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Tuesday, Capricor Therapeutics (NASDAQ:CAPR) shares, currently trading at $12.88, maintained their Buy rating and a $77.00 price target from H.C. Wainwright, following an update on the company’s HOPE-2 OLE trial. According to InvestingPro data, analysts maintain a strong Buy consensus with price targets ranging from $25 to $77. The update was presented at the Muscular Dystrophy Association (MDA) conference on March 17, 2025, detailing the efficacy of deramiocel in treating Duchenne muscular dystrophy (DMD).
The HOPE-2 OLE trial is a continuation of the Phase 2 study, which was a randomized, double-blind, placebo-controlled trial. It evaluated the ability of deramiocel to slow disease progression and preserve upper limb function in DMD patients. Participants received intravenous infusions of deramiocel, consisting of 150 million cells per infusion, or a placebo every three months. The company’s strong liquidity position, with a current ratio of 4.19 and minimal debt, as reported by InvestingPro, supports its ongoing clinical development programs.
The study’s authors highlighted that patients treated with deramiocel experienced a significantly slower decline in their Performance of the Upper Limb 2.0 (PUL 2.0) total score compared to an external comparator group. Over more than three years of follow-up, the average decline in the deramiocel group was 3.46 points, versus a 7.19 point decline in the comparator group, representing a 52% reduction in disease progression.
Additional data from the trial showed a consistent reduction in disease progression in patients treated with deramiocel. The mean annual PUL 2.0 decline over three years was 1.8 points in the first year, 1.2 points in the second year, and 1.1 points in the third year. Furthermore, during a one-year gap in treatment, patients who were originally randomized to deramiocel showed a slower rate of decline compared to untreated patients.
The safety profile of deramiocel was also reaffirmed, as the therapy was well tolerated with no new safety signals identified. The long-term benefit-risk profile continues to be favorable. H.C. Wainwright’s analysis suggests that the HOPE-2 OLE study results support the potential long-term safety and therapeutic durability of deramiocel. The firm reiterated its Buy rating and $77 price target for Capricor Therapeutics, reflecting confidence in the therapy’s prospects. With the stock showing strong momentum, gaining over 150% in the past six months, InvestingPro analysis indicates the company is slightly undervalued based on its Fair Value model. Discover more insights and 13 additional ProTips for CAPR in the comprehensive Pro Research Report, available exclusively to InvestingPro subscribers.
In other recent news, Capricor Therapeutics has announced positive long-term results from its HOPE-2 clinical trial for deramiocel, indicating a 52% reduction in the progression of Duchenne muscular dystrophy (DMD) over three years. The U.S. Food and Drug Administration (FDA) has accepted Capricor’s Biologics License Application (BLA) for deramiocel, granting it Priority Review status with a target action date of August 31, 2025. The therapy’s BLA submission is supported by cardiac data from Phase 2 trials, highlighting improvements in cardiac function for DMD cardiomyopathy, a leading cause of death among DMD patients.
H.C. Wainwright has maintained a Buy rating on Capricor’s stock with a price target of $77, citing the strong safety and efficacy records of deramiocel. The firm believes that deramiocel could become a first-in-class treatment option for DMD patients. Capricor is actively preparing for the potential approval and commercialization of deramiocel, enhancing pre-commercial activities, expanding manufacturing capabilities, and establishing strategic partnerships.
The FDA’s acceptance of the BLA marks a significant step towards potentially bringing the first therapy for DMD cardiomyopathy to market. Deramiocel has also received Orphan Drug Designation from both the FDA and the European Medicines Agency, along with RMAT and ATMP designations in the U.S. and Europe, respectively. Capricor could be eligible for a Priority Review Voucher if the FDA approves deramiocel by September 30, 2026.
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