CAPRICOR THERAPEUTICS, INC. Q2 FY2025 earnings call

• Linda Marbán discussed the Biologics License Application (BLA) for Deramiocel, noting they received a CRL from the FDA in July but stand by the strength of their submission. The HOPE-3 trial is fully enrolled, blinded, and will not be unblinded until clarity on the approval path is obtained. - The exosome program's StealthX vaccine cleared IND and initiated Phase I trial under NIAID. - Capricor has over $120 million in cash, well-positioned to support operations into late 2026, and is preparing launch readiness activities for Deramiocel if approved.

In the second quarter of 2025, Capricor Therapeutics had revenues of $0 compared to approximately $4 million in the second quarter of 2024. For the first half of 2025, revenues were $0 vs. approximately $8.9 million in the first half of 2024. R&D expenses excluding stock-based compensation in Q2 2025 were approximately $20.1 million vs. ~$11.7 million in Q2 2024; for the first half of 2025, it was ~$36.3 million vs. ~$21.8 million in the first half of 2024. General and administrative expenses excluding stock-based compensation in Q2 2025 were approximately $4 million vs. ~$1.8 million in Q2 2024; for the first half of 2025, it was ~$7 million vs. ~$3.6 million in the first half of 2024. Net loss in Q2 2025 was approximately $25.9 million vs. ~$11 million in Q2 2024; first half 2025 net loss was ~$50.3 million vs. ~$20.8 million in the first half of 2024.

• Waiting for FDA adjudication on the path forward for unblinding HOPE-3 and resubmitting the BLA. - Anticipating outcomes from the upcoming Type A meeting with the FDA to clarify approval requirements. - Potential for approval to bring a priority review voucher and a $80 million milestone payment from Nippon Shinyaku.

• Regulatory risk related to the FDA's review of the Deramiocel BLA and HOPE-3 trial unblinding. - Uncertainty in FDA's approach to rare disease therapies under new leadership. - Potential impact of changes in the FDA review team on the approval process.

Q: Plan for unblinding HOPE-3?

A: We are waiting for adjudication from FDA as to what their requirements will be for HOPE-3, and then we will submit a statistical analysis plan and proceed with unblinding only after that has been accepted.

Q: Visibility on StealthX program?

A: Our StealthX program is exciting. We look forward to the NIA data. While vaccines aren't a core focus, if our candidate meets U.S. government criteria and demonstrates efficacy, it could open business development opportunities.

Q: R&D expense increase?

A: It encompasses ongoing clinical studies (like HOPE-3) and CMC preparations. We'll have more granular items on burn rate as we get more clarity from FDA.