Positive long-term results from the company’s ongoing HOPE-2 open label extension (“OLE”) clinical trial were announced today by Capricor Therapeutics, a biotechnology company that develops transformative cell and exosome-based therapeutics for the treatment of rare diseases.
These results show that the company’s lead asset, deramiocel, has the potential to slow the progression of the disease and preserve upper limb function in patients with Duchenne muscular dystrophy (“DMD”). This year’s Muscular Dystrophy Association Clinical and Scientific Conference, which took place in Dallas, Texas, from March 16 to 19, featured the data as a late breaking poster.
Capricor Therapeutics Deramiocel Clinical Trials
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 (PUL 2.0) total score of 3.46 points, compared to a 7.19-point decline in the external comparator group (p=0.019). This equates to a % 52 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 (2.8 points per year) compared to untreated patients (3.7 points per year).
- Favorable safety profile – Deramiocel was well tolerated with no new safety signals identified and continues to maintain a favorable long-term benefit-risk profile.
