Marinus Pharmaceuticals, Inc.

Greetings and welcome to the Mariners Pharmaceuticals second quarter 2021 business update call. At this time, all participants on a listen-only mode. After the speaker's presentation, there will be a question and answer session. To ask a question during the session, you will need to press star 1 on your telephone. If you require any further assistance, please press star 0. And now it is my pleasure to introduce your host, Sasha Dimoni Ellis, Vice President of Corporate Affairs and Investor Relations. You may begin, Ms. DeMonellis.

Thank you. With me from Marinus are Dr. Scott Bronstein, Chief Executive Officer, Dr. Joe Houlihan, Chief Medical Officer, Steve Sandsill, Chief Financial Officer, and Kimberly McCormick, Senior Vice President of Regulatory Affairs. Also on the call is Christy Schaefer, Chief Commercial Officer, who will be available during Q&A. Before we begin, I would like to remind everyone that some of the statements made 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 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, Scott Braunstein. Scott?

Thank you, Sasha, and welcome to our second quarter 2021 business and financial update. Building on our busy first quarter, we've continued to make significant progress across our oral and IV clinical programs. Just last week, we made two important announcements. First, we entered into an exclusive collaboration with Orion Corporation for European commercialization of Ganaxalone in CDKL5 deficiency disorder, tuberous sclerosis complex, and refractory status epilepticus. We believe that the Orion team will be an excellent strategic partner for the Ganaxalone franchise. We also announced that we have submitted a new drug application to the FDA for Ganaxalone for the treatment of seizures associated with CDD. Within 60 days of the submission, we expect to receive a filing notification letter from the FDA. That communication should occur prior to the end of the third quarter. Not only are these two events meaningful accomplishments, but they represent important milestones in the evolution of our business. The European collaboration provides significant capital to continue advancing the development of Ganax Loan across Europe, and the FDA acceptance of the NDA for filing provides an opportunity to draw on the Oak Tree Credit facility, extending our cash runway and allowing us to continue to expand our pre-commercial activities. In addition, the organization is executing on a number of regulatory milestones. During Q2, we submitted our Phase III TSC trial design to the FDA, and the agency indicated that they were in overall alignment with our clinical development plan. I will leave the details to Kim. We continue to prepare for the TSC Phase III trial that we have named Trust TSC with an expected first patient enrolled in the fourth quarter of this year. I would like to thank the TSC Alliance whose support has been instrumental in driving this program forward. Let me move to an important update for the IV franchise. We received a notice of allowance for the patent application for the RSC IV dosing and method of treatment from the US Patent and Trademark Office. We anticipate the patent to be formally granted later in the third quarter. When granted, we expect the patent to run through 2040. The patent application highlights the dosing regimen and method of treatment for RFC and aligns with the phase three trial. We believe this patent has the potential to create meaningful shareholder value following the completion of our phase three US registrational study, the RAISE trial. The organization is continuing their work on other key initiatives. Plans for the CDD launch are progressing. we look towards an FDA action date on the Ganaxalone NDA by the end of the first quarter of 2022. As a reminder, FDA approval would result in a three-month period for DEA scheduling, followed by a mid-2022 launch. As mentioned, we have signed a European collaboration agreement with Finland-based Orion Corporation. After a diligent and competitive process, we believe that Orion checked all the boxes that we were looking for. A strong presence across Western Europe in rare neurological disorders, a desire to be a leader in orphan diseases and epilepsy, significant expertise in the hospital and the ICU, as well as an extensive European commercial infrastructure. Importantly, Orion offers a strong cultural fit to our organization, and we look forward to working together towards future milestones. Orion has been granted exclusive rights to commercialize the oral and IV formulations of Ganaxalone in the European Economic Area, the UK, and Switzerland, where they will be responsible for all reimbursement and pricing approvals. We've received approximately $30 million as an upfront payment and are eligible to achieve up to approximately $115 million in R&D reimbursement, development and commercialization payments, as well as tiered royalties on future net sales that could reach the high teens for the oral programs and low 20s for the IV program. We believe that this deal structure strikes the right balance on upfront payments and royalties, allowing Marinus and our shareholders to benefit from the long-term success of the Binaxalone franchise. Importantly, this agreement supports a mid-22 European launch of oral Ganaxalone for CDD and underscores our commitment to the CDD patient community. We are on track to file a European marketing authorization application for Ganaxalone in CDD by the end of Q3 2021. In the near term, we see this collaboration having the potential to provide an important new alternative for patients and families living with CDD throughout Europe. Both organizations are committed to improving the availability of Ganaxolone to refractory CDD patients, including the desire to support a geographically broader expanded access program. In keeping with our commitment to the CDD patient community, we plan to support a collaborative observational study in CDD sponsored by the Lulu Foundation, a nonprofit organization dedicated to advancing research to better the understanding and development of therapeutics for CDD. This observational study is designed to provide a high-quality natural history database on a variety of clinical outcomes to better describe disease severity, unmet need, and inform future trials. We are excited to be a part of this research consortium and are committed to contributing critically necessary resources to the CDD community. Moving to our TSC program, we remain on track to announce top line data from the open label phase two trial in the third quarter. As I noted earlier, we believe that we have reached overall alignment with the FDA on an appropriate regulatory path. We are also expecting to obtain scientific advice from the EMA and now expect that meeting to occur in the first quarter of 2022. We continue planning for phase three site initiations to begin in the third quarter with the first patient expected to be enrolled in the fourth quarter of 2021. We are currently targeting initial sites in the U.S. and will add EU sites following our regulatory interaction with the EMA. We plan on providing enrollment timelines for a trust TFC trial later this year. Turning to our IV programs, Due to COVID-19 priorities in several major academic medical centers participating in the RAISE trial, we've experienced site initiation and enrollment delays. We believe these delays are primarily caused by staff turnover due to COVID-19 fatigue and the need for clinical sites to devote significant resources to patients with COVID-19. These challenges most acutely impacted second quarter site enrollment. Fortunately, our clinical operations team believes that the majority of these issues are now behind us. Consequently, we expect to have the vast majority of our sites open by the end of third quarter 2021 with our top line rate trial data readout in the second half of 2022. We've already seen July site initiation numbers improving, giving us confidence in this timeline. We are actively engaged with our sites and will provide additional updates during our October R&D day. As a reminder, planning continues for the RAISE-2 trial to support European registration of IV Ganaxalone in RSE. This trial is on track to launch in the first half of 2022. The Phase II Reset Trial of Adjunctive Ganaxalone in Established Natus Epilepticus, or ESC, is planned to begin U.S. enrollment in the first half of next year. Before turning the call over to Kim, I wanted to share our thinking on Lennox-Gastaut Syndrome, or LGS. Of note, six CDD patients with an LGS phenotype were treated in the Marigold study. We have submitted that data for presentation at the annual American Epilepsy Society meeting. We have also reviewed the company's prior Phase II LGS study and noted that the majority of patients did not achieve an adequate serum blood concentration of Ganaxalone. We believe that our current TID dosing regimen, including an appropriate titration schedule, should lead to higher blood concentrations that were seen in that earlier study similar to those observed in the Marigold study. That said, the LGS development landscape is highly competitive, and patients and families suffering from LGS are looking for the best possible anti-epileptic therapy. Our team is committed to the right investment in LGS and that our clinical work should be closely tied to Marinus' next-generation oral program. We believe that these next-generation formulations, or novel pro-drugs, will deliver a solid drug candidate with improved bioavailability, more reliable steady-state plasma concentrations, and create the potential for incremental dose titration and higher efficacy. Further on the new formulation front, we continue to evaluate several platform technologies for oral and iVegan Absalom. We plan to evaluate these new formulations in Phase I studies in 2022 and ultimately additional refractory epilepsy indications. We expect to advance at least one of these formulations into the clinic next year and currently have reason to believe that it is more likely that we will advance two formulations in 2022. We are enthusiastic that our investments are the first step to a second-generation platform. More to come on our reformulations during Marinus' R&D day. I also want to welcome Dr. Santiago Arroyo to the Board of Directors. Dr. Arroyo brings a distinguished career in academic neurology and clinical research and development, including his leadership in the neurology department at the John Hopkins Hospital. In addition, he has extensive experience in epilepsy drug development, contributing to numerous new therapeutic alternatives. With that, I would like to now turn the call over to Senior Vice President of Regulatory Affairs, Kim McCormick, for an update on our regulatory interactions. Kim?