Capricor Therapeutics Inc. (NASDAQ:CAPR) revealed new data from its ongoing HOPE-3 open-label extension study.
The biotechnology firm is evaluating its lead asset, Deramiocel, which aims to treat upper limb impairment in patients with Duchenne muscular dystrophy (DMD).
Deramiocel is under FDA review with a decision date of November 22.
In July, the FDA advisory panel voted 9-3 that the company failed to provide sufficient evidence supporting Deramiocel’s effectiveness, echoing FDA staff concerns over post hoc changes to key analyses after the late-stage trial concluded.
In the FDA briefing document released on Monday, the staff noted that the submitted data from Study HOPE-3, considered together with the results from Study HOPE-2, do not provide substantial evidence of effectiveness for deramiocel in DMD.
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Reduced Rate Of Decline
Trial results highlight a significant reduction in the rate of disease progression. Patients in the delayed-start placebo group experienced a 2.05-point decline in their PUL 2.0 total score during their first year.
However, after transitioning to Deramiocel in the second year, this decline slowed to just 0.49 points, representing a 76% reduction in the rate of functional loss.
Meanwhile, the early-start cohort receiving Deramiocel continuously showed consistent stabilization, declining by 0.95 points in the first year and 0.89 points in the second year.
The Performance of Upper Limb (PUL 2.0) is a specialized, 22-item clinical assessment tool designed to measure arm and hand function in DMD patients across both ambulant (walking) and non-ambulant (wheelchair-using) stages.
Outperforming Natural History
After 24 months, both study groups demonstrated favorable outcomes compared to natural history predictions.
The early-start arm observed a 3.42-point decline versus a predicted 5.88-point drop, underscoring the therapy’s ability to preserve upper limb function in patients over an extended period.
The delayed-start arm also outperformed natural history, recording a 3.76-point drop compared to a predicted 5.35-point decline.
CAPR Price Action: Capricor Therapeutics shares were down 9.39% at $8.110 at the time of publication on Monday, according to Benzinga Pro data.
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