Marinus on Track to Capitalize on Recent Seizure Drug Approval
Marinus Pharmaceuticals shared positive news in its Q1 financial report, led by updates from its Phase III trial of a drug for refractory status epilepticus (RSE) and research efforts for rare diseases.
The Radnor, PA-based pharmaceutical firm closed the quarter with $126.3 million in cash and cash equivalents, a slight bump from the $122.9 million it reported in the three months leading up to December 31, 2021. Marinus generated $14.2 million in revenue versus $1.8 million in the same period in 2021, due primarily to a one-time $12.7 million revenue recognition associated with its collaboration with Orion Corporation.
In addition, Marinus was able to scale back on expenses for research and development, logging $18 million in the first quarter compared to $18.6 million in Q1 2021.
"It’s been a productive year thus far as we prepare for our first commercial launch and make important advancements across our clinical programs," Scott Braunstein, M.D., chief executive officer of Marinus, said in a statement.
Marinus is poised to launch the oral suspension of Ztalmy (ganaxolone) in the U.S. in July, pending scheduling from the U.S. Drug Enforcement Administration. Ganaxolone is a neuroactive steroid GABAA receptor modulator that acts on brain targets that have anti-seizure effects. It is being developed as an oral and IV dose for adult and pediatric patients. The company said the market access team for the launch is already in place, and all marketing efforts are on track for the planned release.
In March, the company received an extra $30 million in funds under its credit agreement with Oaktree Capital Management after the U.S. Food and Drug Administration approved Ztalmy in March for seizures associated with cyclin-dependent kinase-like 5 deficiency disorder (CDD). Marinus also received a rare pediatric disease priority review voucher.
The company has also resumed the Phase III RAISE trial assessing ganaxolone for RSE and is conducting screening, recruitment and U.S. site activations, alongside planned target site expansions in Australia, Canada, Israel and other U.S. locations.
Marinus paused recruitment for the trial in February after routine stability monitoring showed visible particulates of aluminum phosphate in the drug's formulation. After a review, the independent Data Monitoring Committee did not identify any concerning safety events in patients who had already received the drug.
Initial results from this trial are expected in the second half of 2023.
The other ongoing studies, Phase II RESET (in the U.S.) and Phase III RAISE II (in Europe) are also expected to start taking participant enrollments by the end of 2022 and the first half of 2023, respectively.
In addition to RSE, Marinus is looking into using ganaxolone for Lennox-Gastaut Syndrome (LGS) and is preparing for Phase I patient dosing, with topline data expected to be shared by mid-2022. After the Phase I data is assessed, the firm will commence Phase II for LGS in the second half of 2022.
Other ganaxolone indications Marinus is pursuing include CDKL5 Deficiency Disorder, where it is currently complying with requirements for a Marketing Authorization Application with the European Medicine Agency, and Tuberous Sclerosis Complex (TSC), where it is actively recruiting for Phase III TrustTSC trial participants in the U.S.
"We believe the diligent efforts of our internal teams to achieve the resumption of our Phase III RAISE trial in refractory status epilepticus this month keeps us on track for this key trial, and our next generation formulation development will provide meaningful data this year for our next steps in new indications," Braunstein added.