Rapport Therapeutics (RAPP) 44th Annual J.P. Morgan Healthcare Conference summary

Company vision and platform

• Aims to be a leader in precision neuroscience, leveraging receptor-associated protein technology for targeted therapies.

• Created through collaboration with Third Rock and J&J, bringing deep institutional knowledge.

• Focuses on developing highly specific compounds for neurological conditions, improving efficacy and tolerability.

Lead program RAP-219 and clinical progress

• RAP-219, a forebrain-restricted TARP gamma-8 AMPA modulator, showed robust phase 2 data in focal onset seizures.

• Pivotal studies for RAP-219 to begin in Q2 2026, following positive FDA interaction.

• Expanding indications to primary generalized tonic-clonic seizures and bipolar mania, with proof-of-concept data for bipolar expected in H1 2027.

• Developing a long-acting injectable (LAI) formulation, with first human PK data expected in 2027.

Market opportunity and unmet needs

• Focal onset seizures: 1.8M patients, $15B market; primary generalized tonic-clonic seizures: 800K patients, $7B market; bipolar mania: 1.5M patients, $40B market.

• High unmet need due to limited efficacy and tolerability of current CNS drugs; RAP-219 aims to address these gaps.

• LAI formulation expected to extend clinical utility and revenue durability.