Marinus Pharmaceuticals, Inc.

Ladies and gentlemen, greetings and welcome to Marinus Pharmaceuticals' fourth quarter and full year 2023 financial results and business update call. Today's call is being recorded and all lines have been placed on mute to prevent any background noise. After the speaker's remarks, there will be a question and answer session. If you would like to ask a question during that time, simply press the star key followed by the number one on your telephone keypad. If you would like to withdraw your question, press star one a second time. Thank you, and it is my pleasure to introduce your host, Sonia Weigel, Senior Vice President of Investor Relations, Human Resources, and Corporate Affairs. Ms. Weigel, you may begin.

Thank you, and good afternoon. With me from Marinus are Dr. Scott Bronte, Chairman and Chief Executive Officer, Christy Schaefer, Chief Commercial Officer, Dr. Joe Houlihan, Chief Medical Officer, and Steve Fanfield, Chief Financial Officer and Chief Operating Officer. Before we begin, I would like to remind everyone that some of the statements we are making today are forward-looking statements under the securities laws. These forward-looking statements involve substantial risks and uncertainties that could cause our clinical development program, future results, performance, or achievements to differ significantly from those expressed or implied by such forward-looking statements. These risks and uncertainties and risks associated with our business are described in the company's reports filed with the Securities and Exchange Commission, including Form 10-K, 10-Q, and 8-K. I will now turn the call over to our CEO, Dr. Scott Bronstein.

Thank you, Sonia. Marinus concluded 2023 with a strong finish across all fronts, commercial, clinical, and operational. On today's call, I'll provide a brief overview of some of the key areas before turning it over to our leadership team. Starting with an update on ZTALME, we finished 2023 with another strong quarter of enrollment and robust quarterly growth. As a result of the progress made by our commercial team, we expect to achieve profitability on our ZTALME commercial investment by the second quarter of 2024, ahead of our previous two-year target. Christy will provide a summary of our revenue results in her remarks, as well as an update on our investments to continue to grow the CDD business and our launch plans as we prepare for two critical phase three data readouts in the second and fourth quarters of this year. Our commercial partners in the EU, China, and MENA regions continue to make important progress to support Zotomi launches around the globe. In China, the Tenacia team has been granted priority review of the NDA submission in PDD, as well as contributing to the enrollment of the TRUST-TSC trial. In Europe, Orion continues to plan for the launch of the TALMI in select European countries in 2024. Finally, in the MENA region, we are targeting that our partner biologics will begin their distribution strategy in the second half of this year. Concurrently, we are expanding our manufacturing investments to ensure that we can adequately supply not only our global partners, but the broader market opportunities for Zetalme over the coming years. Turning to our clinical pipeline, I'll first share an update on our Phase III RAISE trial of ibuprofen and refractory status epilepticus. As we announced in our press release this afternoon, we are pleased to report that we have met the enrollment criteria for the interim analysis and now have more than 90 patients enrolled in the trial. We expect to deliver the interim results to the data monitoring committee over the coming weeks and plan to announce the outcome within the first half of the second quarter. Based on continued strong enrollment seen over the past six months, we project approximately 100 patients to be included in the secondary endpoint analyses. This growing data set should drive a robust package for both the FDA filing and our health economic outcomes. We expect to have the comprehensive trial results over the summer and to present this data at a series of medical meetings in the fourth quarter. We are currently planning for an NDA submission in the first quarter of 2025, and are expecting a priority review. We see the recent uptick in enrollment as a strong reflection of the potential market opportunity for the IV franchise. Domestically, we believe the addressable market for RSE is approximately 35,000 patients per year, and we have the unique opportunity to bring a novel therapy to physicians. We plan to build our leadership in the hospital by continuing to invest in future status epilepticus research while making the appropriate commercial investment with the goal of ascertaining value-based pricing and broad physician adoption. Let me move to an update on our oral pipeline. Approximately 85% of the patients have been enrolled in our Trust TSC trial, and the discontinuation rate is below 7%. Due to some minor delays in screening, we expect to complete enrollment in the Trust TSC trial during the first half of the second quarter. As a result, we now anticipate our top-line Phase 3 results in the first half of the fourth quarter of this year, rather than the end of Q3. We could not be more pleased with the baseline demographics of the patients enrolled, the high percentage of patients rolling over to the open-label portion of the study, and the low overall discontinuation rates, which are substantially different than what we saw in Phase 2. We believe the quality of this dataset will support a compelling pricing strategy consistent with what we've seen to date for Zytalmy. The commercial team continues to make the appropriate investments to prepare for a potential launch in 2025, and we are eager to offer patients suffering from refractory TSC a novel anti-seizure therapy. Based on our market analysis, the addressable patient population in refractory TSC is projected to be about 10,000 patients in the United States. By leveraging our current commercial organization, we believe successful expansion of this opportunity will require a modest incremental investment. As a result, our goal is to drive profitability for the entire Zatomi franchise within six to 12 months of the 2025 TSC launch. 2024 will be a pivotal year for the company as we have built a solid foundation that has us well-positioned to drive future growth. Together, the CDD, RSD, and TSC markets represent a multibillion-dollar opportunity where we believe we can take a firm leadership position for these disease states and other refractory epilepsies. With an established commercial and clinical track record, we look forward to building our momentum for Zetalny while also reporting on these key data milestones later this year. I'll now turn the call over to our Chief Commercial Officer, Christy Schaefer.