Capricor Therapeutics announces late-breaking presentations at WMS

Capricor Therapeutics 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 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. 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 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.