Radiprodil

GRIN Therapeutics' Astroscape study assesses radiprodil's efficacy and safety in TSC and FCD type II patients. Radiprodil, an NMDA receptor modulator, targets GluN2B overexpression in these conditions. The open-label trial includes 20 TSC and 10 FCD type II patients, focusing on seizure control and non-seizure outcomes. Challenges include condition heterogeneity and unmet needs for targeted therapies. The study's primary completion is estimated for July 2025.

Phase 1 Honeycomb study data showed radiprodil (GRIN Therapeutics) was well-tolerated and significantly reduced seizure frequency in GRIN-related neurodevelopmental disorder patients, supporting further development. 15 patients with gain-of-function variants in GRIN1, GRIN2A, GRIN2B, or GRIN2D were analyzed, with a median 86% reduction in seizure frequency. 71% of patients experienced at least a 50% reduction in countable motor seizures, and 1 patient was seizure-free. Clinician and caregiver assessments indicated clinical improvement. The study aims to progress to Phase 3 development.