Marinus Pharmaceuticals, Inc.

Greetings and welcome to the Marinus Pharmaceuticals first quarter 2021 business update call. At this time, all participants are in a listen-only mode. A brief question and answer session will follow the former 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. As a reminder, this conference is being recorded. And it is now my pleasure to introduce your host, Sasha Kamouni. Vice President, Investor Relations, and Corporate Communications. You may begin, Mr. Mooney.

Thank you. With me from Marinus are Dr. Scott Rodstein, Chief Executive Officer, Dr. Joe Houlihan, Chief Medical Officer, Christy Schaefer, Chief Commercial Officer, Steve Fansfield, Chief Financial Officer, and Kimberly McCormick, Vice President, Regulatory Affairs. 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 forms 10-K, 10-Q, and 8-K. I will now turn the call over to Scott.

Thank you, Sasha, and welcome to our first quarter 2021 business and financial update. This year is off to a tremendous start. We've continued to make progress across our oral and IV clinical programs, have intensified preparations for a commercial launch, deepened discussions with potential European partners, have made exciting headway in our next-generation formulation research, and have strengthened our management team, scientific advisory board, and board of directors. To support this continued progress, today we announced $125 million credit financing that is being funded by Oaktree Capital Management, a global asset management firm specializing in alternative investment strategies. This financing provides us with access to additional capital to support our research programs and commercialization efforts, as well as extending our cash runway through the end of 2022. The completion of this agreement assists in our ability to monetize the rare pediatric disease priority review voucher we anticipate would be granted with this potential approval of Ganaxolone next year. We believe the financing exemplifies Oaktree's confidence in our NDA submission, our company's ability to deliver on upcoming clinical milestones, while providing capital to support our ongoing and future research and development programs, as well as preparing for our first potential commercial launch. The NDA for the use of Ganaxalone in CDKL5 deficiency disorder, or CDD, is on track for submission by the middle of this year. This is the first step in developing a sustainable oral franchise to address the meaningful opportunities that we envision over the long term. As a reminder, the sufficiency of the Marigold data set to support approval will be determined during the FDA review process. The Marigold study showed strong efficacy and a favorable safety profile, and the open label data from that study continues to display durability that has been noted since Ganaxolin's early preclinical studies. Let me give you a quick update on Europe. The company has had encouraging interactions with the European Medicines Agency surrounding our planned oral Ganaxalone EU submission, and we remain on track to submit a pre-marketing authorization application by the end of the third quarter. We continue to have constructive discussions with potential European commercial partners and hope to have a partner selected over the coming weeks to months. we would expect oral Ganaxalone, if approved, to be commercially available in Europe beginning in the middle of next year. We have been intensifying our commercial planning efforts in front of a potential mid-year 2022 CDD launch in the U.S. This is the first call that you will hear prepared remarks from our Chief Commercial Officer, Christy Schaefer. Christy has been building a fantastic team filled with experts in both the orphan disease space as well as the hospital arena. Our medical science liaisons are working diligently to prepare for their educational efforts with the clinical community. Our message will focus on the mechanism of action of Ganaxalone and its modulation of the extrasynaptic GABA receptor and the unique characteristics and results of that binding. We believe that Ganaxalone will have a differentiated profile based on efficacy, safety, and durability. More to come on our commercial plans from CRISPY. Let me move to our TSC program. We expect to share top-line data from the 23-patient Phase II study with the investment community around the middle of the third quarter. We have completed the study design for our planned Phase III trial of Ganaxalone in tuberous sclerosis complex, or TSC, and an end-of-Phase II meeting with the FDA based on an interim data analysis is expected early in the third quarter. As a result, we are planning for site initiations to begin for the Phase 3 trial in the latter portion of the summer, while the first patient enrolled is anticipated in the fourth quarter of 2021. We are targeting completion of site selection by the end of Q3, and we expect to share our timelines for the Phase 3 study by the end of September. We also plan to meet with EMA early in the fourth quarter to obtain scientific advice on the phase three trial and clinical development program. We plan to submit an orphan drug designation request for the use of Ganaxalone in TSC in both the US and EU later this year. We believe Ganaxalone has the potential to play a critical role in the chronic treatment of refractory epilepsies. To further support our expanded clinical efforts, We announced today that we have strengthened our scientific advisory board with the appointment of several leading experts in clinical neurology and pediatric seizure disorders. Joining our SAB are new members, Dr. Elizabeth Thiel from Massachusetts General Hospital and a professor of neurology at Harvard Medical School, Dr. Ilana Pisana-Knight, pediatric epileptologist in the pediatric epilepsy section at the Cleveland Clinic, and Dr. Nicola Specchio, head of the epilepsy unit in the Department of Neuroscience at Bambino Gesu Children's Hospital, Rome, Italy. I'd like to shift gears and move to the IV program. Our Phase III clinical trial of IV Ganaxalone for the treatment of refractory status epilepticus, or RSE, the RAISE trial, continues to advance. I know that many of you have been tracking both the rate of new COVID infections and hospitalizations in the U.S., and we are thankful that the numbers continue to significantly move in the right direction. COVID hospitalizations are below 40,000 U.S. patients for the first time in almost a year and showing continued progress, which is encouraging news. Despite these challenging hospitalization numbers, interest in the study over the past several months has continued to be robust. Our clinical operations team has done a tremendous job to open sites in major academic centers in the middle of this pandemic. We continue to open sites and evaluate enrollment accordingly. We are happy to report that currently total patient per site numbers are tracking in line with our expectations thanks to the diligent interactions from our clinical development team, including Dr. Henry DeCavage and Dr. Maja Gasior. It is still early in the study. However, at this time, we are reiterating top line data readout for the RAISE trial in the first half of 2022, despite the persistent COVID headwinds that have affected site activations for the past six months. More than half the trial sites are expected to be open by the end of the second quarter, with the vast majority open by the third quarter. Planning also continues for a separate European RSC trial recently named the RAISE-II trial to begin in the first half of 2022. A meeting was recently held with the EMA where we engaged in a very constructive dialogue and that Joe will describe in further detail. Our European strategic discussions encompass both the oral and IV franchises, and we believe a partner would bolster our ability to execute on a second appropriately powered IV registrational trial. Our hope is that we will have a partner in place to help fortify our execution effort, specifically around site selection and medical education. We currently believe that the RAISE-II trial would not only serve as a pivotal registrational trial for European approval, but has the potential to have an important clinical implications for the global market. More to come from Joe. As we continue to build the team and strengthen the organization, this will be the first Business Update conference call for a newly appointed CFO, Steve Fanstiel. Steve has been a major contributor in the completion of the credit financing, and his amazing effort and strong leadership skills could not have come at a better time for the organization. Steve has only been with us a few weeks, but the entire leadership team is thrilled with his ability to jump right in and contribute in a meaningful way. Finally, we are pleased to welcome Dr. Sarah Nocture to our board of directors. Sarah brings tremendous experience and success in managing global regulatory and drug development and has been involved in the approval of several novel drugs for rare diseases. Sarah is currently serving as an Alnylam Pharmaceuticals Chief Diversity, Equity, and Inclusion Officer. We plan on tapping into our expertise as we continue to build our corporate culture and drive to achieve our core values at Marinus, commitment, innovation, and community. I would now like to turn the call over to Christy Schaefer. Christy joined us as our first Chief Commercial Officer six months ago. I am pleased to have her provide more on her early thoughts as we plan for our first potential commercial launch.