Marinus Pharmaceuticals, Inc.

Ladies and gentlemen, thank you for standing by, and welcome to Marinus Pharmaceuticals' fourth quarter 2020 financial results. At this time, all participant lines are in a listen-only mode. If you require any further assistance at any time, please press zero on your telephone. After the speaker's remarks, there will be a question and answer session. If you would like to ask a question during this time, simply press star and then number one on your telephone keypad. To withdraw your question, press the pound key. I would now like to turn the call over to Sasha Damouni, Vice President of Investor Relations and Corporate Communications Please go ahead.

Thank you, and good morning, everyone. With me from Marinus are Dr. Scott Bronstein, Chief Executive Officer, Dr. Joe Houlihan, Chief Medical Officer, Dr. Alex Ahmadi, Vice President of Scientific Affairs, Ed Smith, Chief Financial Officer. Also on the call to participate in the Q&A is Christy Schaefer, our Chief Commercial Officer, and Kimberly McCormick, Vice President of Regulatory Affairs. Before we begin, I would like to remind everyone that some of the statements made today could be termed as forward-looking under the securities laws. These forward-looking statements are subject to certain risks and uncertainties that are associated with our business and covered in part in the company's Form 10-K and 10-Q, as filed with the Securities and Exchange Commission. I will now turn the call over to Scott.

Thank you, Sasha. Good morning, everyone, and welcome to our fourth quarter and fiscal year-end 2020 business and financial update. The fourth quarter was another period of tremendous progress, providing strong momentum for what is shaping up to be a very exciting 2021 for Marinus. Before I touch on this year, let me share with you some of the highlights from the fourth quarter, which included compelling clinical trial and research presentations, at the American Epilepsy Society annual meeting focused on the use of Ganaxalone in treating both CDD and supra-refractory status epilepticus, raising $70 million in a December equity offering and enhancing our investor base, as well as launching an expanded access program in the United States for patients suffering from CDD who did not participate in our Phase III clinical trial. If we look at 2021, this year has already been incredibly exciting as we have announced several important milestones. Firstly, we are enthusiastic to share promising top-line data from the Phase 2 Violet Trial in PCDH 19-related epilepsy, which Alex will review following my opening remarks. Next, we are happy to report that enrollment will complete this week in the ongoing Phase II trial, evaluating efficacy and safety of adjunctive Ganaxalone treatment in patients with tuberous sclerosis complex, or TSC. The top-line study results are on track and expected in Q3 of this year. Importantly, after analyzing an interim evaluation of the COM study, the open-label Phase II trial in TSC. We are confident that the data supports moving to a global, randomized, double-blind, placebo-controlled Phase III registration trial. Joe will review this in more detail, but our hope is to meet with the FDA to discuss the Phase III trial design by the middle of Q2. In conjunction with this news, we are happy to report that the NDA for the use of Ganaxalone in CBD-related epilepsy is on track for submission by mid-2021. We also reported having enrolled our first patient in the Phase III clinical trial of intravenous Ganaxalone, the RAISE trial, for the treatment of refractory status epilepticus. We continue to screen and enroll additional patients in the trial and look to have the vast majority of sites fully activated by the middle of this year. Despite the challenges that have been created by the COVID pandemic, we continue to believe that we are on track for top-line data from the RAISE study in the first half of 2022. Before expanding on our 21 plans, I would like to turn the call over to Dr. Alex Zimetti, Head of Scientific Affairs, to discuss our results from the VIOLA trial, our proof-of-concept study in patients with PCDH-19-related epilepsy. Alex?