RMD - Capricor Advances in DMD Therapy With Positive Study Data | Benzinga
Capricor Therapeutics (NASDAQ: CAPR), a biotechnology firm focused on innovative therapies for rare diseases, has announced favorable three-year results from the ongoing HOPE-2 open-label extension (OLE) study on CAP-1002 for Duchenne muscular dystrophy.
Patients treated with CAP-1002 showed sustained improvements in upper limb function and cardiac performance compared to an external dataset from Cincinnati Children's Hospital Medical Center, indicating potential long-term benefits.
Statistical Significance in Disease Attenuation
The HOPE-2 OLE study revealed a statistically significant reduction in the decline of upper limb function (PUL 2.0) and stabilization of left ventricular ejection fraction among CAP-1002-treated patients over three years. This contrasts sharply with the expected decline observed in untreated DMD patients, suggesting that CAP-1002 can potentially slow disease progression and improve quality of life.
CAP-1002 has demonstrated a well-tolerated safety profile throughout the study. This favorable safety data supports the continued use of CAP-1002 in treating DMD and provides reassurance for patients and healthcare providers about the long-term use of this therapeutic approach.
Strategic Pathway to FDA Approval
Capricor highlighted the significance of these findings in a recent Type-B meeting with the FDA, which could expedite the pathway to the ...