Capricor reports promising DMD treatment results

SAN DIEGO – Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company with a market capitalization of $609 million and an impressive six-month stock return of over 160% according to InvestingPro, has announced positive long-term results from its HOPE-2 clinical trial, suggesting its lead asset, deramiocel, may significantly slow the progression of Duchenne muscular dystrophy (DMD). Data from the trial, presented at the Muscular Dystrophy Association Conference in Dallas, Texas, indicates a 52% reduction in disease progression over three years in patients treated with deramiocel.

The study compared the average decline in Performance of the Upper Limb (PUL 2.0) total score between patients receiving deramiocel and those in an external comparator group. The treated cohort’s average decline was 3.46 points versus 7.19 points in the untreated group, a statistically significant difference.

Yearly assessments showed a consistent reduction in disease progression among patients on deramiocel, with a mean annual PUL 2.0 decline of 1.8 points in the first year, 1.2 points in the second, and 1.1 points in the third. Additionally, patients who had a gap year in treatment continued to experience a slower decline compared to untreated individuals.

The therapy’s safety profile was also highlighted, with no new safety concerns identified, suggesting a favorable long-term benefit-risk profile. Capricor’s CEO, Linda Marbán, Ph.D., emphasized the importance of these findings for patients and families dealing with DMD, as deramiocel appears to not only slow but alter the disease’s trajectory. Analysts share this optimism, with price targets ranging from $25 to $77, according to InvestingPro data, reflecting significant potential upside from current levels.

Deramiocel (CAP-1002) comprises allogeneic cardiosphere-derived cells, known for their immunomodulatory and antifibrotic properties. The therapy is in late-stage development for DMD treatment, with the U.S. Food and Drug Administration (FDA) having accepted a Biologics License Application for associated cardiomyopathy. The FDA has set a target action date of August 31, 2025.

DMD is a severe genetic disorder leading to progressive muscle weakness and chronic inflammation, primarily affecting males, with an estimated patient population of 15,000-20,000 in the United States. Current treatments are limited, and there is no cure.

This news is based on a press release statement from Capricor Therapeutics. The company focuses on developing cell and exosome-based therapeutics for rare diseases, with over 100 scientific publications and 200 human subjects treated in several clinical trials. The full poster presentation will be available on the Capricor website after the conference. With a strong current ratio of 4.19 and revenue growth of 65.33% in the last twelve months, the company maintains a solid financial foundation for its development programs. Investors seeking deeper insights into Capricor’s financial health and growth prospects can access comprehensive analysis and 14 additional ProTips through InvestingPro’s detailed research reports.

In other recent news, Capricor Therapeutics has made significant strides with its Biologics License Application (BLA) for deramiocel, a cell therapy for Duchenne muscular dystrophy (DMD) cardiomyopathy. The U.S. Food and Drug Administration (FDA) has accepted the BLA and granted it Priority Review status, setting a target action date of August 31, 2025. This development marks a critical step toward potentially introducing the first therapy for DMD cardiomyopathy, a condition with no current approved treatments. The BLA is supported by data from Phase 2 trials and comparisons with FDA-funded natural history data.

Capricor is actively preparing for potential approval and commercialization, including expanding manufacturing capabilities and forming strategic partnerships. In addition, the therapy has received Orphan Drug Designation from both the FDA and the European Medicines Agency (EMA), along with RMAT and ATMP designations in the U.S. and Europe, respectively. H.C. Wainwright has reaffirmed its Buy rating on Capricor, citing the strong safety and efficacy profile of deramiocel as reasons for optimism. The firm maintains a price target of $77, reflecting confidence in the therapy’s potential market impact.

Furthermore, Capricor has completed its BLA submission on schedule, triggering a $10 million milestone payment from its partner, Nippon Shinyaku. If approved, deramiocel could become a quarterly-administered, lifelong treatment option for DMD-cardiomyopathy patients.

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