Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS), a company focused on developing innovative therapies for seizure disorders, has shared new data from its Phase 3 RAISE trial on intravenous (IV) ganaxolone for refractory status epilepticus (RSE) at the Neurocritical Care Society (NCS) Annual Meeting. The trial, which evaluated IV ganaxolone in patients with RSE unresponsive to at least two antiseizure medications, aimed to assess two co-primary endpoints: cessation of status epilepticus (SE) within 30 minutes and avoidance of IV anesthesia within 36 hours.
Key secondary findings presented include:
- Median time to SE cessation was 4.2 minutes with IV ganaxolone, compared to 307.2 minutes for placebo (nominal p<0.0001).
- 45% of patients on IV ganaxolone avoided treatment escalation within 24 hours versus 19% for placebo (nominal p=0.0059).
Previously reported primary endpoint results showed:
- 80% of patients achieved SE cessation within 30 minutes with IV ganaxolone versus 13% for placebo (p<0.0001).
- The second co-primary endpoint—avoiding IV anesthesia for 36 hours—did not reach statistical significance, with 63% of ganaxolone patients versus 51% for placebo (p=0.162).
- Median reduction in EEG seizure burden over 36 hours was 93% for ganaxolone compared to 36% for placebo (nominal p=0.003).
- Serious adverse event rates were similar for ganaxolone and placebo groups, with hypotension more frequent in the ganaxolone arm.
Dr. Brandon Foreman, presenting the data, highlighted the rapid antiseizure effect of IV ganaxolone, noting that 80% of patients experienced SE cessation within 30 minutes. He acknowledged that while the second co-primary endpoint was not statistically significant, secondary measures such as EEG seizure reduction showed meaningful clinical benefits.
Joseph Hulihan, M.D., Marinus’s Chief Medical Officer, emphasized the trial’s significance as the first placebo-controlled study in RSE, providing valuable insights into ganaxolone’s potential benefits for this critical condition. The company plans to discuss the findings with the FDA to determine next steps for IV ganaxolone.
The RAISE trial received partial funding from the Department of Health and Human Services’ Biomedical Advanced Research and Development Authority (BARDA).
About Status Epilepticus
Status epilepticus (SE) is a life-threatening condition characterized by prolonged or recurrent seizures, often requiring emergency treatment. Affecting up to 150,000 people annually in the U.S., SE carries significant risks of morbidity, mortality, and high healthcare costs. Refractory SE (RSE) occurs when standard treatments fail to control seizures.
About IV Ganaxolone
Ganaxolone is a neuroactive steroid that enhances inhibitory signaling by targeting GABAA receptors, making it suitable for SE treatment. It has orphan drug designation from the U.S. FDA for status epilepticus.
About Marinus Pharmaceuticals
Marinus Pharmaceuticals is a commercial-stage company advancing treatments for seizure disorders, including ZTALMY® (ganaxolone) oral suspension, which was launched in the U.S. in 2022.
