Marinus Pharmaceuticals, Inc.

Greetings and welcome to the Marinus Pharmaceuticals third quarter 2023 financial results and business update call. At this time, all participants are in a listen-only mode. After the speaker's presentation, there will be a question and answer session. If you would like to ask a question during this time, simply press star 1 on your telephone keypad. If you wish to withdraw your question, you again press star 1. For time, please restrict yourself to one question. You may queue back up for a follow-up if time permits. And now, it is my pleasure to introduce your host, Sonya Weigel, Senior Vice President, Investor Relations, Human Resources, and Corporate Affairs. You may begin, Ms. Weigel.

Thank you, and good morning. With me from there is Dr. Scott Bronstein, Chairman and Chief Executive Officer, Christy Schaefer, Chief Commercial Officer, T.J. Lyons, Chief Business 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 security laws. These forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs future results, performance, or achievements that 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 report 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, and welcome to our call. Before I turn to our progress in the third quarter, I want to express my appreciation to those of you who attended in person or via webcast our investor and analyst events in September. I also want to thank our key opinion leaders, Dr. Dar, Dr. Koenig, and Dr. Khan, who reviewed their clinical experience treating patients with status and TSC. The third quarter of 2023 was marked by strong Zetalme sales growth, meaningful progress in expanding global access to Zetalme, continued advancement of our late-stage clinical programs, and ongoing development of our second-generation oral Ganax Loan product. We are pleased to report that Zetalme sales in the third quarter slightly exceeded our mid-September expectations, with revenues of $5.4 million and we are once again raising our guidance to the range of $18.5 to $19 million. Just four full quarters into our launch, we are pleased to report our annual revenue run rate is well north of $20 million. These results validate our efforts to build a strong commercial organization and execute on our rare disease-focused strategy. Christy Schaefer, our Chief Commercial Officer, will review our achievements and the steps we are taking to prepare for two new potential launches in 2025. In addition to the early commercial success in the US, we are making meaningful progress in expanding access to Zetalme for patients around the world. Today, we announced the launch of our new global managed access program designed to provide physicians the opportunity to formally request the use of Zetalme for their patients and geographies where the product is not commercially available and as supported by local regulatory requirements. We are pleased to be collaborating with Durban, an experienced leader in this area, who will facilitate the program. Thomas Lyons, our Chief Business Officer, will provide an overview following comments from Christy. Our commercial partners in the EU, China, and MENA regions have also made notable progress. the Center for Drug Evaluation of the China National Medical Product Administration granted priority review of the NDA submitted by Tenacia Biotechnology for Zetalme and CDD. Additionally, Orion Corporation continues to plan for the commercial launch of Zetalme in selected European countries in 2024, and we expect our partner in the MENA region, Biologics, to have product available for distribution by the end of this year. We are proud that in such a short period of time, Marinus has created a global network that will provide the opportunity for CDD patients and their families to access our novel anticonvulsant therapy. Let me take a few moments to review our clinical pipeline, starting with an update on the phase three RAISE trial of IV gonaxolone in refractory status epilepsia. In our last update provided at our investor and analyst event in September, We noted the RAISE trial enrollment was on an upward trajectory since early August, following the activations of all clinical sites. Although total enrollment has continued to grow and screening activity remains quite high, the rate of enrollment has continued to show more variability than we expected. Based on current enrollment trends, we now project to enroll the number of patients required for the interim analysis by the end of the first quarter of 2024. rather than our previous guidance of January. As a result, we now anticipate top line data in the second quarter of 2024 if the predefined stopping criteria for the interim analysis are met. The entire organization remains acutely focused on advancing our phase three clinical trials in refractory status epilepticus and TSC. We are confident in the benefits that IV Ginexolone could bring to critically ill patients and the significant commercial opportunity for the first novel therapy for acute status in well over a decade. We are committed to successfully completing both the RAISE and TRUST TSC trials in 2024, and continue to make the investment to prepare for these commercial launches. Preparations are underway for an NDA filing, and we don't expect the additional delay to significantly impact the timing of our commercial launch. Finally, with the success of the TALMI, the recent extension of our cash runaway into Q4 2024, and tightening of our spend to focus on our most valuable market opportunities, we believe we have the appropriate resources required to complete two key data readouts and prepare for a bright future. Before I turn to our oral franchise, I want to give a brief update on our reset trial in established SE. We have chosen to discontinue the Phase II reset trial and establish status to focus additional resources on the expansion of our refractory clinical programs, including further investigation of a potential development opportunity in super refractory status. Our efforts will continue to focus on completing the RAISE trial, accelerating the enrollment for RAISE II, as well as generating additional data that has the potential to support further studies and SRSE, and RSE Health Economic Outcomes Research. We will evaluate every investment the company is making and prioritize the opportunities that we believe will maximize shareholder value. Moving to our oral franchise, we are actively enrolling patients in our Global Phase III Trust TSC trial. We believe that SITOMI can address a significant unmet medical need for patients suffering from refractory seizures associated with TSC, and we are currently the only product in Phase III development for this indication. Blinded discontinuity rates in this trial remain low, which gives us high confidence in the tolerability and potential efficacy of our new titration schedule. We expect top-line data mid-2024 and have every reason to believe that this study can replicate the success of Zitomib, seen in the Marigold trial, and our real-world experience to date. With several key readouts in 2024, We believe we have laid a strong foundation for near and long-term growth and have prioritized key programs in our cash runaway accordingly. With that, I'd like to turn the call over to our Chief Commercial Officer, Christy Schaefer.