Phase III trial of CCD 1042 (ganaxolone) in focal onset seizures fails to meet endpoint- Marinus Pharmaceuticals

Marinus Pharmaceuticals has announced top-line results from its Phase III clinical trial of CCD 1042 (ganaxolone) in adults with drug-resistant focal onset seizures. In this trial, ganaxolone did not meet the primary endpoint of demonstrating a statistically significant difference from placebo. Consistent with previously conducted studies, ganaxolone was generally safe and well tolerated. The study missed its primary endpoint of percent change in the 28-day seizure frequency from baseline. The median percent reduction of focal onset seizures in the ganaxolone group was 21.28% compared to 10.25% with placebo during the titration and the 12-week treatment period. The safety profile of ganaxolone in this study was consistent with previously conducted studies.

Marinus plans to discontinue its program in adult focal onset seizures and focus its efforts on advancing ganaxolone in status epilepticus and pediatric orphan indications.

Comment: Ganaxolone is a CNS-selective GABAA modulator being developed in three different dose forms (IV, capsule, and liquid) intended to maximize therapeutic reach to adult and pediatric patient populations in both acute and chronic care settings.