Marinus Pharmaceuticals, Inc.

Greetings, and welcome to the Marinus Pharmaceuticals first 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. And now it is my pleasure to introduce your host, Sasha Damouni-Ellis, Senior Vice President of Corporate Affairs and Investor Relations. You may begin, Ms. Damouni-Ellis.

Thank you, and good morning. With me from Marinus Pharmaceuticals are Dr. Scott Bronstein, Chairman and Chief Executive Officer, Christy Schaefer, Chief Commercial Officer, Dr. Alex Ameddi, Chief Scientific Officer, and Steve Samsell, 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 programs, 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, Scott Bronstein.

Thank you, Sasha, and welcome to our call. During the first quarter of 2023, we made important progress advancing our oral and IV Ginexalone programs, further establishing Marinus as a leader in the development of innovative treatment options for patients with rare genetic epilepsy and refractory seizure disorders. We continue to make considerable strides with the launches of Ptolemy, are pleased with our first quarter sales of $3.3 million, and are on track to meet our previously stated full-year 2023 guidance of $15 to $17 million. Christy Schaefer, our Chief Commercial Officer, will be reviewing some of the key initiatives for 2023 that we believe will support the continued growth of the Ptolemy. We've also made significant progress in our commitment to bring Ganaxalone to more patients around the world. Let me start with an update on our European TDD marketing authorization application. As part of the day 195 assessment report, the European Medicines Agency communicated that the major objection related to the choice of regulatory starting material has been resolved. We expect to receive the CHMP opinion for Zotami and CVD by the end of May and are confident with the progress that we have made to date. As a reminder, in August of 2021, we entered an exclusive agreement with Orion Corporation for the commercialization of Ganaxalone in Europe and have been collaborating with them to prepare for the potential launch. We look forward to sharing the formal CHMP opinion with you later this month. Furthermore, we are pleased to have entered into an exclusive distribution and supply agreement with Biologics for Zytalmy in several markets in the Middle East and North Africa. Biologics is a multinational pharmaceutical company based in Dubai. We hope to have Zytalmy available for patients in the MENA region in early 2024. In parallel, we are working closely with Tenacia, our partner in China, and expect them to file a new drug application by the end of this year. We are engaged in active discussions with several strategics who are interested in bringing our products to market in Japan. Our current and future SUS partnerships are a central part of our commitment to reach the global patient community. Moving to our clinical pipeline, we are excited to announce our plans to conduct an interim analysis for the Phase III RAISE trial in refractory status epilepticus later this year. Alex will discuss the process in more detail shortly, but if the results meet the predefined stopping criteria, we expect to report interim top-line data in the second half of this year. Our leadership continues to prepare for a potential RFC filing, including a growing effort from our commercial, regulatory, CMC, and medical science teams. Additionally, we are gratified that physicians across the U.S. are requesting the use of ibuprofen for their most severe super refractory patients, and some have seen important clinical improvements. We are actively recruiting for the Phase III TRUST TSC trial in the U.S., Europe, and Canada, and expect the vast majority of clinical sites will be ready to enroll patients by mid-year, including new sites in Israel, Italy, Belgium, Australia, and China. Due to administrative and regulatory delays in certain ex-U.S. countries, many of these which have now been resolved, we expect top-line data no later than the middle of 2024. Importantly, we learned with CDV, efficacy and tolerability are key attributes to success in the refractory epilepsy population, and we are confident that the current protocol design maximizes our opportunity for a clinically meaningful outcome. Let me conclude with a few additional business updates. We continue to advance our IP portfolio and recently received a notice of allowance from the U.S. Patent and Trademark Office for a new method of use patent for Ganaxalone in TSC. When granted, the term of this patent will run through 2040. We have spent considerable time and energy broadening our patent portfolio and look forward to the final issuance of both this new method of use patent for the oral franchise in TSC and an additional dosing method of use patent for our IV franchise in the treatment of RFC. We believe that there are additional opportunities to expand our patent portfolio based on our scientific advancements in the next several years. We look forward to our upcoming phase three data readouts and the clinical progress in our second generation platforms. We expect a number of additional milestones before the end of the year, including enrolling our first patient in the RAISE-2 trial and completing enrollment of the first cohort of patients in the RESET trial in established status epilepticus. We believe our second-generation formulations have the potential to be the future of the Denasalone franchise and are planning to initiate a multiple ascending dose study with our first new formulation in the third quarter with preliminary results expected by year-end. If these results are consistent with the single ascending dose modeling, this formulation would be utilized in a future trial in Lennox-Gastaut syndrome with the clinical program design expected to be finalized in the first quarter of 2024. Finally, we are pleased to welcome Marvin Johnson to our Board of Directors. Marvin has over 34 years of diverse commercial operations experience at Merck and will be invaluable in supporting the Zetalny commercial efforts while preparing for potential hospital launch of IV Ganaxilone. Now, I would like to turn the call over to our Chief Commercial Officer, Christy Schaefer.