Marinus Pharmaceuticals, Inc.

Greetings and welcome to the Marinus Pharmaceuticals third quarter 2020 earnings call. At this time, all participants are in a listen-only mode. A brief question and answer session will follow the formal presentation. If you would like to ask a question, dial star 1 on your telephone keypad. As a reminder, this conference is being recorded. And it's now my pleasure to introduce your host, Sasha Damouni-Ellis, Vice President, Investor Relations and Corporate Communications. You may begin, Ms. Damouni-Ellis.

Thank you and good afternoon, everyone. With me from Marinus are Dr. Scott Bronstein, Chief Executive Officer, Dr. Joe Houlihan, Chief Medical Officer, and Edward Smith, Chief Financial Officer. 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, of course, are subject to certain risks and uncertainties that are associated with our business and that could cause our future results to differ significantly from those expressed or implied by the forward-looking statements. For a description of these risks and uncertainties, see the company's Form 10-K and 10-Qs as filed with the Securities and Exchange Commission. I will now turn the call over to Scott.

Thank you, Sasha. Good afternoon, everyone, and welcome to our third quarter 2020 business and financial update. I want to begin by saying this is one of the most exciting periods in Marin's history. Our team is growing quickly and working incredibly hard to help drive our operations forward. Consider what the organization has accomplished in just the last few months. Firstly, we achieved the primary endpoint in the Marigold study, the pivotal Phase III clinical trial, evaluating oral gonadolone in children and young adults with CDKL5 deficiency disorder, or CDD. Secondly, we've been awarded a five-year, potentially $84 million cost-sharing contract with BARDA for refractory status epilepticus. Finally, we remain highly focused on our Phase III IV Ginexolim clinical trial in refractory status epilepticus or the RAISE trial. Our team has continued to work diligently to finalize sites, and it is our expectation that we will have the vast majority of these sites activated by the end of Q1 2021. which is incrementally earlier than we had anticipated a year ago prior to the world being affected by COVID-19 virus. In addition to these three major accomplishments, we continue to enjoy similar success advancing our multiple programs and initiatives. I would like to highlight them for you. Our tuberous sclerosis complex, or TSC Phase II study enrollment curve, is showing marked improvement, likely due to the positive top-line Marigold study data, the additional number of new sites that have opened, our partnering with the TSC Alliance, and the easing of COVID-19 restrictions. This open-label design gives us the opportunity to perform an interim data analysis and consider an end-of-Phase II meeting with the FDA sometime in the first half of next year should we see a consistent clinical signal. We strongly believe that the dosing of these patients three times a day and up to 1,800 milligrams is a critical piece of the study design, replicating the Marigold dosing schedule. A meaningful number of patients with TSC continue to suffer from significant seizure burdens despite currently available therapies. We are on track for top-line data in the summer of 2021. Let me next turn to the VIWIT study, our Phase II double-blind placebo-controlled clinical trial examining the use of oral Ganaxolone in PCDH19 patients. As a reminder, a proportion of children with PCDH-19 suffer from intractable cluster seizures despite treatment with multiple anti-epileptic therapies. Recruitment completed in July, and all patients will finish the double-blind portion of the trial in Q1 of 2021. Once again, the dosing paradigm is identical to the Marigold study. The VILETS study will not only evaluate the efficacy of canaxolone when compared to placebo, but it will also examine the role of allopredenolone sulfate as a biomarker-directed therapy. We currently believe that both our IV and oral programs will yield important therapeutics in major markets around the world. Accordingly, we successfully submitted the EU status epilepticus request for scientific advice with the European Medicines Agency and expect to engage in a pre-submission meeting to review that trial design in the first quarter of next year. As a reminder, the European regulators prefer clinical trial programs that compare a novel therapy to a currently existing agent. We will share the study protocol in detail with you once we have met with the EU regulators and are confident in our path forward. Let me provide a brief overview of both our IV and oral clinical programs before turning the call over to Joe for additional detailed discussion. Starting with our IV franchise, we have been making tremendous progress with our status epilepticus program. Having quickly satisfied the FDA's protocol-specific questions, we now have activated our first sites, and those sites are ready to screen and enroll refractory status epilepticus patients. We expect the number of eligible sites to grow steadily over the next several months. Over 75% of the sites have been approved and preselected to participate in the study, And we expect the vast majority of these sites to be finalized by the year end and open by the end of first quarter 2021. The Marinist team has done a tremendous job meeting with sites and choosing the best centers to participate in this study. The team of recently hired medical science liaisons, or MSLs, have partnered with our clinical operations team and together have been instrumental in focusing on neurocritical care and neurointensivists since those physicians are on the front lines of treating patients experiencing RSAs. Our clinical operations team and our MSLs are also increasing awareness and engagement of the RAISE trial through the review of the Phase II clinical trial data and critical scientific exchange, respectively. As a reminder, our patient population in the RAISE trial is primarily nonconvulsive status epilepticus, and that is expected to be a determining factor in driving enrollment. We believe that the clinical trial paradigm used in the Phase II study drove rapid enrollment, and we will use a very similar informed consent procedure and treatment algorithm in the Phase III study. We firmly believe that canaxolone can provide a rapid onset of action, have a durable effect in patients with status epilepticus, and potentially play an important role in preventing the devastating consequences of uncontrolled seizures. We believe that our trial sites share our enthusiasm in this novel therapy. There is no question that we are competing with COVID-19 for both resources and time across U.S. hospitals. However, despite the intense pressure that U.S. hospitals are feeling today, our investigator community and interested sites have been highly engaged with Marinus. Our team has continued to be creative and work hard to keep our site activations on track, and as a result, we still expect to report top-line data in the first half of 2022. As an interesting aside, several physicians have requested, on an emergency IND basis, access to Ganaxalone to treat patients with super refractory status epilepticus. Our entire team takes such requests very seriously, and we feel it is our obligation to offer an option for patients who have been unable to come off general anesthesia following their bout with status epilepticus. We have thought very hard about how to best dose and treat these patients. I want to thank Dr. Joe Houlihan and the entire clinical team for their efforts. These patients are truly left with no other alternatives. We'll share more details on this at AES, where we expect to have a notable presence this year. To help lead our SE programs, Dr. Henry Bukavage, who served on our Scientific Advisory Board and as the principal investigator in our Phase II RSE study, has been appointed Vice President of Clinical Development. Henry's knowledge surrounding neurosteroids, his vast clinical experience, and his insights and contributions to our scientific approach will help us further advance Ganaxalone in additional indications in clinical trials, including the upcoming study in Europe that I just mentioned. Furthermore, we are planning a trial in an earlier treatment line to RSC known as established status epilepticus. The vast majority of these patients are suffering from convulsive status and have been treated with and failed benzodiazepines. Treatment success rates in established status are slightly below 50%, and as a result, it is equally important unmet medical need. We estimate this market to be approximately 50,000 to 75,000 additional patients in the U.S., and these patients are primarily treated in the emergency room. Further details of this trial design will also be discussed at AES. Finally, we have an ongoing preclinical work and intramuscular formulation of the Naxalone and continue to drive additional formulation partnerships and research projects to enhance our intermediate and long-term strategies for the oral franchise. Before I leave the IV program, I would like to spend a little time highlighting our BARDA contract that we announced in September. As briefly mentioned, BARDA agreed to fund up to $51 million of an $84 million contract for programs related to RSC development. The agreement covers a base period during which BARDA will provide subject matter expertise and $21 million to fund on a cost share basis, additional preclinical work, and the company's planned Phase III clinical trial of Ganaxloan for the treatment of RSCs. We believe that this preclinical work and our planned phase three trial will be the foundation of a long-lasting business relationship with the BARDA team. We've already held our BARDA kickoff, and it was a large collaborative team effort comprised of several scientists and multiple other experts from both sides. We see the BARDA team as an important resource, and we are pleased to be working with them. Their interactions to date have been thoughtful and extremely valuable to our scientific process. We are grateful to BARDA for their collaborative approach throughout this process and the opportunity to continue to innovate in the field of seizure disorders. Let me turn to the oral franchise and the tremendous progress we have made in the last several months. As I previously noted, we recently reported positive top-line data from the pivotal Phase III Marigold study. This was the first double-blind placebo-controlled trial to provide evidence of efficacy specific to the CDD patient population and the first Phase III trial to examine three-times-a-day dosing of gonadsalone in pediatric patients. We had a chance to share the top-line data with the CDD community in October. The next few months will be equally exciting. Marinus is planning to meet with the FDA in the first quarter of 2021 to support an NDA submission by mid-2021. We also expect to launch our expanded access program in early December this year, enabling patients who are unable to participate in the Phase III trial access to the drug. Our early feedback on the EAP program has been extremely positive to date. We feel that this is important for us as an organization and as part of the biotech community to offer gonadsalone to patients who are suffering from CDD and were not able to participate in our Phase III trial. We expect further external commercialization discussions in CDD, especially as it pertains to strategic alliances and our global commercialization strategies. as well as continued interaction with the community of medical professionals focused on treating epilepsy at the American Epilepsy Society annual meeting in December. We believe that AES provides a fantastic platform to discuss novel anti-epilepsy therapies, and we are focused on making that meeting an important success for Ganaxilin. We plan to present further details of the Marigold study, including pharmacokinetic data, at AES in December, which will be a critically important roadmap to the future development of oral gonadsalone. We believe the data will strongly support our confidence in the results of the Marigold study as a pivotal trial. We will be holding a separate investor event at AES, which Sasha will go into further details later on. Our CDD commercial strategy continues to evolve, and I am pleased to announce that concurrent with our Q3 business update this afternoon, we also announce and welcome Christy Schaefer to Marinus as our Chief Commercial Officer. Christy's rare disease expertise and tremendous sales and marketing background provides the experience and leadership needed to prepare the U.S. commercial launch of Ganaxalone and CDD and a potential debut of Ganaxalone in status epilepticus soon after. Christy has spent almost 20 years in the biotech, pharma, and medical device spaces, most recently at Alexion Pharmaceuticals. Christy is energetic and a fantastic leader, and we are thrilled to have her on board. We've also begun to build our team of MSLs to help drive a publication strategy, continue to build our relationship with advocacy groups, and expand our scientific plan for 2021. This team has been instrumental in helping identify clinical research sites for all our trials and leading our AES presence. On the IP front, as noted in our 10-Q, Ovid Therapeutics contacted us and disclosed that it owns two recently issued method of use patents that include claims to the use of Ganaxilone to treat CDD and PCVH-19. As you are aware, we have been developing Ganax Loans for the treatment of focal onset seizures and rare genetic epilepsy since 2005. Meanwhile, both OVID patents originated from a provisional patent application that was filed on August 11, 2016. We do not believe the OVID patents will impact our timeline for development, NDA submission, and regulatory approval. nor do we believe that the OVID patents will delay our planned launch. I have asked our lawyers to investigate these patents and all the possible options with respect to them, including challenging the patents at the USPTO and or in the courts. Another solution could certainly involve a royalty payment based on an in-licensing of the patent states. At this time, we are not going to say anything further, but we are confident that our plans for GNAX loans will not be delayed or disrupted. Regarding our partnership conversations, including those for the ex-U.S. rights to CDDs, those are ongoing. We remain committed in this process and other means of non-diluted financing for the companies. These endeavors are the product of our broader clinical strategy to unlock the potential of Ganaxalone across a range of rare seizure disorders. Our plans to expand the Ganaxalone franchise into TSC will continue to move forward. We remain on track to report top-line data in the first half of 2021. Finally, our Violet study is also making steady progress. Earlier this year, we transitioned this trial to a proof-of-concept study evaluating allo-S as a biomarker in patients with a confirmed PCVH-19 mutation. We believe the trial has the potential to detect a meaningful signal as a proof-of-concept for the allopredaniline sulfate biomarker hypothesis, as well as for the three-times-a-day dosing regimen, which is intended to inform future development and indication selection. This trial is on track and data should report out in the first half of 2021 with the company's intention to subsequently submit the results of the study for publication. With that, I would now like to turn the call over to our Chief Medical Officer, Joe Houlihan. Joe?