Rapport Therapeutics (RAPP) Jefferies London Healthcare Conference 2024 summary

Company overview and scientific platform

• Focused on precision neuroscience using receptor-associated proteins to develop targeted small molecules.

• Formed through a partnership with Third Rock and J&J, licensing assets and a discovery platform.

• Core scientific team from J&J joined, supporting continuity and expertise.

• Lead program RAP-219 targets AMPA receptors in the forebrain, aiming for best-in-class potential in focal epilepsy and other indications.

• Active discovery programs with two late-stage candidates approaching development nomination.

Lead program RAP-219: Mechanism and clinical profile

• RAP-219 modulates AMPA receptors with high precision, avoiding hindbrain effects and improving tolerability.

• Demonstrates high receptor occupancy and seizure suppression without sedation or motor impairment in preclinical models.

• Once-daily dosing, long half-life, and minimal drug-drug interactions enhance patient convenience.

• Dosing schema allows rapid achievement of therapeutic levels while minimizing adverse events.

• Phase 1 studies showed on-target pharmacology and manageable, transient side effects.

Clinical development and trial design

• Phase 2 proof-of-concept study in focal epilepsy patients with RNS devices, using long episode (LE) biomarker for efficacy.

• LE reduction of 30%-40% predicts at least 50% reduction in clinical seizures.

• Study enrolls 20 patients, with an 8-week treatment period and objective biomarker endpoints.

• Open-label design chosen for efficiency and confidence in objective biomarker data.

• Clinical seizure data also collected, though study is powered for LE reduction.