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Capricor Therapeutics Reports: Phase 2 Study Evaluating CAP-1002 in Late-Stage Duchenne Muscular Dystrophy Published in The Lancet

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Capricor Therapeutics (NASDAQ: CAPR), 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 that The Lancet, a renowned peer-reviewed global medical source for clinical and global health, has published positive results from Capricor’s HOPE-2 Phase 2 clinical trial using lead asset, CAP-1002, to treat patients in advanced stages of Duchenne muscular dystrophy (DMD).

Capricor Therapeutics notes specifically, the study demonstrated that young men in the advanced stages of DMD experienced improvements in both skeletal and cardiac muscle function after receiving four doses of CAP-1002 over the course of one year. The paper is titled, “Repeated intravenous cardiosphere-derived cell therapy in late-stage Duchenne muscular dystrophy (HOPE-2): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial.”

“Robust research is imperative to truly usher in a new class of treatments for these patients in dire need and historically Duchenne trials have not focused of the most severely affected non-ambulatory patients with the greatest disease burden,” said Dr. Craig McDonald, the national principal investigator for HOPE-2, UC Davis professor and chair 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 an eminent publication such as The Lancet, represents a major achievement for the development of CAP-1002 and further validates the power of this cell therapy for all patients with DMD regardless of their stage of disease.”

“CAP-1002 has shown safety and very promising efficacy in patients with DMD in two successful clinical trials to date, setting the stage for a novel therapeutic that can be life changing for patients with DMD,” said Linda Marbán, Ph.D., Chief Executive Officer of Capricor Therapeutics. “Further, our recently announced partnership with Nippon Shinyaku has secured us a commercialization and distribution partner in the US, experienced in rare diseases with specific expertise in DMD. Capricor is initiating our Phase 3, HOPE-3 clinical study imminently as we move towards potential commercialization.”

HOPE-2 Study Details

HOPE-2 was a randomized, double-blind, placebo-controlled, Phase 2 clinical trial with the Company’s lead investigational therapy, CAP-1002, in boys and young men who have DMD and are in the later stages of the disease’s progression. Presently, this comprises approximately half of all DMD patients. 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 a placebo every three months. Data from a total of 20 patients was analyzed (12 placebo and eight treated) at the 12-month mark. Approximately 80 percent of 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 one 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 and hand) function, with a conceptual framework reflecting the progression of weakness in upper limb 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 with a common pre-medication regimen, there were no serious safety signals identified by the HOPE-2 Data and Safety Monitoring Board (DSMB).

Pat Furlong, Founding President and CEO of Parent Project Muscular Dystrophy, a nonprofit organization leading the fight to end Duchenne said, “The results from this study are highly encouraging for people with Duchenne, especially our non-ambulatory community who have limited therapeutic options. Further, CAP-1002 has demonstrated cardiac benefits in this patient population where heart failure continues to be the leading cause of mortality. These data support the hope that CAP-1002 may one day become an important therapeutic option and slow the advancement of the disease.”

About CAP-1002

CAP-1002 consists of allogeneic cardiosphere-derived cells, or CDCs, a type of progenitor cell that has been shown in pre-clinical and clinical studies to exert potent immunomodulatory activity and is being investigated for its potential to modify the immune system’s activity to encourage cellular regeneration. CDCs have been the subject of over 100 peer-reviewed scientific publications and have been administered to over 200 patients across several clinical trials.

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy is a devastating genetic disorder characterized by progressive weakness and chronic inflammation of the skeletal, heart and respiratory muscles. Patients suffering from DMD typically lose their ability to walk in their teenage years and generally die of cardiac or respiratory complications by age 30. It occurs in one in every 3,600 live male births across all races, cultures and countries. DMD afflicts approximately 200,000 boys and young men around the world. Treatment options are limited, and there is no cure.

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