Core Insights - Rapport Therapeutics announced that the Phase 2a clinical trial of RAP-219 met its primary endpoint, showing a statistically significant reduction in long episodes (LEs) in patients with drug-resistant focal onset seizures [1][4][6] - The company plans to advance RAP-219 into two Phase 3 pivotal trials in Q3 2026, following the promising results from the trial [1][11] Efficacy Results - In the 8-week treatment period, 85.2% of patients achieved a ≥30% reduction in LEs from baseline (p<0.0001) [4][5] - 72.0% of patients achieved a ≥50% reduction in clinical seizures from baseline (p<0.0001) [4][7] - 24% of patients achieved seizure freedom during the treatment period (p<0.0001) [4][6] Trial Design - The Phase 2a trial was a proof-of-concept, multi-center, open-label study involving 30 patients with drug-resistant focal onset seizures [3][10] - Patients received escalating doses of RAP-219 over an 8-week period, starting with 0.75 mg daily for 5 days, followed by 1.25 mg daily [3] Safety and Tolerability - RAP-219 was generally well tolerated, with 78.5% of treatment-emergent adverse events (TEAEs) being mild [9][12] - No serious adverse events were reported, and the discontinuation rate due to TEAEs was low at 10% [12] Future Development Plans - The company plans to initiate an open-label long-term safety trial by the end of 2025 for patients who participated in the Phase 2a trial [13] - Rapport is also developing a long-acting injectable formulation of RAP-219 to improve patient adherence [14] Broader Applications - Beyond epilepsy, RAP-219 is being evaluated in a Phase 2 trial for bipolar mania, with topline results expected in the first half of 2027 [15]
Rapport Announces Positive Topline Results from Phase 2a Clinical Trial of RAP-219 in Patients with Focal Onset Seizures