Capricor Therapeutics announces long-term data from HOPE-2 OLE study

Capricor Therapeutics (CAPR) announced long-term data from its ongoing HOPE-2 open label extension clinical trial, demonstrating the potential of the Company’s lead asset, deramiocel, to slow disease progression and preserve upper limb function in patients with Duchenne muscular dystrophy. The data is presented as a late breaking poster at this year’s Muscular Dystrophy Association Clinical and Scientific Conference, which began on March 16 and runs through March 19 in Dallas, Texas. In a cohort-matched external comparator analysis, the study showed that patients treated with deramiocel over three years experienced an average decline in Performance of the Upper Limb total score of 3.46 points, compared to a 7.19-point decline in the external comparator group. This equates to a 52 percent slowing of disease progression, reinforcing deramiocel’s potential long-term therapeutic durability. Additional findings include: Treatment effect increases year over year – Patients on deramiocel showed a reduction in disease progression, with a mean annual PUL 2.0 decline of 1.8 points in Year 1, 1.2 points in Year 2 and 1.1 points in Year 3. Potential disease-modifying effects – During a 1-year gap of treatment, those originally randomized to deramiocel showed a slower rate of decline compared to untreated patients. Favorable safety profile – Deramiocel was well tolerated with no new safety signals identified and continues to maintain a favorable long-term benefit-risk profile.