Rapport Therapeutics (RAPP) Stifel 2025 Healthcare Conference summary

Company overview and scientific approach

• Focuses on precision neuroscience using receptor-associated proteins to achieve targeted modulation of specific brain receptors and sensory neurons.

• Lead program, RAP-219, is a TARP gamma-8 AMPA modulator showing promise in epilepsy and being explored for bipolar mania and as a long-acting injectable.

Clinical development and regulatory plans

• Proof-of-concept study in epilepsy demonstrated robust efficacy and tolerability, with a 78% median seizure reduction and 24% seizure freedom rate.

• Two parallel phase 3 registrational studies are planned, with FDA end-of-phase-two meeting expected by year-end and phase 3 initiation guided for Q3 2026 or earlier.

• Global phase 3 trials will be conducted, with a focus on quality patient enrollment and leveraging sites with high interest.

Safety and efficacy insights

• Tolerability profile in phase 2 was strong, with most adverse events mild to moderate and a 10% discontinuation rate.

• No concerning psychiatric adverse events observed; main tolerability issues with comparator drugs relate to sedation and gait impairment.

• Efficacy signal in highly refractory patients is expected to translate well to broader phase 3 populations.