Rapport Therapeutics (RAPP) Jefferies Global Healthcare Conference 2026 summary

Strategic overview and milestones

• Focus on precision neuroscience with receptor-associated proteins as drug targets, especially TARPγ8 for RAP-219.

• RAP-219 targets focal epilepsy and other indications, with phase III studies now underway.

• Bipolar mania phase II readout and open label extension data for focal onset seizures expected in Q4 this year.

• LAI (long-acting injectable) formulation of RAP-219 in IND-enabling activities, with phase I human PK data targeted for 2027.

Clinical data and efficacy insights

• Phase II data for RAP-219 in focal onset seizures showed 77.8% median reduction in clinical seizures and 71% reduction in electrographic seizures.

• Drug is potent, has a 22-day half-life, and minimal drug-drug interaction risk, supporting use in polypharmacy.

• Washout data showed continued efficacy due to long half-life, with seizure reduction increasing to 90% at week 12.

• Open label extension studies are ongoing, focusing on long-term safety and enrolling patients from phase II and future phase III trials.

Phase III program and trial design

• FOCUS 1 and FOCUS 2 phase III trials are enrolling, with multiple dose levels to inform prescribing.

• Efficacy from phase II is expected to translate to phase III, with no discount assumed for efficacy.

• Studies are powered similarly to contemporary trials, aiming for a 15-20% placebo-adjusted delta.

• Mid dose is the statistical anchor across both studies, with FDA alignment on design.

• Data from phase III expected in roughly three years, balancing speed and patient quality.