Marinus Pharmaceuticals, Inc. (MRNS) announced on Monday the topline results from their Phase 3 RAISE trial. This double-blind, randomized, placebo-controlled study evaluated the safety and efficacy of intravenous (IV) ganaxolone for treating refractory status epilepticus (RSE).
RSE is characterized by prolonged continuous or rapidly recurring seizures that do not respond to first- and second-line therapies, and is associated with significant morbidity and mortality.
The RAISE trial successfully met its first co-primary endpoint by demonstrating rapid cessation of status epilepticus in highly refractory patients. However, it did not achieve statistical significance for its second co-primary endpoint, which measured the proportion of patients who did not progress to IV anesthesia.
Marinus Pharmaceuticals plans to continue analyzing the full RAISE dataset and intends to engage with the U.S. Food and Drug Administration (FDA) to discuss a potential path forward for IV ganaxolone in the treatment of RSE.
The company remains committed to offering IV ganaxolone for patients with super refractory status epilepticus under emergency investigational new drug applications.
Financially, Marinus expects its cash and cash equivalents to be sufficient to cover operating expenses, including capital expenditures and working capital requirements, through the second quarter of 2025.
