Rapport Therapeutics (RAPP) TD Cowen 45th Annual Healthcare Conference summary

Company strategy and leadership

• Focused on precision neuroscience using receptor-associated proteins, with RAP-219 as the lead program in development.

• Appointed a new Chief Medical Officer, Jeff Sevigny, with extensive neuroscience experience.

• Created through a partnership with Johnson & Johnson and Third Rock, bringing in key scientists and J&J as an investor.

Lead program RAP-219: Mechanism and clinical progress

• RAP-219 targets TARP gamma-8, an AMPA receptor-associated protein localized in forebrain structures, avoiding hindbrain-related side effects.

• Completed four phase I trials, confirming receptor occupancy and a favorable safety profile.

• Phase 2A proof of concept study in focal onset seizures is ongoing, with data expected in Q3 2024.

• The compound's long half-life allows for gradual accumulation, reducing adverse events.

Clinical trial design and endpoints

• The epilepsy trial uses the RNS device to measure long episodes, a biomarker for clinical seizures.

• A 30% reduction in long episodes is targeted, correlating with a >50% reduction in clinical seizures.

• The study enrolls about 20 refractory focal epilepsy patients, with both electrographic and clinical seizure data collected.

• The trial design enables faster proof of concept data generation, aiming for phase 3 readiness within a year.