Marinus Pharmaceuticals’ attempt to expand the use of its seizure medicine in a new formulation has hit a pothole. As a result, the company is “evaluating potential cost-saving strategies,” CEO Scott Braunstein, M.D., said in a release.
An interim analysis of the phase 3 RAISE trial, evaluating intravenous (IV) ganaxolone as a treatment for refractory status epilepticus (RSE), failed to meet pre-defined “stopping criteria,” the company said in a Monday release.
While an independent Data Monitoring Committee (DMC) recommended the study be continued, Marinus said it has stopped enrollment in the trial at approximately 100 patients. When topline results are ready this summer, the Philadelphia company will decide whether to continue its development of IV ganaxolone.
“While we are disappointed that RAISE did not meet the early stopping criteria, we will only be able to determine the trial’s outcome once we unblind and analyze the full data set,” Braunstein said in a statement.
Cost reductions to extend the company’s cash runway are expected to be implemented in the current quarter, Marinus said, adding that it ended March with cash and cash equivalents of $113 million.
With the news, Marinus' share price was in free fall, dropping 78% on Monday morning.
In March of 2022, Marinus' ganaxolone was approved as Ztalmy for patients age 2 and older with seizures associated with the rare genetic condition CDKL-5 deficiency disorder (CDD), which triggers seizures that can severely affect neurological development. Marinus gained a priority review voucher with the nod, which it sold to Novo Nordisk for $110 million.
In its approved use, Ztalmy is a liquid that's administered orally three times per day.
Marinus expects Ztalmy to generate sales of between $7.4 million and $7.6 million in the first quarter and between $32 million and $34 million in 2024.
The company also is investigating ganaxolone as a treatment for another rare genetic seizure disorder, tuberous sclerosis complex, which causes non-malignant tumors in the brain, skin, kidney heart, eyes and lungs and leads to epilepsy in most patients.
The company expects to complete enrollment of roughly 130 patients in a phase 3 trial in the indication by mid-May, with topline data on track to be available early in the fourth quarter.
Monday’s news deflates Marinus’ hopes in status epilepticus, which sends roughly 150,000 per year in U.S. to the emergency room. Those who are unresponsive to first- or second-line therapy are put into a medically-induced coma to stop seizures, an option that leads to poor outcomes including higher infection rates and longer hospitalizations.
It also casts doubt on Marinus' next-generation IV formulation of ganaxolone, designed to provide improved pharmacodynamic and pharmacokinetic properties, the company said. IV ganaxolone has received orphan drug designation from the FDA for the potential treatment of status epilepticus.