RADNOR, Pa. - Marinus (NASDAQ:MRNS) Pharmaceuticals, Inc. (NASDAQ:MRNS), has announced that the Phase 3 RAISE trial of intravenous ganaxolone for refractory status epilepticus (RSE) will continue as planned despite not meeting early stopping criteria. The independent Data Monitoring Committee (DMC) has recommended the trial's progression following an interim analysis, with enrollment completed at roughly 100 patients. Topline results are anticipated in the summer of 2024.
The RAISE trial's findings will be crucial in determining the future development of IV ganaxolone. Marinus remains blinded to the trial data until its conclusion. The trial is partly funded by the Biomedical Advanced Research and Development Authority (BARDA).
In a separate development, Marinus expects to finish patient enrollment for the Phase 3 TrustTSC trial of ZTALMY® (ganaxolone) oral suspension CV in mid-May, with topline data expected early in the fourth quarter of 2024. A supplemental New Drug Application to the U.S. Food and Drug Administration is planned for the first half of 2025.
Marinus is also working on a second-generation ganaxolone formulation to potentially improve safety, efficacy, and tolerability, which could allow for less frequent dosing.
Financially, Marinus has reported preliminary net product revenue for Q1 2024 of between $7.4 and $7.6 million from the commercial launch of ZTALMY in the U.S. The company also reported preliminary unaudited cash, cash equivalents, and short-term investments totaling $113.3 million as of March 31, 2024. In response to these financial results, Marinus is reviewing cost reduction activities to extend its cash runway, expected to be implemented in Q2 2024.
Status epilepticus (SE) is a serious neurological condition where prolonged seizures occur. It affects up to 150,000 patients annually in the U.S. and can lead to significant morbidity, mortality, and healthcare costs. RSE patients are those who do not respond to first and second-line treatments.
Ganaxolone is a neuroactive steroid that modulates GABAA receptors and has been granted orphan drug designation by the U.S. Food and Drug Administration for the potential treatment of SE.
This news is based on a press release statement from Marinus Pharmaceuticals.
InvestingPro Insights
As Marinus Pharmaceuticals (NASDAQ:MRNS) continues to progress with its clinical trials, the financial health and stock performance of the company remain areas of interest for investors. According to recent data from InvestingPro, Marinus has a market capitalization of approximately $413.03 million. Despite the ongoing trials and the launch of ZTALMY in the U.S., Marinus faces financial challenges, with a negative P/E ratio of -2.87, indicating the company is not currently profitable.
InvestingPro Tips suggest that Marinus is quickly burning through cash and analysts have revised their earnings downwards for the upcoming period, which could be concerning for long-term viability. Additionally, the company suffers from weak gross profit margins, as evidenced by a gross profit margin of -226.96% over the last twelve months as of Q1 2023. This could impact the company's ability to fund ongoing and future trials without seeking additional capital or cost-cutting measures.
On a more positive note, Marinus's liquid assets exceed its short-term obligations, which may provide some financial flexibility in the near term. However, with a high Price/Book multiple of 24.64, the stock may be considered overvalued relative to its book value, which could deter value-focused investors.
For those considering an investment in Marinus Pharmaceuticals, there are 9 additional InvestingPro Tips available, which could provide deeper insights into the company's financial health and stock performance. Interested readers can explore these tips at https://www.investing.com/pro/MRNS. To further enhance your research, use the coupon code PRONEWS24 to get an additional 10% off a yearly or biyearly Pro and Pro+ subscription at InvestingPro.
This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.