Capricor Therapeutics’ Deramiocel: Overcoming Setbacks with Promising Phase 3 Trial and Long-term Efficacy Data

H.C. Wainwright analyst Joseph Pantginis maintained a Buy rating on Capricor Therapeutics today and set a price target of $24.00.

Joseph Pantginis has given his Buy rating due to a combination of factors that highlight the potential of Capricor Therapeutics’ deramiocel, despite recent setbacks. The company received a Complete Response Letter (CRL) from the FDA regarding its Biologics License Application (BLA) for deramiocel, a treatment for cardiomyopathy in Duchenne muscular dystrophy (DMD) patients, citing insufficient evidence of effectiveness from the HOPE-2 trial. However, Capricor plans to address these concerns with data from the upcoming Phase 3 HOPE-3 trial, expected in the third quarter of 2025, which could provide the necessary evidence to support the BLA resubmission.
Moreover, long-term data from the HOPE-2 open-label extension study indicates that deramiocel continues to demonstrate safety and efficacy, with minimal decline in left ventricular ejection fraction over four years. This reinforces the potential for deramiocel to stabilize cardiac function and preserve skeletal muscle in DMD patients. The FDA’s willingness to engage in a Type A meeting and Capricor’s proactive steps to address the CRL suggest a viable path forward, supporting Pantginis’s positive outlook and Buy rating for Capricor’s stock.

In another report released on June 30, Alliance Global Partners also initiated coverage with a Buy rating on the stock with a $20.00 price target.