Capricor hits primary endpoint in Duchenne extension study, readying its FDA pitch

It has been a very bumpy ride for Capricor Therapeutics over the past several years, including a halted J&J partnership and venture into penny stock status. However, the latest news from the San Diego-based biotech shows that its experimental Duchenne drug may be a durable, long-term treatment.

The company posted the one-year results from its open-label extension study for their candidate dubbed CAP-1002. The extension from its Phase II study, named HOPE-2, was conducted in boys and young men with later-stage Duchenne muscular dystrophy who paused and then resumed treatment after a year. Researchers measured patients in the one-year follow-up against the original study’s treatment arm effect and in two subsets of the original placebo group.

Capricor’s drug hit the primary endpoint on an upper limb muscle function scale when compared to all three groups. When pitted against the treatment arm, CAP-1002 induced a statistically significant difference in the scale,…
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