Rapport Therapeutics, Inc. (RAPP) reported that its investigational therapy RAP-219 continued to deliver clinically meaningful benefits even after dosing ended, with patients experiencing a 90% median reduction in seizures during the first four weeks of follow-up.
Tackling Focal Onset Seizures
Focal onset seizures (FOS) are common forms of epilepsy, often resistant to standard treatments. Symptoms can significantly disrupt daily life, and existing therapies may not fully control episodes. RAP-219, designed as a precision small molecule therapy, aims to address these limitations by targeting specific brain regions linked to seizure activity.
Phase 2a Study Results
The Phase 2a study enrolled 30 adults with drug-resistant focal onset seizures. Participants received RAP-219 for eight weeks, followed by an eight-week observation period without treatment. Results showed sustained seizure control due to the drug's long half-life of approximately 22 days. In weeks 9-12, patients achieved a 90% median reduction in clinical seizures compared to baseline, while weeks 13-16 maintained a 59% reduction. Long episodes, an objective biomarker of epileptiform activity, also showed consistent improvement.
Safety Profile
RAP-219 was generally well tolerated, with only mild treatment-related side effects reported. Serious adverse events occurred but were not considered drug-related by investigators.
Next Steps in Development Two pivotal Phase 3 trials, FOCUS 1 (RAP-219-FOS-301) and FOCUS 2 (RAP-219-FOS-302), are planned to begin in the second quarter of 2026, to further evaluate RAP-219 in focal onset seizures. An open-label extension study is already underway to monitor long-term safety.
Beyond epilepsy, Rapport is conducting a Phase 2 trial evaluating RAP-219 in bipolar mania, with topline results expected in the first half of 2027, and a Phase 3 trial in primary generalized tonic-clonic seizures is expected to begin in the first half of 2027. Additionally, Rapport continues development of a long-acting injectable (LAI) formulation of RAP-219, with Phase 1 initial PK results expected in 2027.
Precision Approach
Rapport's approach leverages selective targeting of TARP?8, a protein expressed in brain regions where seizures originate, while minimizing effects in areas associated with intolerable side effects. The company believes this precision strategy could position RAP-219 as a differentiated, best-in-class therapy for neurological disorders.
RAPP has traded between $7.73 and $42.27 over the past year. The stock closed Tuesday's trading at $36.95, down 0.51%.
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