Capricor Therapeutics CAPR Announces PositiveTopline Results from Pivotal Phase 3HOPE-3 Study of Deramiocel in DuchenneMuscular Dystrophy

CAPR: Pivotal Phase 3 randomized, double-blind, placebo-controlled study (n=106) met the primary endpoint (PUL v2.0) and the key secondary cardiac endpoint (LVEF), both achieving statistical significance (p=0.03 and p=0.04, respectively)

Statistical significance was achieved in all type 1 error controlled secondary endpoints

Results demonstrate clinically meaningful and statistically significant skeletal and cardiac benefits, supporting Deramiocel as a potential first-in-class therapy designed to treat Duchenne cardiomyopathy, the leading cause of mortality in Duchenne

Deramiocel maintained a favorable safety and tolerability profile consistent with prior clinical experience

Company plans to submit its response to the Complete Response Letter incorporating HOPE-3 data, following prior alignment with FDA
Conference call and webcast today at 8:00 a.m. ET

Capricor Therapeutics CAPR, a biotechnology company developing transformative cell and exosome-based
therapeutics for the treatment of rare diseases, today announced positive topline results from its pivotal Phase 3 HOPE-3 trial evaluating Deramiocel, the Company’s investigational cell therapy for the treatment of Duchenne muscular dystrophy (DMD).
“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, Ph.D., Chief Executive Officer 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 is a randomized, double-blind, placebo-controlled, Phase 3 clinical trial evaluating Deramiocel in boys and young men with Duchenne muscular dystrophy. The study randomized 106 participants across 20 leading U.S. clinical sites. Participants received intravenous Deramiocel at 150 million cells per infusion or placebo every three months for a
12-month period. The average age of participants was approximately 15 years, and all were on a stable corticosteroid regimen throughout the study. Baseline demographics were well balanced between treatment arms, approximately 90 percent were receiving cardiac medications at baseline, and over 75 percent had a clinical diagnosis of cardiomyopathy.
Deramiocel maintained a favorable safety and tolerability profile consistent with prior clinical experience.

Topline Efficacy Results

Endpoint % Slowing of Progression 3 (Deramiocel vs. Placebo) p-value
Performance of Upper Limb (PUL v2.0)
Total Score¹ (Primary, n=105)
54% p=0.029
Left Ventricular Ejection Fraction (LVEF
%)² (Key Secondary, n=83) 91% p=0.041

¹ n reflects the number of patients in the ITT population with evaluable PUL v2.0 assessments at 12 months.
² n reflects the number of patients in the ITT population with centrally reviewed and evaluable cardiac MRI LVEF assessments at 12 months.
3 Percent slowing is calculated as the treatment difference divided by the placebo change from baseline.

“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, M.D., Distinguished Professor of Physical Medicine & Rehabilitation and Pediatrics at UC Davis Health, and National PI of the HOPE-3 trial. “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.”
“The cardiac findings from HOPE-3 represent a significant advance in the management of Duchenne muscular dystrophy cardiomyopathy,” said Jonathan Soslow, M.D., MSCI, 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 continued, “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.”
We expect that detailed HOPE-3 results will be submitted for presentation at a future scientific meeting and for publication in a peer-reviewed journal.

CAPR Conference Call and Webcast
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