RADNOR, Pa. - Marinus (NASDAQ:MRNS) Pharmaceuticals, Inc. (NASDAQ:MRNS), a company specializing in the development of treatments for seizure disorders, has recently completed enrollment for its global Phase 3 TrustTSC clinical trial. The trial is evaluating the effectiveness of oral ganaxolone in treating seizures associated with tuberous sclerosis complex (TSC) in both children and adults.
The TrustTSC study has involved participants who are highly refractory to current treatments, experiencing significant seizure burdens despite multiple antiseizure medications, including mTOR inhibitors.
Chief Scientific Officer Alex Aimetti expressed gratitude towards the TSC community for their participation and support, highlighting the company's commitment to providing a new therapeutic option for TSC patients.
Chief Medical Officer Joseph Hulihan noted that the Phase 3 trial incorporated a modified titration schedule for ganaxolone, aiming to reduce somnolence-related adverse events and improve treatment response. With less than two percent of participants discontinuing due to these adverse events, the trial may demonstrate an improved tolerability profile for ganaxolone.
Anticipated in the first half of Q4 2024, topline data from the TrustTSC trial will inform the company's planned submission of a supplemental New Drug Application to the U.S. Food and Drug Administration in April 2025, for which Marinus seeks priority review.
Moreover, Marinus announced the issuance of a new method of use patent (U.S. Patent No. 11,980,625) for ganaxolone in the treatment of TSC by the United States Patent and Trademark Office (USPTO), set to expire in 2040. This patent further strengthens Marinus's intellectual property position, being the second of its kind granted for ganaxolone in TSC.
The TrustTSC trial (NCT05323734) is a randomized, double-blind, placebo-controlled study with a primary endpoint focused on the percent change in 28-day TSC-associated seizure frequency. Participants from various regions, including the U.S., Western Europe, Canada, Israel, Australia, and China, were required to have inadequate seizure control despite prior treatments.
Tuberous sclerosis complex is a rare genetic disorder that can lead to non-cancerous tumors, skin abnormalities, and neurological issues, including refractory seizures. It is one of the primary causes of genetic epilepsy, with up to 90% of TSC patients experiencing neurological manifestations.
This news is based on a press release statement from Marinus Pharmaceuticals.
InvestingPro Insights
As Marinus Pharmaceuticals (NASDAQ:MRNS) forges ahead with its TrustTSC clinical trial, the financial health and market sentiment surrounding the company can be critical for investors monitoring the progress. According to InvestingPro data, Marinus boasts a market capitalization of $71.41 million, reflecting the company's valuation amidst its clinical developments.
Despite a challenging financial landscape, with a reported gross profit margin of -261.86% for the last twelve months as of Q1 2024, Marinus has managed to grow its revenue by 30.53% during the same period. This growth indicates a potential upside in the company's ability to generate income, even as it tackles high operational costs.
From an investment standpoint, the InvestingPro Tips highlight that Marinus's stock is currently in oversold territory based on its Relative Strength Index (RSI), which may interest traders looking for entry points. Moreover, the company's liquid assets have exceeded short-term obligations, suggesting a degree of financial stability in the near term.
Still, analysts have tempered expectations, with three analysts revising their earnings downwards for the upcoming period and consensus indicating that profitability is not on the horizon for this year.
For investors seeking a deeper dive into Marinus Pharmaceuticals' investment profile, InvestingPro offers additional insights, with a total of 14 InvestingPro Tips available. These tips provide a more nuanced understanding of the company's financial health and market performance. Interested readers can take advantage of an exclusive offer using the coupon code PRONEWS24 to get an additional 10% off a yearly or biyearly Pro and Pro+ subscription, unlocking further valuable investment information.
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