Marinus' Oral Ganaxolone Lowers Seizure Frequency In Genetic Disorder

Marinus Pharmaceuticals Inc MRNS announced topline data from its open-label Phase 2 trial evaluating oral ganaxolone in patients with seizures associated with tuberous sclerosis complex (TSC).
The primary endpoint showed a median 16.6% reduction in seizure frequency, with 30.4% of patients achieving a 50% or more seizure reduction.
Patients with focal seizures (n=19) showed a median 25.2% reduction in focal seizure frequency during the trial.
Meaningful 50% responder rates were observed in subgroups of highly refractory seizure patients on concomitant medications cannabidiol (n=12) or everolimus (n=11).
Ganaxolone was generally well-tolerated though one treatment-related serious adverse event of seizure was reported.
Related: Marinus Files US Application For Ganaxolone For Genetic Epilepsy, Inks European Commercialization Pact With Orion.
A global Phase 3 trial of adjunctive ganaxolone in approximately 160 TSC patients will start enrollment during Q4 2021.
The primary endpoint is the percent change in 28-day TSC-associated seizure frequency.
The FDA granted orphan drug designation to ganaxolone for treatment in TSC.
Related content: Benzinga's Full FDA Calendar.
Price Action: MRNS shares closed 0.74% at $12.04 on Tuesday.