Press release
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MARINUS PHARMACEUTICALS NEWS RELEASE Marinus Pharmaceuticals Reports Topline Ganaxolone Phase 2 Open - Label Results in Tuberous Sclerosis Complex and Receives FDA Orphan Drug Designation 8/17/2021 Primary endpoint showed a median 16.6 % reduction in 28 - day seizure frequency relative to baseline in tuberous sclerosis complex ( TSC ) patients ( n = 23 ) Secondary endpoint of 50 % responder rate at 30.4 % , consistent with the Marigold Phase 3 CDKL5 deficiency disorder trial results 25.2 % reduction was observed in focal seizures ( n = 19 ) , the most common seizure type in TSC patients Meaningful 50 % responder rates in subgroups of highly refractory seizure patients on concomitant medications cannabidiol ( n = 12 ) or everolimus ( n = 11 ) Phase 3 planning for TrustTSC trial ongoing with first patient expected to be enrolled in Q4 2021 U.S. Food and Drug Administration ( FDA ) has granted orphan drug designation to ganaxolone in TSC Conference call and webcast to be held today at 4:30 p.m. ET RADNOR , Pa .-- ( BUSINESS WIRE ) -- Marinus Pharmaceuticals , Inc. ( Nasdaq : MRNS ) , a pharmaceutical company dedicated to the development of innovative therapeutics to treat seizure disorders , today announced topline data from its open - label Phase 2 trial evaluating safety and efficacy of adjunctive oral ganaxolone treatment in 23 1