Marinus Pharmaceuticals Provides Update on the Phase 3 RAISE Trial and Reports Preliminary First Quarter 2024 Financial Results - Seite 2
The preliminary first quarter 2024 net product revenue results and cash, cash equivalents, and short-term investments included in this release were calculated prior to the completion of a review by the Company’s independent registered public accounting firm and are therefore subject to adjustment.
About Status Epilepticus
Status epilepticus (SE) is a life-threatening condition resulting from either the failure of the mechanisms responsible for seizure termination or from the initiation of mechanisms which lead to abnormally prolonged seizures.1 SE is the one of the most common neurological emergencies in the U.S., affecting up to 150,000 patients each year, and is associated with substantial morbidity, mortality, and healthcare costs.2,3,4 Patients who do not respond to 1st- and 2nd-line treatments (benzodiazepines and intravenous antiseizure medications) are considered to have refractory SE (RSE).4,5
About Intravenous (IV) Ganaxolone
Ganaxolone is a neuroactive steroid that works by modulating both synaptic and extrasynaptic GABAA receptors via a unique binding site to potentiate two types of inhibitory signaling.6 IV ganaxolone has pharmacokinetic and pharmacodynamic properties well-suited for the treatment of status epilepticus, with rapid and sustained SE cessation observed in pre-clinical and clinical studies.7,8,9 IV ganaxolone has received orphan drug designation from the U.S. Food and Drug Administration for the potential treatment of status epilepticus.
About the RAISE Trial
The RAISE (Randomized Therapy in Status Epilepticus) trial (NCT04391569) is a Phase 3 double-blind, randomized, placebo-controlled clinical trial to evaluate the safety and efficacy of IV ganaxolone in patients with refractory status epilepticus. The RAISE protocol provides for an independent data monitoring committee to conduct an unblinded interim analysis when two-thirds of participants, or approximately 82 patients, have completed the trial.
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About Marinus Pharmaceuticals
Marinus is a commercial-stage pharmaceutical company dedicated to the development of innovative therapeutics for seizure disorders. The Company first introduced FDA-approved prescription medication ZTALMY (ganaxolone) oral suspension CV in the U.S. in 2022 and continues to invest in the potential of ganaxolone in IV and oral formulations to maximize therapeutic reach for adult and pediatric patients in acute and chronic care settings. For more information about Marinus visit www.marinuspharma.com.