Capricor Therapeutics (CAPR) Corporate presentation summary

Deramiocel program and regulatory status

• Deramiocel is an investigational allogeneic cardiosphere-derived cell therapy for Duchenne muscular dystrophy (DMD); Phase 1, 2 and 3 studies reported positive safety and efficacy findings.

• HOPE-3 met its 12-month primary endpoint on PUL total score (p=0.029), with a 4.55% mean treatment difference; results were published in The Lancet.

• The BLA remains under active FDA review; PDUFA target date extended from Aug. 22 to Nov. 22, 2026, following a major amendment with HOPE-3 24-month open-label extension data and additional analyses.

• The refined target indication focuses on upper-limb impairment in patients older than 9 years; further FDA engagement is anticipated during review.

Clinical evidence and safety

• HOPE-3 was a randomized, double-blind, placebo-controlled Phase 3 study of 106 participants at 20 U.S. sites; Deramiocel or placebo was infused intravenously quarterly.

• HOPE-3 showed a 4.55-point PUL 2.0 total-score least-squares mean difference (95% CI 0.47–8.63; p=0.029) and a 12.36-point difference in LVEF for the cardiomyopathy population (95% CI 2.35–22.38; p=0.0165).

• Across earlier trials, HOPE-2 met its PUL mid-level primary endpoint (p=0.01; 2.6-point absolute treatment difference); its five-year extension showed less PUL total-score decline versus matched external controls.

• In HOPE-3, treatment-related or administration-related adverse events were mostly mild or moderate; no grade 3–5 events occurred in the Deramiocel arm, and 1.9% had serious events related to treatment or administration.

• Deramiocel is intended as a potential first-in-class DMD therapy that can be used alongside gene therapies, corticosteroids, exon-skipping therapies and standard cardiac medications.

Commercial and operational readiness

• Estimated U.S. DMD prevalence is 15,000, with 150,000–200,000 patients worldwide; the global market is estimated at $27 billion by 2030.

• The presentation cites more than 1,300 IV infusions in about 200 DMD patients and describes a favorable safety profile; U.S. commercial capabilities and patient support are being built.

• Cash balance was $238 million, with runway through 2027 excluding product-sale revenue and potential PRV monetization; 58.1 million shares outstanding and about 350 FTEs.

• A potential priority review voucher sale is estimated at $150–200 million; the manufacturing facility completed an FDA pre-license inspection in 2025.

• Deramiocel is produced from transplant-qualified human hearts and cryopreserved; the process supports 150 million-cell doses, with four production cycles per year.