Prime Medicine (PRME) Earnings Call Presentation

Pipeline and Programs - Prime Medicine expects to file IND and/or CTA for Wilson's Disease program (PM577) in the first half of 2026 and announce initial clinical data in 2027[10] - Prime Medicine anticipates filing IND and/or CTA for AATD program in mid-2026 and expects initial clinical data in 2027[10] - Prime Medicine will share in vivo proof-of-concept data in CF and initiate IND-enabling studies[10] Technology and Safety - Prime Editing is designed to edit, correct, insert, and delete DNA sequences in any target tissue[12] - Prime Editing has a highly differentiated safety profile with no off-target activity detected in any lead program, including no detectable double-strand breakage, off-target edits, bystander edits, or large deletions/translocations[14, 15, 16, 17, 25] Partnerships and Financials - Prime Medicine entered into a strategic research collaboration and license agreement with Bristol Myers Squibb (BMS) to develop and commercialize multiple ex vivo T cell products in immunology and oncology, receiving $110 million upfront and potentially over $3.5 billion in milestones plus royalties[34, 85] - Prime Medicine entered into an agreement with the CF Foundation for up to $15 million to support the development of Prime Editors for Cystic Fibrosis[33, 77] - Prime Medicine held cash equivalents, investments, and restricted cash of $158.3 million as of March 31, 2025, providing a cash runway into the first half of 2026[98] Liver Programs - In Wilson's Disease, Prime Editors efficiently correct the two most prevalent disease-causing mutations, H1069Q and R778L, reducing liver copper by approximately 75%, decreasing urinary copper by about 80%, and increasing fecal copper by around 100% in vivo[52, 56] - In AATD, Prime Editor efficiently corrects the mutation resulting in M-AAT protein restoration in vivo, with the potential to correct mutated protein back to wild type without bystander or off-target edits[69, 71] - Prime Medicine achieved >70% hepatocyte editing using Prime Editors delivered with universal liver LNPs in humanized mice[37]