Capricor to Present Phase 3 HOPE-3 Data on Deramiocel in Duchenne at MDA 2026

Capricor Therapeutics will deliver a late-breaking oral presentation of Phase 3 HOPE-3 data evaluating Deramiocel in Duchenne muscular dystrophy (DMD) at the 2026 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, taking place March 8–11 in Orlando, Florida.

The company also confirmed that it has submitted the HOPE-3 clinical study report (CSR) to the U.S. Food and Drug Administration to support the ongoing review of its Biologics License Application (BLA).

MDA 2026 Presentation Details

• Date: March 11, 2026
• Time: 2:45 p.m. ET
• Title: Confirmation of Musculoskeletal and Cardiac Benefit in DMD from Deramiocel, an Allogeneic Cell Therapy, in the Phase 3 HOPE-3 Study
• Presenter: Craig McDonald, Professor of Physical Medicine & Rehabilitation and Pediatrics at UC Davis Health and National Principal Investigator of HOPE-3
• Location: Florida 4

Late-breaking status typically reflects the clinical importance and timeliness of new data.

Regulatory Update: CSR Submitted to FDA

The HOPE-3 clinical study report was submitted at the FDA’s request following prior regulatory discussions.

The submission is intended to address items raised in a Complete Response Letter (CRL) and support the continued review of Capricor’s BLA for Deramiocel in DMD. The FDA may assign a new Prescription Drug User Fee Act (PDUFA) target action date as part of the process.

Capricor CEO Linda Marbán stated that the company remains focused on advancing toward a potential approval decision and making the therapy available to patients as efficiently as possible.

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a severe, X-linked genetic disorder caused by the absence of functional dystrophin, a protein essential for muscle integrity.

Key facts:

• Affects approximately 15,000 individuals in the U.S.
• Primarily impacts boys
• Leads to progressive degeneration of skeletal, respiratory, and cardiac muscle
• Cardiomyopathy and heart failure are leading causes of death
• No cure currently exists

Treatment options remain limited, particularly for preserving cardiac function.

About Deramiocel

Deramiocel (CAP-1002) is an allogeneic cell therapy composed of cardiosphere-derived cells (CDCs).

Mechanism of action:

• CDCs secrete extracellular vesicles known as exosomes
• Exosomes modulate immune activity by shifting macrophages toward a healing, anti-inflammatory phenotype
• Designed to preserve both cardiac and skeletal muscle function

Deramiocel has:

• Been studied in more than 250 peer-reviewed publications
• Been administered to over 250 patients across multiple clinical trials

The therapy has received multiple regulatory designations, including:

• Orphan Drug Designation (U.S. and EU)
• Regenerative Medicine Advanced Therapy (RMAT) designation (U.S.)
• Advanced Therapy Medicinal Product (ATMP) designation (EU)
• Rare Pediatric Disease Designation (U.S.), which could qualify Capricor for a Priority Review Voucher upon approval

The HOPE-3 Phase 3 Trial

HOPE-3 (NCT05126758) is a multicenter, randomized, double-blind, placebo-controlled Phase 3 study.

Trial design:

• 106 participants enrolled
• Included both ambulatory and non-ambulatory boys with DMD
• Participants received Deramiocel or placebo every three months
• Four total doses administered during the first 12 months

The late-breaking presentation will highlight data supporting musculoskeletal and cardiac benefits.

What Comes Next?

With HOPE-3 data now set for high-profile presentation and the CSR under FDA review, Capricor enters a pivotal period.

A potential new PDUFA date could clarify the regulatory timeline for Deramiocel — a therapy that, if approved, may offer a novel disease-modifying approach targeting both cardiac and skeletal muscle decline in Duchenne muscular dystrophy.

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