Capricor Therapeutics Inc (NASDAQ: CAPR) shares surged 15.7% on Tuesday after Oppenheimer upgraded the biotech to Outperform and raised its price target to $54, citing regulatory developments for its experimental Duchenne muscular dystrophy treatment, deramiocel.
The upgrade follows the U.S. Food and Drug Administration’s acceptance of new data submitted by Capricor since last month’s advisory committee meeting as a major amendment. This adjustment set a revised Prescription Drug User Fee Act (PDUFA) target action date of November 22, delaying the original review timeline. Oppenheimer analyst Leland Gershell noted that the FDA may consider an upper limb function-based Duchenne indication, which aligns with the Phase 3 trial’s primary endpoint, suggesting clearer efficacy signals than previously observed for cardiomyopathy.
Capricor CEO Linda Marbán indicated the company plans to disclose 24-month data from the HOPE-3 open-label extension study ahead of the PDUFA date. The additional information, including data to be submitted, will support the regulatory review process. The FDA’s decision to classify the new submissions as a major amendment underscores the significance of the additional clinical insights provided.
Gershell emphasized that the regulatory update and follow-up discussions with Marbán reinforced the firm’s constructive outlook on deramiocel’s approval prospects, predicting a gradual upward revaluation of the stock as market sentiment adjusts.
