Capricor Therapeutics (CAPR) Study Update summary

Regulatory and Clinical Strategy

• Capricor is preparing a Biologics License Application (BLA) for full approval of deramiocel in DMD cardiomyopathy, aiming for a broad, mutation-agnostic label and expedited market entry, with rolling submission starting October 2024 and completion by year-end.

• The BLA is supported by HOPE-2 and HOPE-2 open label extension data, benchmarked against natural history datasets from major medical centers, with strong support from the FDA following recent meetings.

• Deramiocel has received Orphan Drug, RMAT, and Rare Pediatric Disease designations, and potential FDA approval could grant a Priority Review Voucher.

• Manufacturing has transitioned to San Diego with internal GMP capability, and commercial readiness is supported by a partnership with Nippon Shinyaku for US, Japan, and global distribution.

• The company is planning for a potential advisory committee and is confident in the strength and safety of the application.

Clinical Data and Efficacy

• Deramiocel demonstrated statistically significant improvements in cardiac function, including ejection fraction and end-systolic volume, in HOPE-2 and its open label extension, compared to natural history controls.

• Sustained or improved cardiac function was observed over 24–36 months, while natural history data showed a significant decline in similar patients.

• Benefits were seen across a broad DMD population, including those with lower baseline ejection fractions, with earlier intervention yielding stronger results.

• The therapy is safe, well-tolerated, and easy to administer, with over 200 patients treated and minimal side effects reported.

• Deramiocel’s mechanism is immunomodulatory, anti-inflammatory, antifibrotic, and pro-angiogenic, supporting endogenous cardiac repair, with FDA-accepted potency assays.

Market Opportunity and Expansion

• The addressable market includes all DMD patients, as 100% develop cardiomyopathy; early intervention is encouraged, with no age restriction anticipated on the label.

• Rapid adoption is expected among cardiologists due to the lack of existing approved therapies and the favorable safety profile.

• Plans are in place for post-approval label expansion to Becker muscular dystrophy, DMD skeletal muscle myopathy, and other orphan cardiomyopathies.

• The product is expected to be used alongside gene therapies, exon skippers, corticosteroids, and other treatments, with no anticipated drug-drug interactions and potential for synergistic benefits.

• Payers are expected to support reimbursement due to the unmet need and unique position as the only approved therapy for DMD cardiomyopathy.