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Therapeutic Capricor (NASDAQ: CAPR), a biotechnology company focused on developing transformative cell- and exosome-based therapies for the treatment and prevention of a wide range of diseases, announced today that The Lancet, a renowned and peer-reviewed global medical source for clinical and global health, has published positive results from Capricor’s HOPE-2 Phase 2 clinical trial using the lead asset, CAP-1002, to treat patients in advanced stages of Duchenne muscular dystrophy (DMD). Specifically, the study demonstrated that young men with advanced stages of DMD experienced improvements in skeletal muscle and heart function after receiving four doses of CAP-1002 over the course of a year. The paper entitled “Cardiosphere-Derived Repeated Intravenous Cell Therapy in Advanced-Stage Duchenne Muscular Dystrophy (HOPE-2): A Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase 2 Trial”, can be consulted here.
“Strong research is imperative to truly usher in a new class of treatments for these patients in need and Duchenne’s trials historically have not focused on the most severely affected and disease-affected non-ambulatory patients.” said Dr. Craig McDonald, the national principal investigator for HOPE-2, a UC Davis professor and director of the Department of Physical Medicine and Rehabilitation and lead author of the study. “We believe that the publication of these Phase 2 results in a leading publication such as The Lancet, represents a major step forward for the development of CAP-1002 and further validates the potency of this cell therapy for all patients with DMD, regardless of whatever their stage of development. disease.”
“CAP-1002 has shown very promising safety and efficacy in patients with DMD in two successful clinical trials to date, paving the way for a new treatment that can change the lives of patients with DMD,” Linda said. Marbán, Ph.D., President and CEO of Capricor. “In addition, our recently announced partnership with Nippon Shinyaku has secured us a commercialization and distribution partner in the United States experienced in rare diseases with specific expertise in DMD. Capricor is launching our Phase 3 clinical study, HOPE-3 imminently as we move toward potential commercialization. »
Details of the HOPE-2 study
HOPE-2 was a randomized, double-blind, placebo-controlled, Phase 2 clinical trial of the company’s lead investigational treatment, CAP-1002, in boys and young men with DMD who were in the late stages of disease progression. Currently, this includes about half of all patients with DMD. The trial was conducted at nine sites across the United States. Study patients were treated intravenously with either CAP-1002 (150 million cells per infusion) or placebo every three months. Data from a total of 20 patients were analyzed (12 on placebo and eight on treatment) after 12 months. Approximately 80% of patients were non-ambulatory and all patients were on a stable steroid regimen. Demographic and baseline characteristics were similar between the two treatment groups.
Final data analysis demonstrated that young men with advanced stages of DMD experienced improvements in skeletal and cardiac measures after receiving four doses of CAP-1002 over the course of a year. Trial subjects were assessed using Upper Limb Performance (ULP), a validated tool specifically designed to assess upper (shoulder), mid (elbow), and distal (wrist and hand) function, with a conceptual framework reflecting the progression of weakness in upper extremity function. CAP-1002 was generally safe and well tolerated throughout the study. With the exception of hypersensitivity reactions early in the clinical trial, which were mitigated by a common premedication regimen, no serious safety signals were identified by the HOPE-2 Data and Safety Monitoring Board (DSMB ).
Pat Furlong, Founding President and CEO of Parent Project Muscular Dystrophy, a non-profit organization leading the fight to end Duchenne, said, “The results of this study are very encouraging for people with Duchenne, especially our non-ambulatory community which has limited treatment options. Additionally, CAP-1002 has demonstrated cardiac benefits in this patient population where heart failure continues to be the leading cause of death. These data support the hope that CAP-1002 may one day become an important treatment option and slow disease progression. “