RADNOR, Pa. - Marinus (NASDAQ:MRNS) Pharmaceuticals, Inc. (NASDAQ:MRNS), a biopharmaceutical company focused on developing treatments for seizure disorders, has received approval from the China National Medical Products Administration (NMPA) for its ganaxolone oral suspension. This medication is indicated for the treatment of epileptic seizures in patients aged two years and older with CDKL5 deficiency disorder (CDD), a rare and severe neurodevelopmental condition.
The approval in China was backed by results from the Phase 3 Marigold trial, which demonstrated a significant median reduction in major motor seizure frequency for patients treated with ganaxolone compared to those on placebo. The trial's primary endpoint was met with a 30.7% reduction versus a 6.9% reduction in the placebo group (p=0.0036). Further, an open-label extension study showed a median 49.6% reduction in seizure frequency over 12 months of treatment.
Prior to this approval, no treatments for CDD had been authorized in China. Dr. Xiaoxiang Chen, CEO of Tenacia Biotechnology, which has partnered with Marinus for the development and commercialization of ganaxolone in Mainland China, Hong Kong, Macau, and Taiwan, emphasized the urgent need for innovative therapies for CDD. He expressed commitment to delivering the first and only treatment option to patients in China.
Kimberly McCormick (NYSE:MKC), Chief Regulatory and Quality Assurance Officer at Marinus, highlighted the approval as a significant advancement for patients with CDD and a reflection of the company's global commitment to addressing rare genetic epilepsies.
Ganaxolone, known as ZTALMY® in the U.S. and European Union, functions as a GABAA receptor modulator in the brain and has been recognized for its anti-seizure effects. Marinus supplies ganaxolone for all global markets under its collaboration agreements.
The information in this article is based on a press release statement from Marinus Pharmaceuticals.
In other recent news, Marinus has completed enrollment for its global Phase 3 TrustTSC clinical trial. This trial evaluates the effectiveness of oral ganaxolone in treating seizures associated with tuberous sclerosis complex in both children and adults. Marinus seeks priority review for its planned submission of a supplemental New Drug Application to the FDA in April 2025, based on the topline data from the TrustTSC trial anticipated in the first half of Q4 2024.
In other developments, Marinus announced the issuance of a new method of use patent for ganaxolone in the treatment of TSC by the United States Patent and Trademark Office, set to expire in 2040. This patent strengthens Marinus's intellectual property position. Furthermore, according to H.C. Wainwright & Co and JMP Securities, Marinus holds a buy rating and a market outperform rating respectively, indicating analysts' positive expectations.
InvestingPro Insights
In light of Marinus Pharmaceuticals' recent regulatory success in China, financial metrics and expert analysis from InvestingPro provide a deeper understanding of the company's market position. Despite the positive news, Marinus Pharmaceuticals is grappling with financial challenges, as indicated by a significant cash burn rate. Investors should note that the company has been quickly depleting its cash reserves, which could impact its ability to sustain operations without seeking additional funding or revenue streams.
Additionally, Marinus Pharmaceuticals' stock performance has been under pressure. Over the past six months, the company's stock has tumbled, with an 85.73% decline. This trend underscores the market's reaction to the company's financial health and future profitability concerns, as analysts do not expect Marinus to be profitable this year. Moreover, with a current market capitalization of $76.36 million and a negative price-to-earnings (P/E) ratio of -0.53, the valuation reflects skepticism about the company's earnings potential in the near term.
On a more positive note, Marinus Pharmaceuticals' liquid assets exceed its short-term obligations, which provides some financial flexibility in managing its day-to-day operations. This is a crucial factor for a biopharmaceutical firm that may face unpredictable expenses and revenue flows due to the nature of drug development and regulatory approvals.
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