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Capricor Therapeutics Announces Positive Final Data From its Phase 2 HOPE-2 Trial in Patients with Duchenne Muscular Dystrophy Treated with CAP-1002–Trial Met its Primary Efficacy Endpoint of Mid-level Performance of Upper Limb (PUL) v1.2 (p=0.01)– –Additional Positive Endpoints of Full PUL v2.0 (p=0.04) and Cardiac Endpoint of Ejection Fraction (p=0.002)– –One-Year Results Demonstrated CAP-1002 Slowed Decline by 71% (Mid-level PUL v1.2)– –CAP-1002 Significantly Improved Cardiac Function in Patients– –Results Presented Today at World Muscle Society Annual Meeting in Late Breaking Oral Presentation– –Principal Investigator Dr. Craig McDonald and Capricor Management Team will Host a Conference Call and Webcast Today at 8:30 a.m. ET– LOS ANGELES, Sept. 24, 2021 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR) (“Capricor” or “the Company”), a biotechnology company focused on the development of transformative cell and exosome-based therapeutics for the treatment and prevention of a broad spectrum of diseases, announced today positive final data from the HOPE-2 clinical trial using CAP-1002 to treat patients in advanced stages of Duchenne muscular dystrophy (DMD). The HOPE-2 clinical trial met its primary efficacy endpoint of mid-PUL v1.2 as well as various skeletal and cardiac endpoints suggesting clinically relevant slowing of disease progression. CAP-1002 is Capricor’s cell-based therapeutic candidate whose mechanism of action is immunomodulatory, anti-fibrotic and has been shown to regenerate skeletal and cardiac muscle cells. This final data will be presented today at this year’s World Muscle Society Virtual Congress (WMS). Dr. Craig McDonald, the national principal investigator for the HOPE-2 clinical trial and UC Davis professor and chair of the Department of Physical Medicine and Rehabilitation commented, “This groundbreaking study is extremely exciting as we saw statistically significant changes of CAP-1002 in both skeletal and cardiac function. For these older boys who have limited therapeutic options, these data support the belief that CAP-1002 may become an important therapeutic option and possibly slow the progression of DMD.” HOPE-2 was a randomized, double-blind, placebo-controlled, Phase 2 clinical trial of the Company’s lead investigational therapy, CAP-1002, in boys and young men who have DMD and are non-ambulant, the later stage of the disease process. The trial was conducted at nine sites across the United States. Study patients were treated via intravenous delivery with either CAP-1002 (150 million cells per infusion) or placebo every 3 months. Data from a total of 20 patients was analyzed (12 placebo and 8 treated) at the 12-month time-point in the intent to treat (ITT) population. Approximately 80% of the patients were non-ambulant and all patients were on a stable regimen of steroids. Demographic and baseline characteristics were similar between the two treatment groups. Final data analysis demonstrated that young men in the advanced stages of DMD experienced improvements in skeletal and cardiac measurements after receiving four doses of CAP-1002 over the course of 1 year. Subjects in the trial were evaluated using the Performance of the Upper Limb (PUL), a validated tool specifically designed for assessing high (shoulder), mid (elbow) and distal (wrist & hand) function, with a conceptual framework reflecting the progression of weakness in upper limb function. Final Efficacy Results
‡Negative value favors CAP-1002. ITT (intent-to-treat) population shown. CAP-1002 was generally safe and well tolerated throughout the study. With the exception of two hypersensitivity reactions early in the clinical trial, which were mitigated with a common pre-medication regimen, there were no serious safety signals identified by the HOPE-2 Data and Safety Monitoring Board (DSMB). “The significance of this data is vitally important to patients and the DMD community. The data suggests that CAP-1002 slowed the decline of DMD in patients for whom few options currently exist,” said Dr. Linda Marbán, Ph.D., Chief Executive Officer of Capricor. “Now that we have clarity from the FDA and based on the strength of this data set, we are poised to embark on the HOPE-3 pivotal trial once we have secured an appropriate partner that can help drive CAP-1002 forward towards commercialization. Most importantly, we are thankful to the patients and families who participated in this study so that we can demonstrate the impact of CAP-1002 in treating DMD.” These data were recently accepted at this year’s World Muscle Society Virtual Congress as a late-breaking oral presentation, due to its high-impact research findings that are of great interest to congress participants. The late-breaking results will be presented today, September 24, 2021. This is the second clinical trial investigating CAP-1002 showing similar results in the treatment of DMD patients. Capricor completed the HOPE-Duchenne (Phase 1/2) trial in 2019, the results of which were published in Neurology, the medical journal of the American Academy of Neurology. The Company has initiated a technology transfer with Lonza, a leading global CMO to prepare for commercial manufacturing of CAP-1002. Dr. McDonald is a professor of pediatrics, professor and chair of the Department of Physical Medicine and Rehabilitation, director of Rehabilitation Services and director of the Neuromuscular Disease Clinics at UC Davis Health. He has served as a principal investigator for more than 30 industry-sponsored trials for DMD and is the study chair for the Duchenne Natural History Study of the Cooperative International Neuromuscular Research Group, a consortium of medical and scientific investigators from academic and research centers who share the common goal of aiming to positively impact the lives of neuromuscular disease patients and their families by conducting well-controlled clinical studies. Conference Call and Webcast Details To participate via a webcast, please visit: http://public.viavid.com/index.php?id=146508 to view the slides. The webcast will be archived for approximately 30 days and will be available at http://capricor.com/news/events/. About CAP-1002 About Duchenne Muscular Dystrophy About Capricor Therapeutics Cautionary Note Regarding Forward-Looking Statements CAP-1002 is an Investigational New Drug and is not approved for any indications. None of Capricor’s exosome-based candidates have been approved for clinical investigation. For more information, please contact: Media Contact: Investor Contact: Company Contact: |