RADNOR, Pa. - Marinus (NASDAQ:MRNS) Pharmaceuticals, Inc. (NASDAQ:MRNS) shared new findings from its Phase 3 RAISE trial on intravenous (IV) ganaxolone, revealing promising results for treating refractory status epilepticus (RSE), a life-threatening seizure disorder. The data, presented at the Neurocritical Care Society Annual Meeting, showed that IV ganaxolone significantly reduced the time to seizure cessation and lessened the need for additional treatment within 24 hours.
The RAISE trial, which included patients with RSE unresponsive to at least two antiseizure medications, found that 80% of patients treated with IV ganaxolone experienced seizure cessation within 30 minutes versus 13% with placebo. Moreover, the median time to seizure cessation was notably faster at 4.2 minutes for the treatment group compared to 307.2 minutes for the placebo group.
While the trial successfully met one of its co-primary endpoints, it did not achieve statistical significance for the second, which was preventing progression to IV anesthesia within 36 hours of treatment initiation. Nonetheless, secondary endpoints showed a median reduction in EEG seizure burden of 93% for patients treated with IV ganaxolone, against 36% for those on placebo, and a higher proportion of patients without treatment escalation within 24 hours.
The incidence of serious adverse events was comparable between the treatment and placebo groups, with hypotension more commonly observed in the IV ganaxolone arm. Despite not meeting the second co-primary endpoint, the results suggest IV ganaxolone's potential as a rapid and effective treatment for RSE.
Brandon Foreman, M.D., who presented the data, noted the rapid antiseizure activity of IV ganaxolone in refractory patients and its clinically meaningful benefits. Joseph Hulihan, M.D., Chief Medical Officer of Marinus, highlighted the importance of the findings for managing RSE and the intention to discuss next steps with the FDA.
The development of ganaxolone in the RAISE trial has been partly supported by the U.S. Department of Health and Human Services; Administration for Strategic Preparedness and Response; and Biomedical Advanced Research and Development Authority (BARDA).
This news is based on a press release statement from Marinus Pharmaceuticals.
In other recent news, Marinus Pharmaceuticals has made significant advancements in its operations. The company secured a new U.S. patent for its epilepsy drug, ZTALMY, set to expire in September 2042. The patent covers treatment for various epilepsy disorders, including CDKL5 deficiency disorder, tuberous sclerosis complex, and Lennox-Gastaut syndrome.
The U.S. Patent and Trademark Office Patent Trial and Appeal Board recently upheld Marinus Pharmaceuticals' patent related to the use of ganaxolone, despite challenges from Ovid Therapeutics (NASDAQ:OVID). This development ensures Marinus Pharmaceuticals' exclusive rights to the use of ganaxolone for treating status epilepticus.
Marinus Pharmaceuticals reported an increase in net product revenues, amounting to $8 million for the second quarter, primarily due to their flagship product, ZTALMY. The company is planning for the potential launch of ZTALMY for tuberous sclerosis complex in the second half of 2025.
The company has been the subject of analyst attention recently, with TD Cowen maintaining its Buy rating on Marinus and Oppenheimer upgrading the stock to Outperform. Both firms have expressed confidence in the trial design and potential efficacy of ganaxolone. Despite reporting a net loss before income taxes of $35.8 million for the quarter, Marinus remains on track to meet its revenue guidance for 2024, aiming for net product revenues between $33 million and $35 million. These are some of the recent developments at Marinus Pharmaceuticals.
InvestingPro Insights
The recent clinical trial results for Marinus Pharmaceuticals' (NASDAQ:MRNS) IV ganaxolone treatment for refractory status epilepticus (RSE) come at a critical time for the company. According to InvestingPro data, Marinus has a market capitalization of $99.15 million, reflecting the market's current valuation of the company's potential.
Despite the promising trial results, InvestingPro Tips highlight that Marinus is "quickly burning through cash" and "not profitable over the last twelve months." This aligns with the company's focus on research and development, which is typical for biopharmaceutical firms in the clinical trial phase. 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.
Interestingly, another InvestingPro Tip notes that Marinus has seen a "significant return over the last week," with a 9.09% price total return in the past week. This could be indicative of positive market sentiment following the announcement of the RAISE trial results. The stock has also shown strong returns of 16.13% and 29.5% over the last month and three months, respectively, suggesting growing investor confidence in the company's pipeline.
However, it's important to note that the company's P/E ratio stands at -0.67, reflecting its current unprofitability. This is not uncommon for biotech companies in the development stage, but it underscores the importance of successful clinical trials and potential FDA approvals for Marinus's future.
For investors seeking a more comprehensive analysis, InvestingPro offers additional tips and insights that could provide a fuller picture of Marinus Pharmaceuticals' financial health and market position.
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