Marinus Pharmaceuticals Completes Enrollment for Phase 3 Trial on Tuberous Sclerosis Complex

Marinus Pharmaceuticals

RADNOR, PA — Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS) recently completed enrollment for its global Phase 3 TrustTSC trial, which is evaluating the efficacy of oral ganaxolone in treating seizures associated with tuberous sclerosis complex (TSC) in both 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, highlighted improvements made in the trial based on previous findings. “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.”

The company anticipates releasing topline data from the TrustTSC trial in the first half of the fourth quarter of 2024. Marinus plans to submit a supplemental New Drug Application to the U.S. Food and Drug Administration by April 2025, with a priority review request.

In a related development, the United States Patent and Trademark Office (USPTO) has granted Marinus a new method of use patent (U.S. Patent No. 11,980,625) for ganaxolone in treating TSC, expiring in 2040. This is the company’s second method of use patent for ganaxolone in TSC, further strengthening its intellectual property position.

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The TrustTSC trial is a global, randomized, double-blind, placebo-controlled clinical trial. It examines the effects of adjunctive oral ganaxolone treatment in children and adults with TSC-related epilepsy. The trial consists of a four-week baseline period, followed by a 16-week double-blind treatment phase, and then either a two-week down titration period or a four-week crossover period for those entering the long-term open-label phase. The primary endpoint is the percent change in 28-day TSC-associated seizure frequency.

TrustTSC operates in multiple locations, including the U.S., Western Europe, Canada, Israel, Australia, and China. To be eligible, participants needed inadequate seizure control and must have been treated with at least two prior antiseizure medications, although there are exceptions for younger participants. Notably, it is the first controlled trial in TSC to allow participants to use a full range of concomitant medications, including everolimus and cannabidiol.

Marinus Pharmaceuticals continues to make strides in developing treatments for TSC, aiming to provide new options for those burdened by this challenging condition.

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