Marinus Pharmaceuticals(MRNS) - 2023 Q4 - Earnings Call Transcript

Financial Data and Key Metrics Changes - The company reported net product revenue of $19.6 million for the full year 2023, which exceeded the revised guidance range of $18.5 million to $19 million [32][67] - The projected U.S. ZTALMY net product revenues for 2024 are between $32 million and $34 million, representing nearly 70% growth compared to 2023 [33][68] - The company ended 2023 with cash, cash equivalents, and short-term investments of $150.3 million, expected to provide a cash runway into the fourth quarter of 2024 [96] Business Line Data and Key Metrics Changes - The ZTALMY product revenues for Q4 2023 were $6.6 million, compared to $2.3 million in Q4 2022, indicating strong growth [97] - The discontinuation rates for the TrustTSC trial are below 7%, showing confidence in the revised titration schedule [93] - The company has achieved over 85% enrollment in the TrustTSC trial, with a high transition rate to the open-label extension [93] Market Data and Key Metrics Changes - The addressable market for refractory status epilepticus (RSE) is estimated at approximately 35,000 patients per year in the U.S. [29] - The addressable patient population in refractory TSC is projected to be about 10,000 patients in the United States [51] - The company is targeting a significant market opportunity in the MENA region, with plans for distribution strategies to begin in the second half of the year [27] Company Strategy and Development Direction - The company plans to build leadership in the hospital market by investing in future status epilepticus research and making commercial investments to ascertain value-based pricing [29] - There is a focus on expanding the ZTALMY business into TSC, leveraging synergies with the CDD market [57] - The company aims to achieve profitability for the ZTALMY franchise within 6 to 12 months of the 2025 TSC launch [31] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the upcoming Phase 3 data readouts for RSE and TSC, which are expected to drive significant growth for the ganaxolone franchise [72] - The management highlighted the importance of aligning development and execution with key milestones for the acute care business in 2024 [58] - There is optimism regarding the reimbursement dynamics for TSC, based on the success seen with ZTALMY in the CDD population [74] Other Important Information - The company plans to submit a supplemental NDA in the first half of 2025 with a priority review expected [43] - The interim results for the RAISE trial are anticipated to be reported in the second quarter of 2024 [48][89] - The company is preparing for a potential launch in 2025 for the IV formulation of ganaxolone for RSE [56] Q&A Session Summary Question: What are the reimbursement dynamics for TSC based on ZTALMY? - Management noted that over 80% of commercial plans have straightforward guidelines, with no patients denied therapy since launch, indicating strong payer recognition [74] Question: What remains to be done for the NDA package? - The company plans to gather data from approximately 100 patients for the NDA filing, with a pre-NDA meeting with the FDA expected soon after positive data [78] Question: What are the stopping criteria for the RAISE trial? - The stopping criteria are based on co-primary endpoints, including cessation within 30 minutes and lack of progression to IV anesthesia within 36 hours, with a required p-value of 0.0293 for statistical significance [111]