Capricor Therapeutics (CAPR) investor relations material

Executive summary

• Deramiocel's BLA is under FDA review with a PDUFA target date of August 22, 2026, following a complete response to a prior CRL and strong HOPE-3 phase III data; labeling discussions are expected soon.

• Legal action has been initiated to rescind the U.S. distribution agreement with NS Pharma/Nippon Shinyaku due to a flawed pricing structure, with litigation ongoing and the company seeking independent distribution.

• Commercial readiness efforts are accelerating, including hiring a chief commercial officer, building a market access team, and scaling manufacturing capacity to support launch.

• Manufacturing facility in San Diego is FDA-inspected, operational, and expanding to support up to 2,500 patients per year by 2027.

• Pipeline expansion continues, with focus shifting from vaccines to exosome-based therapeutics and plans to pursue additional indications for Deramiocel, including Becker muscular dystrophy and international markets.

Financial highlights

• Cash, cash equivalents, and marketable securities totaled $278.6 million as of March 31, 2026, sufficient to fund operations into Q4 2027, excluding potential product revenue or PRV monetization.

• No revenue recognized for Q1 2026 or Q1 2025.

• Q1 2026 operating expenses were $36.8 million, up from $25 million in Q1 2025, driven by clinical, regulatory, manufacturing, and infrastructure investments.

• Net loss for Q1 2026 was $33.9 million ($0.59/share), compared to $24.4 million ($0.53/share) in Q1 2025.

• R&D expenses increased 45% year-over-year to $27.4 million, and G&A expenses rose 55% to $9.4 million, mainly due to legal and consulting costs.

Outlook and guidance

• Confident in FDA review process and expect labeling discussions soon; commercial launch preparations are underway to ensure rapid patient access post-approval.

• Manufacturing expansion to be fully validated by H1 2027, with stockpiling of commercial doses planned upon label guidance.

• Plans to expand Deramiocel indications to younger DMD patients, Becker muscular dystrophy, and international markets.

• Anticipates eligibility for a priority review voucher upon approval, providing potential non-dilutive capital.

• Expects to spend $100–$125 million in 2026 on Deramiocel, mainly for manufacturing and commercialization prep.