Marinus Pharmaceuticals Reports Topline Ganaxolone Phase 2 Open-Label Results in Tuberous Sclerosis Complex and Receives FDA Orphan Drug Designation

CANNANNEW REPORT

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)–$MRNS #MarinusPharma—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 patients with seizures associated with tuberous sclerosis complex (TSC). The primary endpoint showed a median 16.6 percent reduction in 28-day primary endpoint seizure frequency relative to the four-week baseline period, with 30.4 percent of patients achieving a 50 percent or more seizure reduction. During the trial, patients with focal seizures (n=19) showed a median 25.2 percent reduction in focal seizure frequency. “We believe the totality of the data is encouraging and supports advancing to Phase 3. There was notable activity in focal seizures, a meaningful 50 percent response rate, and consistent results in this refractory patient population, including patients on both cannabidiol and everolimus,” said Joseph Hulihan, M.D., Chief Medical Officer of Marinus. “We look forward to initiating our Phase 3 trial and adding to the body of evidence that supports ganaxolone’s potential as an innovative treatment option…

Excerpt only …
READ MORE BELOW
Source : Marinus Pharmaceuticals Reports Topline Ganaxolone Phase 2 Open-Label Results in Tuberous Sclerosis Complex and Receives FDA Orphan Drug Designation

reposted by Cannabis News World

This site uses Akismet to reduce spam. Learn how your comment data is processed.