Marinus' Oral Ganaxolone Lowers Seizure Frequency In Genetic Disorder

• Marinus Pharmaceuticals Inc (NASDAQ: 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.

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