Rapport Therapeutics (RAPP) Leerink Global Healthcare Conference 2026 summary

Scientific and clinical highlights

• Lead program targets TARP γ8, an auxiliary protein of the AMPA receptor, with high selectivity for brain regions involved in focal-onset seizures, minimizing off-target effects and improving the therapeutic index.

• Phase II proof-of-concept study in refractory focal-onset seizures showed a 72% reduction in electrographic seizures and a 78% median reduction in clinical seizures, with a 24% seizure freedom rate.

• Innovative use of the NeuroPace RNS System as a diagnostic tool enabled objective, biomarker-driven efficacy assessment, allowing for smaller, faster studies.

• Adverse events were mostly mild to moderate, with a 10% discontinuation rate; no maximum tolerated dose was reached, and the drug demonstrated a favorable tolerability profile.

• Phase III program will include two global pivotal studies, each enrolling 320–330 subjects, exploring three dose levels with efficient titration schemas.

Strategic partnerships and pipeline expansion

• Entered a $20 million upfront partnership with Tenacia for Greater China rights to RAP-219, with $308 million in potential milestones and royalties; Tenacia will fund all China development.

• China is now open for patient recruitment in global phase III trials, expected to accelerate enrollment.

• Pipeline includes upcoming studies in primary generalized tonic-clonic seizures (PGTCs) starting in 2027, and an ongoing trial in bipolar mania with data expected in H1 2027.

• IND-enabling activities for a pain and migraine asset are underway, with phase I planned for next year.

• Cash runway extends into H2 2029, supporting completion of key clinical milestones across multiple programs.

Long-acting injectable (LAI) and intellectual property

• RAP-219’s long half-life, potency, and lack of drug-drug interactions make it uniquely suited for LAI development, targeting monthly or longer dosing intervals.

• LAI formulation is in IND-enabling studies, with first human PK results expected in 2027; initial target is 30-day dosing, with potential for 60–90 days or longer.

• LAI could extend patent protection to 2048–2049, enhancing revenue durability and premium pricing opportunities.

• All formulation work is managed in-house, with manufacturing by CDMOs and careful selection of excipients for safety and regulatory compliance.

• LAI is expected to be transformational for epilepsy patients, addressing the risk of missed doses and breakthrough seizures.