Marinus Pharmaceuticals Wraps Phase 3 Trial Enrollment for Tuberous Sclerosis Complex Treatment, Targets New Drug Application in 2025 Following Phase 3 Seizure Treatment Trial

Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat seizure disorders, today announced completion of enrollment in the global Phase 3 TrustTSC trial evaluating oral ganaxolone for the treatment of seizures associated with tuberous sclerosis complex (TSC) in children and adults.

"We are pleased to announce we have completed enrollment in our pivotal Phase 3 trial in tuberous sclerosis complex," said Alex Aimetti, Ph.D., Chief Scientific Officer at Marinus Pharmaceuticals. "The TrustTSC study enrolled highly refractory TSC patients experiencing a significant seizure burden despite treatment with multiple antiseizure medications, including the majority of patients having exposure to mTOR inhibitors. We would like to thank the TSC community for their support, trust and participation as we strive to bring a new treatment option to TSC patients."

Joseph Hulihan, M.D., Chief Medical Officer of Marinus added, "Based on the findings in Marinus' Phase 2 TSC trial, the titration schedule of ganaxolone was modified in the Phase 3 TrustTSC trial with the goal of reducing the frequency of somnolence-related adverse events and improving patients' response to treatment. With less than two percent of TrustTSC participants discontinuing due to somnolence-related adverse events, this improvement in tolerability may have the potential to yield meaningful efficacy outcomes in this patient population."

Topline data from the TrustTSC trial is anticipated in the first half of the fourth quarter of 2024. Marinus is targeting submission of a supplemental New Drug Application to the U.S. Food and Drug Administration in April 2025 with priority review requested.

Marinus also announced that the United States Patent and Trademark Office (USPTO) has granted a new method of use patent (U.S. Patent No. 11,980,625) for ganaxolone in the treatment of TSC, expiring in 2040. This is Marinus' second method of use patent granted for ganaxolone in TSC, further strengthening the Company's intellectual property position.