Monday, October 3, 2022


Biotechnology News Magazine

Capricor Therapeutics Announces First Patient Dosed in Pivotal Phase 3 Study of CAP-1002 for the Treatment of Duchenne Muscular Dystrophy

Double-Blind, Randomized, Placebo-Controlled HOPE-3 Clinical Trial Designed to Enroll approximately 70 Patients

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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 muscular and other select diseases, today announced commencement of dosing in HOPE-3, a Phase 3 clinical trial investigating CAP-1002, a cell therapy for treating late-stage Duchenne muscular dystrophy (DMD).

Capricor Therapeutics notes HOPE-3 is a randomized, double-blind, placebo-controlled study designed to enroll approximately 70 patients in the United States.

Capricor Therapeutics recently announced a partnership with Nippon Shinyaku Co., Ltd. which has commercialization and distribution rights in the U.S. This partnership provides funding for the support of HOPE-3 as well as other potential milestone-based payments to support the clinical development of CAP-1002 in DMD.

“We are delighted to begin dosing patients in HOPE-3. The data from our Phase 2 clinical trial suggest that CAP-1002 can slow loss of function by as much as 70% in terms of upper limb skeletal muscle function. Since there are very limited therapeutic options for these patients and CAP-1002 has been shown to be safe and effective, we are pleased to begin this pivotal trial with the goal of achieving regulatory approval as quickly as possible,” said Linda Marbán, Ph.D., CEO of Capricor. “Beginning this clinical trial is a significant milestone, not only for Capricor, but most importantly for those boys and young men with DMD.”

HOPE-3 participants will be randomized to either CAP-1002 or placebo in a 1:1 ratio. The active arm of participants in the trial will receive 150 million cardiosphere-derived cells (CAP-1002) via intravenous infusion every 3 months for a total of 4 doses. CAP-1002 is comprised of human allogeneic cardiosphere-derived cells, with a differentiated mechanism of action that is immunomodulatory and regenerative. Its broad applicability makes it suitable for patients regardless of genetic mutation. The Phase 3 study’s primary outcome measure will be the Performance of the Upper Limb (PUL) 2.0, a validated tool specifically designed for assessing high (shoulder), mid (elbow) and distal (wrist and hand) function, with a conceptual framework reflecting weakness progression in upper limb function. HOPE-3 will also measure various secondary endpoints including cardiac function assessments. For more information on this study, please visit (NCT05126758).

The regulatory pathway for CAP-1002 is supported by RMAT (Regenerative Medicine Advanced Therapy Designation) as well as Orphan Drug Designation. If Capricor were to receive market approval for CAP-1002 by the FDA, Capricor would be eligible to receive a Priority Review Voucher based on its designation as a rare pediatric disease. Capricor holds worldwide commercial rights to CAP-1002 outside of the United States.

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 human patients across several clinical studies.

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy is a 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.

Read other clinical trial news of interest here.

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