RADNOR, Pa. - Marinus (NASDAQ:MRNS) Pharmaceuticals, Inc. (NASDAQ:MRNS), a biopharmaceutical company focused on developing treatments for seizure disorders, has announced that its Phase 3 trial for ganaxolone did not meet the primary endpoint. The TrustTSC trial aimed to reduce seizure frequency in patients with tuberous sclerosis complex (TSC), but the results did not achieve statistical significance.
The trial, which included 129 children and adults with TSC-related epilepsy, showed a median reduction in 28-day seizure frequency of 19.7% for ganaxolone compared to 10.2% for placebo, with a p-value of 0.09. Despite the numerical favor towards ganaxolone, the lack of statistical significance means the results are unlikely to support a supplemental New Drug Application (sNDA) filing.
Ganaxolone was generally well-tolerated in the trial, with somnolence being the most frequent adverse event reported. Marinus noted that the safety profile was consistent with previous studies.
In light of these results, Marinus has decided to discontinue further clinical development of ganaxolone and is implementing cost reduction measures, including workforce reductions. The company will continue to support ZTALMY® (ganaxolone) oral suspension CV, which is FDA-approved for seizures associated with CDKL5 deficiency disorder.
Additionally, Marinus has engaged Barclays to explore strategic alternatives to maximize shareholder value. This process could lead to various outcomes, including potential transactions, although there is no certainty as to the result or timing of this exploration.
The TrustTSC trial was a global, randomized, double-blind, placebo-controlled study conducted across several countries. Participants were required to have inadequate seizure control despite treatment with at least two prior antiseizure medications.
Tuberous sclerosis complex is a rare genetic disorder that can cause non-cancerous tumors, skin abnormalities, and neurological issues, including refractory seizures. It is a leading cause of genetic epilepsy, with neurologic manifestations seen in up to 90% of TSC patients.
This announcement has been made based on a press release statement from Marinus Pharmaceuticals.
In other recent news, Marinus Pharmaceuticals has reported promising results from its Phase 3 RAISE trial on the seizure treatment drug, ganaxolone. The trial revealed a significant reduction in seizure cessation time and a decreased need for additional treatment within 24 hours. Despite not achieving statistical significance for one of its co-primary endpoints, the trial demonstrated the potential of IV ganaxolone as a rapid and effective treatment for refractory status epilepticus.
In addition to these developments, Marinus secured a new U.S. patent for its epilepsy drug, ZTALMY. The patent, set to expire in September 2042, covers treatment for various epilepsy disorders. The U.S. Patent and Trademark Office Patent Trial and Appeal Board also upheld Marinus Pharmaceuticals' patent related to the use of ganaxolone, ensuring the company's exclusive rights to the drug.
The company's net product revenues increased to $8 million for the second quarter, primarily due to ZTALMY. Marinus is planning for the potential launch of ZTALMY for tuberous sclerosis complex in the second half of 2025. Despite a net loss before income taxes of $35.8 million for the quarter, the company aims to meet its revenue guidance for 2024, targeting net product revenues between $33 million and $35 million.
Analysts have also been following Marinus closely. TD Cowen maintained its Buy rating on the company, while Oppenheimer upgraded the stock to Outperform. Both firms expressed confidence in the trial design and potential efficacy of ganaxolone. These are some of the recent developments at Marinus Pharmaceuticals.
InvestingPro Insights
In light of Marinus Pharmaceuticals' recent setback with its Phase 3 trial for ganaxolone, investors may be seeking a clearer picture of the company's financial health. According to InvestingPro data, Marinus has a market capitalization of $93.09 million, reflecting the market's current valuation of the company following this news.
The company's revenue for the last twelve months as of Q2 2023 stood at $30.26 million, with a revenue growth of 16.56% over the same period. This growth, while positive, may now be overshadowed by the discontinuation of ganaxolone's clinical development and the planned cost reduction measures.
InvestingPro Tips highlight some critical aspects of Marinus' financial situation. The company is "quickly burning through cash," which aligns with the article's mention of implementing cost reduction measures, including workforce reductions. This cash burn rate is particularly concerning given that Marinus is "not profitable over the last twelve months," another InvestingPro Tip that underscores the challenges ahead for the company.
On a slightly positive note, one InvestingPro Tip indicates that "liquid assets exceed short term obligations," suggesting that Marinus may have some financial flexibility as it explores strategic alternatives with Barclays. However, investors should be aware that the "price has fallen significantly over the last year," which is consistent with the disappointing trial results and the company's uncertain future direction.
For those considering Marinus' stock, it's worth noting that InvestingPro offers 5 additional tips that could provide further insights into the company's prospects. These additional tips could be particularly valuable as investors assess the impact of this clinical trial setback on Marinus' long-term outlook.
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