Capricor Therapeutics Duchenne Treatment Shows Strong Heart And Muscle Results In Trial

Capricor Therapeutics Inc. (NASDAQ:CAPR) stock is rallying on Wednesday.

The company shared much-awaited topline results from its pivotal Phase 3 HOPE-3 trial evaluating Deramiocel, the company’s investigational cell therapy for Duchenne muscular dystrophy (DMD).

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Duchenne muscular dystrophy (DMD) is a severe, inherited genetic disorder that causes progressive muscle weakness and degeneration, primarily affecting boys.

The study randomized 106 participants. Participants received intravenous Deramiocel at 150 million cells per infusion or placebo every three months for 12 months.

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Data

The average age of participants was approximately 15 years.

Nearly 54% of patients showed slowing of skeletal muscle disease progression, and around 91% patients showed treatment effect on cardiomyopathy (heart function).

“The HOPE-3 study is the first-ever Phase 3 trial in a largely non-ambulatory population with DMD to successfully meet its primary endpoint and to support the development of an innovative therapy over many years, with this level of impact has been a profound privilege,” said Craig McDonald, an investigator of the HOPE-3 trial.

“The statistically and clinically significant preservation of left ventricular ejection fraction in patients treated with Deramiocel observed in HOPE-3 underscores the potential of Deramiocel to address one of the most critical aspects of the disease,” said Jonathan Soslow, Professor of Pediatrics (Cardiology) at Vanderbilt University Medical Center.

Background

In July, Capricor Therapeutics received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding its Biologics License Application (BLA) for Deramiocel, supported by data from the HOPE-2 trial.

The agency specifically cited that the BLA does not meet the statutory requirement for substantial evidence of effectiveness and that additional clinical data is needed.

CAPR Price Action: Capricor Therapeutics shares were up 8.96% at $6.93 during premarket trading on Tuesday, according to Benzinga Pro data.

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