Rapport Therapeutics (RAPP) Goldman Sachs 47th Annual Global Healthcare Conference 2026 summary

Key clinical program updates

• Phase III studies in focal-onset seizures are actively enrolling globally, with rolling site activation and a partnership in China to aid recruitment and efficiency.

• Phase II data for RAP-219 in focal-onset seizures showed a 78% median reduction in seizure frequency and a 24% seizure freedom rate, supporting confidence in phase III trial design.

• Phase II study in bipolar mania is nearing completion, with top-line data expected in Q4 this year, ahead of previous guidance.

• Open label extension study for RAP-219 is enrolling, with initial safety and efficacy data expected in Q4, focusing on long-term safety exposure.

• Primary generalized tonic-clonic seizure trial and long-acting injectable PK study are planned to start in 2027.

Trial design, regulatory, and operational strategy

• Conservative powering of phase III trials aims to ensure high probability of success and sufficient safety exposure for registration.

• Placebo response in phase III is managed through rigorous patient selection, eDiary training, and collaboration with The Epilepsy Study Consortium.

• FDA is receptive to global data accumulation, including from China, with no anticipated regulatory issues.

• Regulatory strategy for the long-acting injectable will be determined after PK data in 2027, with potential for either bridging or full efficacy studies.

Commercial and strategic outlook

• RAP-219 is positioned as a best-in-class anti-seizure medication with a novel mechanism, strong efficacy, and favorable tolerability, supporting broad use by both epileptologists and general neurologists.

• Market research indicates potential for second-line utilization, which could significantly boost sales trajectory.

• Long-acting injectable formulation could extend IP runway into the late 2040s and transform patient adherence and asset value.

• Partnership with Tenacia in China is expected to accelerate global trial recruitment and future commercialization.