Mirum Pharmaceuticals, Inc.

Hello and welcome to Miriam Pharmaceuticals reports fourth quarter and year end 2024 financial results and provides business update. My name is Elliot and I'll be your coordinator today. If you would like to register a question during today's event, please press star one on your telephone keypad. I would now like to hand over to Andrew McKibbin, Senior Vice President of Strategic Finance and Investor Relations. Please go ahead.

Thanks, Elliot. And good afternoon, everyone. I'd like to welcome you to Mirim Pharmaceuticals' fourth quarter 2024 conference call. I'm joined today by our CEO, Chris Peets, our Chief Medical Officer, Joanne Kwan, and Eric Bierkle, our Chief Financial Officer. Peter Radovich, our President and Chief Operating Officer, could not be with us today as he's at an international launch event in Europe this week. Earlier today, Mirim issued a news release announcing the company's results for the fourth quarter and full year 2024. Copies of this news release and SEC filings can be found in the Investors section of our website. Before we start, I'd like to remind you that during the course of this conference call, we will be making certain forward-looking statements based on management's current expectations, including statements regarding Merrim's programs and market opportunities for its approved medicines and product candidates. These statements represent our judgment as of today and inherently involve risks and uncertainties that may cause actual results to differ materially from the results discussed. We are under no duty to update these statements. Please refer to the risk factors in our latest Form 10-K and subsequent SEC filings for more information. With that said, I'd like to turn the call over to Chris. Chris?

Thanks, Andrew, and good afternoon, everyone. I'm excited to start today's call with a recap of Miriam's achievements over the last year and share an update on our strategic goals for 2025. In the short time since our founding, through our focus on bringing important medicines to patients, we have created a strong business in ultra-rare disease that is cash flow positive with an attractive pipeline and broader patient populations. We are still early in our journey, and we firmly believe that the best is yet to come. Our strategic priorities over the next two years are to, one, further the global growth of our commercial medicines, highlighted by the Marley's continued Allergy Syndrome Expansion, early P-6 success, and the recent approval of Citexly for Cerebro-Tendinous Enthomatosis, or CTX, in the US. Two, advance our high-impact pipeline, driving the list about the potentially pivotal data in adult cholestasis next year, initiating our Fragile X program for MRM3379, and completing enrollment of the EXPAND study for Lismarline cholestatic keratosis. Three, selectively pursue product acquisition and license opportunities in rare disease with potential for significant patient impact and value creation, just as we have since the launch of MIRM, And four, accomplish all of the above with continued scientific and financial discipline. 2024 was a big year for delivering on strategic goals. Total net product sales were $336.4 million, exceeding the upper end of our revised guidance range. Liv Marley finished the year with total net product sales of $213 million, including approximately $155 million from our U.S. business and $59 million internationally. In the second half of last year, PfeC New Patient Starts and International Uptake were drivers of the step up in growth. We believe the strong demand we are seeing for Lygmarly and Algea syndrome and PfeC will continue. Beyond Lygmarly, we also saw healthy growth in our bile acid medicine. We think we are starting to bend the curve here with total 2024 net product sales of $123 million. I'm also happy to say that last week, the FDA approved Citexly, which is our new brand name for Kenodial for CTX in adults. With this approval comes seven years of work on exclusivity and the opportunity to begin promotional efforts with our current field team to reach patients in this underdiagnosed condition. Ultimately, the robust growth dynamics across all of our medicines positions us well for 2025, where we expect to add close to $100 million to our top line. With anticipated net product sales between $420 million and $435 million for the year. 2024 was also a significant year for our pipeline. We expanded Livmarly's label with its approval for PFIC in both the U.S. and Europe. We initiated the Phase III EXPAND study of Livmarly in broader settings of cholestatic pruritus, a significant growth opportunity for the brand. And beyond Livmarly, we achieved positive interim results for Velixibat in both VISTA's PSC and Vantage PBC studies. resulting in breakthrough designation in PBC. And finally, we have expanded our pipeline with the addition of MRM-3379 for fragile X syndrome. This is another high unmet need orphan setting that aligns well with the capabilities we have built in rare genetic neurology for the launch of Cetexly. Pulling all this together, we are set up for an exciting 2025 and beyond, with continued strong execution in both our commercial business and pipeline of potentially high-impact medicines. And with that, I'll turn it over to Joanne to give a brief update on the pipeline. Joanne.