improved functional restoration.

“Capricor remains focused on our mission to develop and commercialize therapies for DMD and the data presented at this year’s WMS support that goal,” said Linda Marbán, Ph.D., Capricor’s Chief Executive Officer. “The 24-month OLE data show long-term efficacy and attenuation of disease progression, which demonstrate its potentially broad therapeutic use across all stages of DMD. Most importantly, CAP-1002 continues to show a favorable safety profile. Building on this momentum, we look forward to delivering on important milestones for this program, including the completion of enrollment of our HOPE-3, Phase 3 study, reporting the outcome of the interim futility analysis in the fourth quarter of 2023 and reporting top-line data in late 2024. While our major focus is on commercializing CAP-1002, we continue to progress our proprietary StealthX™ program by demonstrating that we can target, load and deliver exosomes. This is part of our long-term plan to leverage our exosome platform for the development of therapeutics.”

Session Details: Session 4 was showcased from 3:30-4:30 ET on October 6, 2023 and data was embargoed until that time. Abstract Numbers: LBP12 and LBP13

The full WMS Congress program is available here.

The Capricor posters are available on the publications section of the Capricor website.

About Capricor Therapeutics

Capricor Therapeutics, Inc. (NASDAQ: CAPR) is a biotechnology company focused on the development of transformative cell and exosome-based therapeutics for the treatment and prevention of muscular and other select diseases. Capricor’s lead candidate, CAP-1002, is an allogeneic cardiac-derived cell therapy that is currently in late-stage clinical development for treating Duchenne muscular dystrophy. Further, Capricor has entered into a partnership for the exclusive commercialization and distribution of 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. Capricor is also developing its exosome technology as a next-generation therapeutic platform. Our proprietary StealthX™ exosome platform has potential for a broad range of new therapeutic applications in the field of vaccinology as well as targeted oligonucleotide, protein and small molecule therapeutics to treat or prevent a variety of diseases. For more information, visit capricor.com, and follow Capricor on FacebookInstagram 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; 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; scope, duration, validity and enforceability of intellectual property rights; future royalty streams and revenue 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, 2022, as filed with the Securities and Exchange Commission on March 17, 2023 and in our Quarterly Report on Form 10-Q for the quarter ended June 30, 2023, as filed with the Securities and Exchange Commission on August 8, 2023. 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.

CAP-1002 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 Investor Contact:
Joyce Allaire
LifeSci Advisors, LLC
jallaire@lifesciadvisors.com
617.435.6602

Capricor Company Contact:
AJ Bergmann, Chief Financial Officer
abergmann@capricor.com
858.727.1755


Capricor Therapeutics is focused on the development of transformative cell and exosome-based therapeutics for the treatment and prevention of muscular and other select diseases.

The HOPE-2 study is a clinical trial evaluating the efficacy of Capricor's lead asset, CAP-1002, in treating patients with Duchenne Muscular Dystrophy.

The results showed that CAP-1002 attenuated disease progression in patients with Duchenne Muscular Dystrophy by approximately 64% and demonstrated improvements in cardiac function.

Capricor's StealthX™ technology is a platform designed to enhance the delivery of antisense oligonucleotides (ASOs) to target tissues, potentially improving functional restoration.

The next milestones include the completion of enrollment for the HOPE-3 Phase 3 study, reporting the outcome of the interim futility analysis in Q4 2023, and reporting top-line data in late 2024.

Capricor aims to leverage its exosome platform for the development of therapeutics by demonstrating the ability to target, load, and deliver exosomes.

The Capricor posters are available on the publications section of the Capricor website.
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About CAPR

capricor therapeutics, inc. (nasdaq: capr) is a clinical-stage biotechnology company focused on the discovery, development, and commercialization of first-in-class biological therapeutics for the treatment of rare disorders. our innovative technology builds upon a large body of scientific research and enables us to approach the treatment of diseases in novel ways. capricor’s lead candidate, cap-1002, is an “off-the-shelf” cardiac cell therapy that is currently in clinical development for the treatment of duchenne muscular dystrophy (dmd). cap-1002 consists of allogeneic cardiosphere-derived cells, or cdcs, a unique population of cells which include progenitor cells that have been shown to exert potent immunomodulatory activity, which alters the immune system’s activity to stimulate cellular regeneration. cdcs have been the subject of over 100 peer-reviewed scientific publications and have been administered to approximately 140 human subjects across several clinical trials. capricor has