LOS ANGELES, Feb. 06, 2019 (GLOBE NEWSWIRE) — Capricor Therapeutics (NASDAQ: CAPR), a clinical-stage biotechnology company, today announced that it has resumed per protocol dosing of patients already enrolled in its HOPE-2 clinical trial of CAP-1002, the company’s novel cell therapy candidate to treat Duchenne muscular dystrophy. Approximately 20 young men and boys in advanced stages of Duchenne muscular dystrophy have already been enrolled in the randomized, double-blind, placebo-controlled trial to date.
Capricor had put a voluntary hold on dosing in December after a patient in the HOPE-2 trial had a serious adverse event in the form of anaphylaxis. The investigation suggested the patient may have been allergic to something contained in the investigational product, including an excipient, or inactive ingredient, in the formulation.
To reduce the risk of future events, Capricor initiated a pre-medication strategy commonly used by physicians to prevent and treat allergic reactions. The U.S. Food and Drug Administration (FDA) and the Data and Safety Monitoring Board (DSMB) have granted permission to resume enrollment in the HOPE-2 study.
The HOPE-2 trial is studying the safety and effectiveness of CAP-1002 in older Duchenne patients who are not currently eligible for gene therapy clinical trials. Enrollment of new patients will depend on various factors but will not commence until additional funding is secured.
To reduce expenses and better align resources and personnel on the company’s core lead programs, Capricor has reduced its staff by 21 full-time employees. The reduction in operating expenses is expected to extend the company’s cash, cash equivalents and marketable securities into late 2019. Additionally, Capricor is exploring strategic alternatives, with respect to one or more of its product candidates.
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy is a devastating genetic disorder that causes muscle degeneration and leads to death, generally before the age of 30, most commonly from heart failure. It occurs in one in every 3,600 live male births across all races, cultures and countries. Duchenne muscular dystrophy afflicts approximately 200,000 boys and young men around the world. Treatment options are limited, and there is no cure.
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 immuno-modulatory 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 approximately 140 human subjects across several clinical trials.
About Capricor Therapeutics
Capricor Therapeutics, Inc. (NASDAQ:CAPR) is a clinical-stage biotechnology company focused on the discovery, development and commercialization of first-in-class biological therapeutics for the treatment of rare disorders. Capricor’s lead candidate, CAP-1002, is an allogeneic cell therapy that is currently in clinical development for the treatment of Duchenne muscular dystrophy. Capricor has also established itself as one of the leading companies investigating the field of extracellular vesicles and is exploring the potential of CAP-2003, a cell-free, exosome-based candidate, to treat a variety of disorders. For more information, visit www.capricor.com.