Marinus Pharmaceuticals has announced that its pivotal Phase III RAISE study (NCT04391569) of IV ganaxolone for the treatment of status epilepticus (SE) has failed to meet its pre-defined early stopping criteria. The company now plans to run the trial to completion to determine the next steps of drug development.
The decision was based on the interim analysis from an independent data monitoring committee (DMC) which recommended the continuation of the Phase III study, the company shared in its 15 April preliminary Q1 financial results. Topline results for the trial are anticipated for release in summer 2024.
Upon completion, the trial will have enrolled 100 patients and results will be unblinded to Marinus; at which point, the Radnor, Pennsylvania-based pharmaceutical company will decide the direction of development for the medication.
Ganaxolone, a positive allosteric modulator of both the synaptic and extrasynaptic GABAA receptors, is designed to hyperpolarise cells and dampen neuron excitability that results in the inhibition of abnormal, seizure-causing electrical transmission. While the drug is being developed for an intravenous (IV) route of administration for the treatment of SE, its oral administration is on the market under the brand name Ztalmy as an anti-seizure medication for the treatment of cyclin-dependent kinase-like 5 (CDLK5) deficiency disorder (CDD).
Status epilepticus is a life-threatening condition that occurs when a seizure is prolonged for over 5 minutes or when repetitive seizures occur very close together, with the person unable to recover normal consciousness between episodes.
First approved by the US Food and Drug Administration (FDA) as a treatment for CDLK5 deficiency disorder in March 2022, oral ganaxolone was awarded UK Medicines and Healthcare products Regulatory Agency (MHRA) approval for use in the indication in March 2024. GlobalData’s consensus forecasts anticipate that Ztalmy will generate $263m in global sales in 2029.
GlobalData is the parent company of Pharmaceutical Technology.
The commercial-stage pharma will also be evaluating potential cost-saving strategies to extend its cash runway as its global Phase III TrustTSC study for tuberous sclerosis complex nears enrollment completion, said CEO Dr. Scott Braunstein in the press release. These cost reduction activities are expected to take place in Q2 2024.
Marinus is not the only company making moves in the anti-seizure space. Last week, South Korea-based SK Life Sciences gained FDA approval for mouth and nasogastric tube administrations of its anti-epileptic, Xcopri (cenobamate). The drug is indicated for the treatment of partial onset seizures in adults.
