Mirum Pharmaceuticals, Inc.

Hello everyone and welcome to the Mirren Pharmaceuticals, Inc. second quarter 2021 business update. My name is Simona and I will be your coordinator today. You will have the opportunity to ask a question after the presentation. If you would like to ask a question, you may do so by pressing star followed by one on your telephone keypad. I will now hand you over to your host, Ian Clements, Chief Financial Officer, to begin. Ian, please go ahead.

Thanks, Simone, and good afternoon, everyone. I'd like to welcome you to Mirren Pharmaceuticals' second quarter 2021 conference call. I'm joined today by our president and CEO, Chris Peetz, and our chief operating officer, Peter Radovich. Earlier this afternoon, Mirren issued a news release announcing the company's results for the quarter ended June 30, 2021. Copies of this news release and SEC filings can be found in the investor section of our website. Before we begin, I'd like to remind you that during the course of this conference call, we will be making certain forward-looking statements about MIRM, and that program is based on management's current expectations, including statements regarding MIRM's business plans, development programs, strategies, prospects, commercialization and market opportunities, financial forecasts and guidance, anticipated FDA interactions or announcements, and certain other business methods. These statements are subject to numerous risks and uncertainties, and actual results could differ materially from the results anticipated by these statements. Investors should read the risk factors set forth in Miriam's 10-Q for the quarter ended June 30, 2021, and any subsequent reports filed with the SEC. All statements are made as of today, August 5, 2021, and are based on information currently available to us, and Miriam does not undertake any obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law. With that said, I'd like to turn the call over to Chris Peetz. Chris?

Thanks, Ian. Good morning, or good afternoon, to everyone. Thanks for bearing with our technical difficulties getting kicked off. With our PDUFA date of September 29th for Mirelixibet, our focus is squarely on the realization of this upcoming potential launch. Algeol syndrome is the leading genetic cause of pediatric liver transplant, many of these driven by the severe pruritus and symptomatic burden of disease. These patients are waiting for new treatment options. We estimate that algeol syndrome alone may be a more than $500 million opportunity for Mirelixibet, and we are launch ready and now have assembled a team and financial resources for successful commercialization, all while expanding our pipeline of potentially registrational studies in other rare liver diseases. Miriam is focused on advancing life-changing medicines. Today, we will recap and provide context around some of our most exciting recent steps as we continue to realize our vision of being the leading rare liver disease company. I will cover our regulatory and pipeline updates, as well as the additional value we continue to build within NIRM's clinical programs. Peter will share updates on commercialization, and Ian will summarize our financial highlights. With our Purdue-to-date Tremorilixibet and cholestatic pruritus associated with Allergy Syndrome next month, our team is ready to launch upon a potential approval. Our regulatory review remains on track. and we've been able to accommodate all inspections to date and the FDA has continued to indicate no plans for an advisory committee. If approved in the U.S., Merilixibat would serve as the first treatment approved for patients with Algeo Syndrome. In the ICONIC study, which served as a pivotal study, highly significant improvements in pruritus were observed with more than 80% of participants achieving a one-point reduction in itch score. We have six years of follow-up data and see durable responses in trial participants across the extensions of all three randomized studies of Mariluzabat and Allergy Syndrome. An approval and subsequent commercial launch would mark an important milestone for us at NIRM, and more critically, an important milestone for patients living with this terrible disease. We remain committed to further building on the body of evidence supporting Mariluzabat's potential in Allergy Syndrome. In June, we presented an integrated safety analysis of Merilixibab in patients with Allergil Syndrome at the EZL Congress. These data resulted from five years of Merilixibab treatment across 86 patients and continues to reinforce our understanding of the drug safety and tolerability profile in patients with Allergil Syndrome. Mariluxabat was well tolerated in these patients for more than five years, with the most common treatment emerging adverse events reported being diarrhea and abdominal pain. An iconic diarrhea incidence was at similar rates between Mariluxabat and placebo during the placebo-controlled portion of the study. Also in June, we presented Mariluxabat transplant-free survival data for progressive familial intraparticulospaces type 2, or PfeC2, at the World Congress of Pediatric Gastroenterology, Hepatology, and Nutrition Annual Meeting. As detailed in our presentation, Merilithabat-treated patients who achieved 75% serum bile acid reductions were shown to have 100% five-year native liver survival. These PFIC-2 data are under review with the European Medicines Agency. Building on that, our Phase III March PFIC study continues to advance well. with greater than 65 patients randomized with PFIC subtypes 1, 2, 3, and 4 across two cohorts. As a reminder, March PFIC evaluates a higher dose of Mariluxibet and has been shown to drive greater bile acid clearing. We plan to extend enrollment and expect top-line data in the second quarter of 2022. Further broadening our efforts in pediatric liver disease, our Phase IIb EMBARQ study Evaluating Muralixibet and Biliary Atresia is progressing nicely as well, and we anticipate top-line data in 2023. In adult liver disease, we are excited about advancing our clinical programs for Gullixibet, which we believe is another potentially transformative treatment for several cholestatic liver diseases. Earlier this year, we launched our Phase IIb VISTA study for adults with primary sclerosing cholangitis, and a Phase 2b OHANA study that will evaluate volixibat in intra-hepatical spaces of pregnancy. A Phase 2b study in primary biliary cholangitis is targeted for later this year. We've incorporated regulatory feedback into the design of these volixibat studies, all of which are potentially registrational. We expect interim analyses for the VISTAs and OHANA studies to occur next year. And before heading off to Peter to discuss launch readiness, I want to touch on Mirim's commitment to ensuring broad, global access to Mariluxabat for Allergy Syndrome patients. To facilitate the international reach of Mariluxabat, we recently entered into an exclusive licensing agreement with GC Pharma to develop and commercialize Mariluxabat in South Korea. This builds on our partnership with Cambridge Pharmaceuticals in Greater China, which we announced in April. Both GC Pharma and Cambridge are leaders in rare disease and share our passion to advance life-changing medicines, and we'll pursue expedited approval pathways for Merilixivet for Allergy Syndrome based on our U.S. filing package. We see the high interest level of our partners as testament to the potential of Merilixivet for pediatric cholestasis patients. On that note, I'll turn it over to Peter, who will provide an overview of our launch readiness activities. Peter?