RADNOR, Pa.–(BUSINESS WIRE)–Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat seizure disorders, today reported business highlights and financial results for the fourth quarter and year ended December 31, 2023.
“We are thrilled to announce we have exceeded the enrollment threshold required to conduct an interim analysis in the Phase 3 RAISE trial in refractory status epilepticus, a life-threatening condition,” said Scott Braunstein, M.D., Chairman and Chief Executive Officer of Marinus. “With over 90 patients now randomized following several months of increasingly strong enrollment trends, we are on track to announce topline data in the second quarter, assuming efficacy criteria for the interim analysis are met. We also remain focused on advancing the Phase 3 TrustTSC trial in tuberous sclerosis complex and are confident that the new titration schedule is having the desired effect with the current discontinuation rate below 7%. We continue to see steady adoption of ZTALMY in CDKL5 deficiency disorder and are eager to build on this momentum throughout 2024 as we grow the ZTALMY franchise and prepare for RSE and TSC data this year.”
ZTALMY®
- ZTALMY® (ganaxolone) oral suspension CV net product revenue of $6.6 million for the fourth quarter of 2023 and $19.6 million for the full year ended December 31, 2023
- Continued growth in commercial patients with more than 165 patients active on therapy at the end of 2023
- Full year 2024 projected U.S. ZTALMY net product revenues of between $32 and $34 million
- Orion Corporation continues to prepare for commercial launches of ZTALMY in select European countries in 2024
Clinical Pipeline
Status Epilepticus
- Achieved enrollment target required for an interim analysis in the Phase 3 RAISE trial of intravenous (IV) ganaxolone in refractory status epilepticus (RSE)
- If pre-defined stopping criteria for the interim analysis are met, the Company expects to report topline data in the first half of the second quarter of 2024
- Strong enrollment trends have continued and now expect approximately 100 patients to be randomized by the conclusion of the interim analysis; this larger database will support health economic outcomes
- Targeting submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in early 2025 with priority review expected
- Phase 3 RAISE II trial in RSE (for European registration) underway with enrollment expected to complete in the fourth quarter of 2025
- Trial design underway with plans to submit a new protocol for IV ganaxolone in super refractory status epilepticus (SRSE) to the FDA in the second quarter of 2024
- To date, the Company has received over 25 physician requests for the use of IV ganaxolone to treat SRSE patients
Ganaxolone development in the RAISE trial is being supported or in part by the Department of Health and Human Services; Administration for Strategic Preparedness and Response; Biomedical Advanced Research and Development Authority (BARDA) under contract number 75A50120C00159.
Tuberous Sclerosis Complex and Other Rare Genetic Epilepsies
- Enrollment in the global Phase 3 TrustTSC trial of oral ganaxolone in tuberous sclerosis complex now at 85% and expected to be completed in the second quarter of 2024
- Topline data now anticipated in the first half of the fourth quarter of 2024
- Targeting submission of a supplemental NDA to the FDA in the first half of 2025 with priority review expected
- Company now anticipates initiating a proof-of-concept study with oral ganaxolone to treat a range of epileptic encephalopathies, including Lennox-Gastaut syndrome in late 2024
- IND-enabling studies for a ganaxolone prodrug are expected to be completed by year-end 2024
General Business and Financial Update
- Company expects that cash, cash equivalents and short-term investments of $150.3 million as of December 31, 2023, will be sufficient to fund the Company’s operating expenses, capital expenditure requirements and maintain the minimum cash balance of $15 million required under the Company’s debt facility into the fourth quarter of 2024.
- For the fiscal year 2024, the Company expects ZTALMY U.S. net product revenues of between $32 and $34 million.
Financial Results
- Recognized $6.6 million and $19.6 million in net product revenues for the three and twelve months ended December 31, 2023, respectively, as compared to $2.3 million and $2.9 million for the three and twelve months ended December 31, 2022, respectively.
- Recognized $0.6 million and $11.4 million in Biomedical Advanced Research and Development Authority (BARDA) federal contract revenue for the three and twelve months ended December 31, 2023, respectively, as compared to $1.8 million and $6.9 million for the three and twelve months ended December 31, 2022, respectively.
- Research and development (R&D) expenses were $26.4 million and $99.4 million for the three and twelve months ended December 31, 2023, respectively, as compared to $21.4 million and $79.9 million, respectively, for the same periods in the prior year; the increase was due primarily to increased costs associated with our API onshoring effort, increased TSC and RSE clinical trial activity, and increased headcount.
- Selling, general and administrative (SG&A) expenses were $15.4 million and $61.2 million for the three and twelve months ended December 31, 2023, respectively, as compared to $14.7 million and $56.8 million, respectively, for the same periods in the prior year; the primary drivers of the change were annualization of the U.S. ZTALMY launch costs and increased headcount.
- The Company had net losses of $41.8 million and $141.4 million for the three and twelve months ended December 31, 2023, respectively; cash used in operating activities increased to $118.0 million for the twelve months ended December 31, 2023, compared to $112.9 million for the same period a year ago.
- At December 31, 2023, the Company had cash, cash equivalents and short-term investments of $150.3 million, compared to cash and cash equivalents of $240.6 million at December 31, 2022.
- Readers are referred to, and encouraged to read in its entirety, the Company’s Annual Report on Form 10-K for the fiscal year ended December 31, 2023, to be filed with the Securities and Exchange Commission on March 5, 2024, which includes further details on the Company’s business plans, operations, financial condition, and results of operations.
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, visit www.marinuspharma.com.
