Marinus Pharmaceuticals Shares Tuberous Sclerosis Complex Program Updates

Marinus Pharmaceuticals, Inc., a company focused on developing innovative treatments for seizure disorders, has announced the completion of patient enrollment for their Phase 3 TrustTSC trial. This global trial is evaluating the efficacy of oral ganaxolone in treating seizures related to tuberous sclerosis complex (TSC) in both children and adults. Topline data from this study is expected to be released in the first half of the fourth quarter of 2024.

Alex Aimetti, Ph.D., Chief Scientific Officer at Marinus Pharmaceuticals, expressed satisfaction with the progress of the TrustTSC trial. He emphasized that the trial has enrolled patients who suffer from severe, treatment-resistant TSC-related seizures, despite being on multiple antiseizure medications. Many participants have been exposed to mTOR inhibitors. Aimetti extended gratitude to the TSC community for their support and participation, highlighting the company’s commitment to providing a new therapeutic option for TSC patients.

Joseph Hulihan, M.D., Chief Medical Officer of Marinus Pharmaceuticals, provided additional insights into the trial. He revealed that based on the results from Marinus’ earlier Phase 2 TSC trial, the dosing schedule for ganaxolone was adjusted in the Phase 3 study. The goal was to minimize somnolence-related adverse effects and enhance patient response to the treatment. The trial has shown promising improvements in tolerability with less than two percent of participants discontinuing due to somnolence, which could potentially lead to significant efficacy outcomes.

Alongside the trial's progress, Marinus Pharmaceuticals has also received a new method of use patent for ganaxolone in treating TSC from the United States Patent and Trademark Office (USPTO). This patent, numbered 11,980,625, is the second of its kind for ganaxolone in TSC and is valid until 2040. This strengthens Marinus' intellectual property portfolio, ensuring a more secure market position.

The TrustTSC trial, registered under NCT05323734, is a global Phase 3 randomized, double-blind, placebo-controlled study. It involves adjunctive oral ganaxolone treatment for TSC-related epilepsy in both children and adults. Participants undergo a four-week baseline period, followed by a 16-week double-blind treatment phase. Depending on their response, they may enter a two-week down titration period or a four-week crossover period for those opting for a long-term open-label phase. The primary endpoint of the study is the percentage change in 28-day TSC-associated seizure frequency.

Participants were enrolled from multiple regions, including the United States, Western Europe, Canada, Israel, Australia, and China. Eligibility criteria required participants to have inadequate seizure control and previous treatment with at least two antiseizure medications, with some exceptions for younger patients. Importantly, this trial is the first to allow participants to continue using a full range of concomitant medications, such as everolimus and cannabidiol.

Tuberous sclerosis complex (TSC) is a rare genetic disorder resulting from mutations in the TSC1 or TSC2 genes. It manifests as non-cancerous tumors, skin abnormalities, and significant neurological issues, including refractory seizures and developmental delays. TSC is a leading cause of genetic epilepsy, often presenting in infancy as focal seizures or infantile spasms, with up to 90% of patients experiencing neurological symptoms.

Marinus Pharmaceuticals is dedicated to developing and commercializing innovative therapies for seizure disorders. The company introduced the FDA-approved medication ZTALMY® (ganaxolone) oral suspension CV in the U.S. in 2022, and continues to explore the potential of ganaxolone in both intravenous and oral formulations to benefit patients of all ages in acute and chronic care settings.