-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-
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 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.”
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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
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.
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