Capricor Therapeutics (CAPR) Oppenheimer 35th Annual Healthcare Life Sciences Conference (Virtual) 2025 summary

Technology overview and mechanism of action

• Cardiosphere-derived cells (CDCs) are allogeneic, off-the-shelf cell therapy products derived from explanted human hearts not used for transplantation, ensuring a reliable supply chain.

• CDCs are manufactured using proprietary expansion and culture methods to achieve the therapeutic dose of 150 million cells per infusion.

• Infused CDCs release exosomes that drive cellular repair, reduce inflammation, and improve cardiac and skeletal muscle function.

• Over 300 papers from 55+ labs support the mechanism and bioactivity of CDCs, now known as deramiocel.

• Exosomes from CDCs carry microRNAs with antifibrotic and anti-inflammatory properties, validated by FDA-approved potency assays.

Clinical development and regulatory progress

• Initial trials in adult heart disease underdosed patients, but later studies in Duchenne muscular dystrophy (DMD) used optimized dosing and IV delivery.

• The HOPE-Duchenne and HOPE-2 trials demonstrated improvements in cardiac function and upper limb performance in DMD patients.

• Four years of open-label extension data showed sustained efficacy, leading to positive FDA engagement and a BLA submission at the end of 2024.

• Full approval (not accelerated) is being sought for deramiocel, with a PDUFA date expected by early fall 2025.

• European regulatory progress is ongoing, with no additional clinical trials required by EMA and plans to engage further in 2025.

Commercial partnerships and financial outlook

• Partnership with Nippon Shinyaku (NS) covers the U.S. and Japan, with potential expansion to Europe.

• NS will pay an $80 million milestone at drug approval, and the royalty rate is between 30% and 50%.

• A Priority Review Voucher (PRV) is expected upon approval, potentially worth $150 million.

• Recent fundraising ensures operating capital through 2027, excluding additional NS payments.

• Academic royalties are low single-digit percentages and are considered negligible.