Marinus Pharmaceuticals is implementing a raft of cost-cutting measures in the wake of last month’s phase 3 struggles—including jettisoning a fifth of its workforce.
Employees at the Pennsylvania-based biotech may have been expecting some bad news ever since CEO Scott Braunstein, M.D., warned the company was “evaluating potential cost-saving strategies” in April after an interim analysis of the RAISE trial assessing intravenous ganaxolone as a treatment for refractory status epilepticus (RSE) failed to meet predefined “stopping criteria.”
Now, Marinus has revealed that the strategy will involve reducing its head count by around 20% as well as deferring its investments in manufacturing intravenous ganaxolone. The biotech is also halting enrollment in both the RAISE trial and another late-stage study in RSE called RAISE II.
“Future development in RSE will be assessed following review of the RAISE topline data,” Marinus explained in a first-quarter earnings release.
The cost-cutting won’t stop there. The company also mentioned “additional cost reductions across both R&D and general and administrative functions” as well as a vague reference to “other operational changes to increase overall efficiency of the company’s operations.”
Marinus ended March with $113.3 million in cash and equivalents. The company had previously expected its financial fuel to run out by the end of 2024, but the biotech said the package of cost reductions means it should be able to fund operations into the first quarter of 2025, including maintaining a minimum bank balance of $15 million as required by its debt facility.
The current strategy is to focus its resources on the continued commercialization and development of ganaxolone—approved under the brand Ztalmy for seizures associated with the rare genetic condition CDKL5 deficiency disorder (CDD)—including as part of the late-stage TrustTSC trial.
“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 this morning’s release. “We will also be evaluating potential cost-saving strategies to provide the strongest capital position as we approach enrollment completion in the global phase 3 TrustTSC trial in tuberous sclerosis complex.”
The company reaffirmed its plans to complete enrollment of roughly 130 patients in the TrustTSC trial by mid-May, with top-line data on track to be available early in the fourth quarter. Tuberous sclerosis complex is a seizure disorder that causes non-malignant tumors in the brain, skin, kidney heart, eyes and lungs and leads to epilepsy in most patients.
“We are optimistic that we can replicate the success of our pivotal CDD trial of Ztalmy in the TrustTSC study, where we believe refinements made to the phase 3 titration schedule and low discontinuation rates throughout the trial suggest improved tolerability,” Chief Medical Officer Joseph Hulihan, M.D., said in today’s release.
“Our pipeline is supported by clinical and preclinical data which has demonstrated ganaxolone’s potential in treating seizures resistant to other medications, attributed to its unique ability to modulate both synaptic and extrasynaptic GABAA receptors,” Hulihan added. “This is particularly valuable in indications such as TSC, where approximately 25% of patients suffer from highly refractory epilepsy.”