Marinus Pharmaceuticals, Inc.

Greetings, and welcome to the Marinus Pharmaceutical Third Quarter 2021 Business Update Conference Call. At this time, all participants are in 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 the session, please press star 1 on your telephone keypad. To withdraw your question, press the pound key. And now it's my pleasure to introduce your host, Sarah Damone-Ellis, Vice President of Corporate Affairs and Investor Relations. You may begin, Ms. Dimoni Ellis.

Thank you, and good morning, everyone. With me from Marinus are Dr. Scott Broadstein, Chief Executive Officer, Dr. Joe Houlihan, Chief Medical Officer, and Steve Fansfield, Chief Financial Officer. 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 third quarter 2021 business and financial update. Our teams continue to work diligently throughout the quarter on the regulatory, commercial, legal, and clinical fronts. As it's only been a few weeks since our R&D day, I will limit my initial remarks to our most recent corporate news items. Trials in both our oral and IV franchises remain on track as we continue to advance Ganaxalone across multiple indications, where there is high unmet medical need, both in the acute and chronic setting. In addition, we achieved meaningful advances on the regulatory and patent exclusivity front this quarter and continue to make important progress with our patient-centric advocacy strategy, which I will discuss in further detail shortly. We are pleased to announce that the European Medicines Agency has validated the marketing authorization application for Ganaxalone for the treatment of seizures associated with CDD. The application is being evaluated under accelerated assessment. As a result, the CHMP opinion is expected in the second quarter of 2022 and the European Commission decision early in the third quarter. We're also delighted to share that the European Commission has officially granted orphan drug designation to Ganaxalone in tuberous sclerosis complex. We've now been granted orphan disease status for oral Ganaxalone in CDD and TSC in both the U.S. and Europe. As a reminder, we previously were granted orphan disease status for IV Ganaxalone in refractory status epilepticus in the United States. Let me add some additional color and briefly highlight the growing momentum for the IV franchise. We have seen continued improvements in site activation for our Phase III RAISE trial in refractory status epilepticus after facing several challenges in the second quarter, the result of resource allocations and other direct impacts of the COVID-19 pandemic. We now have 46 sites activated, 44 sites currently recruiting, six of which were added in the month of October. We're pleased to see the success of our clinical operations team, their growing ability to counter the difficult challenges caused by the pandemic as they remain diligently focused on advancing this critically important program. I would like to share some of our excitement on other key corporate objectives. We are actively working on second-generation formulations and prodrugs of Ganaxalone, as well as exploring opportunities to reach more patients around the world. We've been collaborating extensively with the Orion team as they prepare Ganaxalone's commercial readiness in the European Union. In addition, we believe that there is a greater global opportunity for the Ganaxalone franchise. For example, in our discussions with the CDKL5 Alliance in China, We have learned that there are approximately 350 patients registered with the Alliance and that 80 of those families have been considered active, and the number is growing rapidly with the adoption of genetic testing. This is just a single example of why our organization will continue to focus on improving global access to Ganaxalone in its current lead and future indications. December will be an equally busy month for the teams. We will have a strong presence at the American Epilepsy Society 2021 annual meeting where we've had nine abstracts, including a late breaker, accepted for presentation. We are planning to host a virtual event for analysts and investors during the meeting on Monday, December 6th. Before turning the call over to Joe, let me quickly review our overall strategy and a few additional thoughts. Marinus is focused on the treatment of rare epilepsies that have limited therapeutic options. We believe that the Naxalon's novel mechanism of action, its strong efficacy in our lead potential indication, CDKL5 deficiency disorder, and the encouraging safety profile differentiates it in the treatment of CDD and other refractory epilepsies. We have filed oral Ganaxalone in its lead indication, CDKL5, with the FDA and have a PDUPA target action date scheduled in March. We have also recently revealed our proposed trade name, Zitalmi. This brand name has been established and the trademark is registered, subject to final FDA approval. We have a fully staffed medical science liaison team in place, who are actively engaging the medical community at CDD Centers of Excellence across the United States. Our commercial team continues to expand with the vast majority of key leaders in place. Christy Schaefer, our Chief Commercial Officer, and her team continue to prepare for our expected mid-2022 launch following FDA approval and DEA scheduling of Ganaxilin. Steve will be sharing some of the pre-commercial highlights from the quarter and his remarks. Our goal following a potential approval next year is to establish Ganaxalone as a central pillar in the comprehensive management of CDD and to ensure Ganaxalone is available commercially both in the U.S. and New Europe. One additional reminder, if Ganaxalone is approved for the treatment of CDD in the United States, We expect to receive a rare pediatric disease voucher, which will play an important role in bolstering our long-term financial position. Let me move to our development strategy. We are committed to undertaking a series of clinical trials to investigate Ganaxalone and several new indications. We are ready to initiate a registrational phase three program in tuberous sclerosis complex, the TRUST TSC trial, with our first patient expected to be enrolled early in the first quarter of 2022, with the data from that trial expected in the first half of 2024. Our team is already working diligently to meet or exceed those timelines. Joe will review the key learning from our Phase II study and the details of the Phase III design in his prepared remarks. Turning back to the IV franchise, we believe that Canaxalone can dramatically improve outcomes of patients suffering from status epilepticus and prevent the escalation of treatment to IV anesthetics in a significant number of patients. There are three distinct trials that will play a role in our future success. Our Phase III RAISE trial in refractory status epilepticus continues to advance, and we expect to complete this trial in the second half of 2022. Our second Phase III trial, the RAISE II study, remains on track, and we anticipate patient enrollment to begin in the first half of next year. This trial will not only serve as a critical piece of the European approval process, but has the potential ability to broaden the use of ibuprofen in the U.S. patient population. our third status trial is the reset trial and in contrast to the raised trials which are focused on refractory se reset is focused on established status epilepticus which occurs early in the status continuum we anticipate u.s enrollment to begin in the first half of 2022. finally our new formulation programs continue to progress nicely One of our goals is to develop a second-generation formulation with an improved clinical profile, including better bioavailability and a broader titration schedule for physicians. We expect that healthy volunteers will be dosed with our new formulation in the first half of 2022. Our strategic CMC and clinical operations team have been working together to help coordinate the future of our next-generation franchise. We have a second candidate that should enter phase one by the middle of next year. We are also focusing research efforts in the first half of 2022 on developing a sustained release formulation. If studies of a new oral formulation demonstrate the expected TK profile, we plan to move quickly to a phase two trial in Lennox-Gastaut syndrome and would expect to begin that study in the second half of 2022. Joe will discuss this topic in greater detail shortly. Beyond our clinical trials, we are making great strides, strengthening our intellectual property and enhancing our advocacy efforts. We've recently been granted a new method of use patent for ibuprofen, which specifies a dosing and method of treatment in status epilepticus. That patent expires in 2040, and we expect to continue our investment in the IV franchise, which we believe is supported by the newly issued IP. With regard to our advocacy efforts, our view is that every patient matters, particularly in rare and orphan disease work. Our goal is to help educate, engage, and empower patients, their families, caregivers, and the advocacy community. To that end, we've joined forces with the Lulu Foundation and six other biotech and pharmaceutical organizations to undertake a key observational study to better understand the natural history and the utility of various clinical assessments in patients with CBD. I will leave the details of the collaboration to Joe. We are privileged to be partnering with the Lulu Foundation in an effort to improve the scientific knowledge about this serious disorder and support the CBD community. Now, I would like to turn the call over to Joe for some additional comments.