Capricor Therapeutics (CAPR) investor relations material

Executive summary

• BLA for Deramiocel accepted by FDA with a PDUFA target action date of August 22, 2026, marking a major regulatory milestone for potential Duchenne muscular dystrophy (DMD) therapy approval.

• HOPE-3 pivotal phase III trial met primary and all key secondary endpoints, showing significant efficacy in both skeletal and cardiac muscle function stabilization in DMD patients.

• Late-breaking HOPE-3 data presented at MDA 2026 demonstrated additional cardiac and functional benefits, including reduced myocardial fibrosis and improved daily living activities.

• Deramiocel demonstrated a strong safety profile with over 800 infusions and long-term tolerability in open-label extension studies.

• Commercial launch preparations are underway, with the San Diego GMP facility operational and FDA-inspected.

Financial highlights

• Cash, cash equivalents, and marketable securities totaled $318.1 million at year-end 2025, up from $151.5 million at year-end 2024.

• No revenue recognized in Q4 or full year 2025, compared to $11.1 million and $22.3 million, respectively, in 2024, due to completion of prior milestone recognition.

• Operating expenses rose to $29.2 million in Q4 2025 (from $18.8 million in Q4 2024) and $108.1 million for the year (from $64.8 million in 2024), driven by clinical, regulatory, and manufacturing investments.

• Net loss for Q4 2025 was $30.2 million (vs. $7.1 million in Q4 2024); full-year net loss was $105 million (vs. $40.5 million in 2024).

• Total assets at year-end 2025 were $355.9 million, liabilities $50.2 million, and stockholders' equity $305.8 million.

Outlook and guidance

• Cash runway expected to fund operations into Q4 2027, excluding potential product revenue or monetization of a priority review voucher.

• Commercial launch preparations ongoing, with focus on market access, reimbursement, and physician education.

• Manufacturing expansion to support up to 2,500 patients per year by late 2027, with further scale-up planned.

• Potential for global expansion, with regulatory engagement in Europe and Japan and pipeline development for Becker muscular dystrophy.