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Rapport Therapeutics’ Epilepsy Drug Delivers Strong Phase 2a Results as Stock Leaps Over 200%

The company now targets an FDA end‑of‑Phase‑2 meeting in Q4 2025, with two Phase 3 trials slated to begin in Q3 2026.

Overview

  • RAP-219 met its primary endpoint in an open-label Phase 2a study, showing a statistically significant reduction in electrographic long episodes over eight weeks versus baseline.
  • Clinical seizures fell substantially, including a reported 78% median decline, 72.0% of patients achieving at least a 50% reduction, and 24% reaching seizure freedom during treatment.
  • Safety findings indicated the drug was generally well tolerated, with mostly mild or moderate adverse events and a low discontinuation rate, and no serious events reported by study investigators.
  • The 30-patient trial in drug‑resistant focal onset seizures informs a plan for two pivotal studies using traditional clinical seizure endpoints after an FDA end‑of‑Phase‑2 meeting.
  • RAPP shares jumped about 210% in premarket trading on heavy volume, as the company also mapped a long‑term safety study by year‑end 2025 and continued work on a long‑acting injectable and additional indications including bipolar mania.