US-based Capricor Therapeutics has announced that its pivotal Phase 3 HOPE-3 trial of Deramiocel, the company’s investigational cell therapy for DMD has delivered positive topline results, 

 

“HOPE-3 delivered strong and definitive evidence that Deramiocel can meaningfully improve the course of Duchenne muscular dystrophy, demonstrating statistically significant improvements in both skeletal and cardiac function,” said Linda Marbán,  CEO of Capricor. 

 

“These results reinforce the durable benefits seen in HOPE-2 and its open-label extension, which has continued for over 48 months, and highlight the strength, consistency, and reproducibility of Deramiocel’s clinical profile after more than a decade of rigorous clinical development. We believe these pivotal study results, in addition to the evidence from the HOPE-2 and HOPE-2 OLE studies, position us to address the clinical issues in the Complete Response Letter received earlier this year, consistent with prior FDA guidance that HOPE-3 results should be sufficient to support regulatory approval.”

 

HOPE-3 was a randomized, double-blind, placebo-controlled trial that enrolled 106 boys and young men with DMD across 20 US clinical sites. Participants received intravenous Deramiocel at 150 million cells per infusion or placebo every three months for 12 months, while continuing stable corticosteroid therapy. Most participants were taking cardiac medications at baseline, and over 75% had a clinical diagnosis of cardiomyopathy. The therapy maintained a favorable safety and tolerability profile consistent with prior studies.

 

“We believe the HOPE-3 PUL results show statistically and clinically meaningful and significant treatment effects on both upper limb function and cardiomyopathy,” said Craig McDonald, Distinguished Professor of Physical Medicine & Rehabilitation and Pediatrics at UC Davis Health, and National PI of HOPE-3. 

 

“A nearly 54 percent slowing of skeletal muscle disease progression is extraordinary in Duchenne and directly linked to maintaining independence and quality of life in the most severely affected patients with greatest unmet need. The preservation of function reflected in PUL v2.0 translates into real, practical benefits for boys and young men living with this disease, and the effect of Deramiocel on cardiomyopathy will potentially translate to improved long-term survival. 

 

"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.”

 

Cardiac breakthroughs also stood out in the trial. “The cardiac findings from HOPE-3 represent a significant advance in the management of Duchenne muscular dystrophy cardiomyopathy,” said Jonathan Soslow, Professor of Pediatrics (Cardiology) at Vanderbilt University Medical Center. 

 

“Cardiomyopathy is the leading cause of mortality in Duchenne, and stabilizing cardiac function has remained a major unmet need. 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.”

 

Dr Marbán added, “For families living with Duchenne who are looking for therapies that preserve functional ability, protect the heart and maintain independence, today’s results provide real momentum and meaningful progress, offering renewed confidence as we work to advance Deramiocel toward potential regulatory approval.”