Capricor Therapeutics, Inc. Q3 FY2025 earnings call

• HOPE-3 Phase 3 clinical study of Daramycin for Duchenne muscular dystrophy is near top-line readout, enrolling 105 patients in two cohorts (A from LA facility, B from San Diego commercial GMP facility). Safety data clean with no new safety signals.
• San Diego commercial facility fully operational, CMC issues addressed. Published paper on Daramycin's mechanism and released scientific video.
• Exosome platform's StealthX program in Phase 1 clinical trial for COVID-19 vaccine under NIAID funding, with initial data expected in 2026.

In the third quarter of 2025, Capricor Therapeutics had no revenue, contrasting with $2.3 million in 2024. Research and development expenses excluding stock-based compensation were approximately $18.1 million in Q3 2025 compared to $11 million in Q3 2024, and $54.4 million in 2025 versus $32.8 million in 2024. General and administrative expenses excluding stock-based compensation were $4.1 million in Q3 2025 vs $2.2 million in Q3 2024, and $11.1 million in 2025 vs $5.7 million in 2024. Cash, cash equivalents, and marketable securities totaled approximately $98.6 million as of September 30, 2025.

• Anticipate top-line data from HOPE-3 within the next few weeks.
• Aim to submit HOPE-3 results as a response to the CRL and seek rapid review by the FDA, with eligibility for a priority review voucher if approved by September 30, 2026.
• Motivated to achieve approval for Daramycin as early as 2026 to address the unmet need in Duchenne muscular dystrophy-associated cardiomyopathy.

• Uncertainties in clinical trial outcomes affecting regulatory approval.
• CMC-related risks, though addressed, still pose potential challenges.
• Impact of government shutdown on the exosome platform's Phase 1 clinical trial timeline.

Q: Excited for the upcoming HOPE-3 data. What should we expect from the initial data reporting?

A: Top-line data will include primary and key secondary endpoints, and we'll host a call to explain them.

Q: With consideration of left ventricular ejection fraction as a key secondary, any statistical changes in the study?

A: The study was powered to measure ejection fraction, and we were well overpowered, with strong results from prior trials providing solid power.

Q: How does the SAP designate treatment of LVEF as a secondary endpoint if primary is missed?

A: The SAP is traditional, using alpha for the primary endpoint, and we'll look at totality of data with FDA's regulatory flexibility, potentially using cardiac data if primary is missed.

Q: Run us through the cardiac MRI review procedure?

A: External CRO reviews MRIs for quality, then primary and secondary readers, with adjudication by a third reader if there's disagreement, all independent of time point and treatment group.

Q: On regulatory flexibility regarding Cohort B and review time?

A: Focus on Cohort B due to product comparability concerns, review time for Type 2 resubmission is up to 6 months, with speed dependent on data strength and agency interaction.

Q: Potency assay for Daramycin and FDA guidance?

A: Potency assay developed through multiyear program, using RNA Seq and anti-fibrosis assays, each lot must pass quantification, published and explained on website.

Q: Potential label expansion for Daramycin?

A: Initial focus on cardiomyopathy label, then seek skeletal muscle expansion if primary endpoint is statistically significant and cardiomyopathy label is achieved.

Q: Partnership opportunities for exosomes platform?

A: Waiting for data from NIAID study, then will seek partners for the native protein vaccine using exosome platform, with potential for multivalent vaccines.