Benzinga - by Vandana Singh, Benzinga Editor.
Monday, Marinus Pharmaceuticals Inc (NASDAQ:MRNS) announced topline results from the Phase 3 RAISE trial evaluating the safety and efficacy of intravenous (IV) ganaxolone for refractory status epilepticus (RSE).
In the RAISE trial, patients with RSE who failed at least two antiseizure medications were randomized to IV ganaxolone or placebo in addition to standard-of-care treatment.
The intent-to-treat population comprised 96 patients, 49 in the IV ganaxolone and 47 in the placebo arm.
Related: Analysts Question Commercial Viability of Marinus’ IV Ganaxolone After Interim Analysis Miss.
Topline data demonstrated that:
- A statistically significant proportion of patients had status epilepticus cessation within 30 minutes of initiating IV ganaxolone compared to placebo: 80% vs. 13%, respectively (p
- RAISE failed to achieve statistical significance in the proportion of patients not progressing to IV anesthesia for 36 hours following initiation of IV ganaxolone compared to placebo: 63% vs. 51%, respectively (p=0.162).
Marinus continues to believe in the potential of IV ganaxolone as a treatment for RSE, supported not only by the rapid onset of its antiseizure effect but also by the objective evidence of status epilepticus control observed with an additional analysis of continuous electroencephalogram (EEG) monitoring.
Preliminary EEG analyses indicate patients receiving IV ganaxolone demonstrated durable reductions in seizure burden through 36 hours with an 88% median reduction compared to 38% for placebo.
This suggests that the need for IV anesthesia was driven by factors other than status severity and may not represent an accurate measure of seizure control.
The company will continue to analyze the full RAISE dataset and will engage with the FDA to discuss a potential path forward for IV ganaxolone in RSE.
Marinus intends to continue to offer IV ganaxolone for patients with super refractory status epilepticus under emergency investigational new drug applications.
The company expects cash and cash equivalents to be sufficient to fund its operating expenses into the second quarter of 2025.
Price Action: MRNS shares are down 7.52% at $1.355 during the premarket session at last check Monday.
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