Marinus Pharmaceuticals, Inc.

Marinus Pharmaceutical Second 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 would like to withdraw your question, again, press the star 1. And now, it is my pleasure to introduce you to your host, Sonia Wego, Senior Vice President, Investor Relations, Human Resources, and Corporate Affairs, You may now begin, Ms. Weigle.

Thank you and good morning. With me from Marinus are Dr. Scott Bronstein, Chairman and Chief Executive Officer, Christy Schaffer, 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 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, Sonia, and welcome to our call. We've made strong progress this quarter on both the regulatory and commercial front, as well as advancing our oral and IV Ganaxolone clinical programs. positioning us well for the catalyst-rich year ahead. We are pleased to report Zetalme's second quarter sales of $4.2 million. The launch momentum has continued, and as a result, we are raising full-year 2023 Zetalme sales guidance to the range of $17 to $18.5 million. Christy Schaefer, our Chief Commercial Officer, will be reviewing some of the key initiatives for 2023 that we believe will support the continued long-term growth of the Zetalme franchise. Additionally, we see significant potential for Zitalmi following a successful outcome in seizures associated with tuberous sclerosis complex, an indication that would offer important commercial synergies. This will be one of the topics that we will be discussing in more detail during our Investor Day on September 19th. We are making meaningful progress expanding access to Ganaxalone for patients around the world. On July 31st, we announced that the European Commission approved our marketing authorization application for Zytalmy and CDKL5 deficiency disorder. This approval required a tremendous effort from the Mariners team to respond to the many technical and clinical questions raised by the EMA during the review process. To further support global access, we recently submitted the marketing authorization application in the UK. our ex-U.S. partnerships are a central part of our commitment to reach the global patient community. As previously reported, Marinus entered into an exclusive distribution agreement with Biologics in selected markets in the Middle East and Africa, and we hope to have Zotami available for patients in the MENA region in early 2024. In China, our partner Tenacia has made significant progress and we expect the submission of our CDD-NDA in the near term. Moving to our clinical pipeline, I want to start with an update on the Phase III RAISE trial in refractory status epilepticus, which I'm sure is top of mind for many of you. Total enrollment continued to move in the right direction. However, the summer months have been a time of turnover for many clinical site personnel, which we believe has resulted in a slowdown in recruitment. This phenomena has had a much bigger impact than previously anticipated, particularly while our team has continued to work hard and completed the majority of remaining site activations in the second quarter. As a result of this summer slowdown, which Joe will discuss in more detail, we have moved the interim analysis out three months to Q1 of next year. While we are disappointed in this delay, we believe it is critically important to continue to enroll the right patient population for a successful trial outcome. We are confident that the diligent screening efforts by our clinical team and RAISE study sites will drive a placebo rate well within our expectations, creating the opportunity for a meaningful clinical result and putting us in a position to demonstrate a benefit across multiple healthcare utilization measures. The organization is working towards an expected NDA filing and preparing our future commercial execution. Importantly, the one-quarter delay to our interim data review is not expected to impact the timing of our commercial launch and may allow us to submit our NDA filing with a valuable alternative bottle size, which Christy will discuss shortly. We look forward to providing an overview of upcoming milestones and next steps following the interim analysis, as well as an update on enrollment during our investor and analyst event next month. When the interim analysis is conducted, and if it meets the stopping criteria, we plan to begin transitioning the majority of RAISE sites to an open-label extension, and then to shortly transition a subset of these sites to the RAISE 2 study. This will help support a smooth and rapid completion to RAISE 2 with the goal of driving improved timelines for the European filing and a potential US label expansion. The trial is on track to begin enrollment later this year. Let me move to the oral franchise clinical development program and begin with a quick update on the TRUST TSC trial. We are actively enrolling refractory patients in this global study and continue to expect top-line data in mid-2024. Blinded discontinuation rates in the TRUST TSC trial have been low, which supports our belief that adjustments to dose titration made following the Phase II results can drive improvements in tolerability and deliver a clinically meaningful outcome. One of the elements that is critical to the success of the TRUST trial is working closely with our advocacy partners at the TSC Alliance. Next month, we will be attending the International TSC Research Conference, where the team and I will be meeting with a number of key opinion leaders, patients, and advocates. We believe that Zatomi has the potential to address a significant unmet need for TSC patients suffering from refractory seizures, and we are the only product currently in Phase III development for this indication. At the same time, we have spent considerable energy broadening our patent portfolio for the oral franchise, and are pleased that a new method of use patent has been granted by the USPTO for Ganaxalone for its use in treating seizures associated with TSC. This new method of use patent expires in 2040. Finally, and equally exciting, I'm happy to share that we've enrolled the first patient in the multiple ascending dose study using our second generation oral Ganaxalone formulation and are on track for preliminary data by year end. We believe this formulation has the potential to be the future of the oral franchise and will continue to target its use in patients suffering from LGS and other refractory epilepsies. We saw a meaningful improvement in pharmacokinetics in our single ascending dose trial and are hopeful that this MADS study will reinforce our belief that the second generation oral program will allow twice a day dosing and provide physicians the ability to dose titrate to higher serum concentrations of Ganaxalone than can be achieved today. Both of these attributes are critical to offering an enhanced solution to the broader refractory population. With that, I would like to turn the call over to our Chief Commercial Officer, Christy Schaefer.