Capricor Therapeutics

After an FDA advisory committee voted against approval for cardiomyopathy in Duchenne muscular dystrophy, Capricor will amend its BLA to pursue an upper-limb skeletal muscle indication supported by the HOPE-3 primary endpoint. The HOPE-3 data showed a statistically significant 4.5% slowing of upper-limb decline and supportive safety data from over 1,300 infusions, but a key secondary ejection-fraction result weakened under revised analysis. Financially, cash fell to $237.9 million and Q2 net loss widened to $40.7 million as operating costs rose, while short interest remains elevated at about 33.7% of float. » Altro su finance.yahoo.com

Capricor Therapeutics shares rose 15.7% after Oppenheimer upgraded the stock from “Perform” to “Outperform” and set a $54 price target. The move follows new FDA information on Deramiocel for treating Duchenne muscular dystrophy; the FDA accepted submitted documents as a substantive amendment and set a new PDUFA date of November 22. Analyst Leland Gershall now sees improved approval chances, noting the agency could consider an indication for upper-limb function, which corresponds to the primary endpoint of the Phase 3 study. Capricor plans to release additional 24‑month data from the HOPE-3 study and further information ahead of the PDUFA date. » Altro su de.investing.com

The FDA has extended the review deadline for the biologics license application for Deramiocel by three months to November 22, 2026, after Capricor submitted an amendment. The amendment includes data from the 24-month, open-label extension phase of the Phase III HOPE-3 study and additional analyses narrowing the indication to upper-limb function. The FDA considered the submission a substantive amendment; HOPE-3 met its primary endpoint. In the US, Deramiocel has orphan status, RMAT designation and rare pediatric disease designation, which could make it eligible for a Priority Review Voucher if approved. » Altro su de.investing.com