Mirum Pharmaceuticals, Inc.

Hello, everyone, and welcome to the Mirim Pharmaceuticals Third Quarter 2024 Financial Results and Business Update. My name is Seb, and I'll be the operator for your call today. If you would like to ask a question during the Q&A session, please press star 1 on your telephone keypad. If you would like to withdraw your question, please press star 2. I will now hand you over to Andrew McKibben, Vice President of Investor Relations and Finance. Please go ahead.

Thanks, Seb, and good morning, everyone. I'd like to welcome you to Merum Pharmaceuticals' third quarter 2024 conference call. I'm joined today by our CEO, Chris Peets, our President and Chief Operating Officer, Peter Radovich, our Chief Medical Officer, Joanne Kwan, and Eric Bierkolt, our Chief Financial Officer. Earlier today, Merum issued a news release announcing the company's results for the third quarter 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-Q 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 morning, everyone. It has been another outstanding quarter for Merum. With strong commercial execution and advancing pipeline, we have made excellent progress across our 2024 strategic objectives, accelerating our growth as a leader in rare disease. As a reminder, our goals this year have been to drive continued growth across our three commercial medicines, expand the labels for both Livmarley and Pfeffik and Kenodial and CTX, leverage the strength of IVAT inhibition in the adult settings of PSC and PVC, and continue expanding our portfolio of medicines for rare diseases. We have delivered on all of these this year. Specifically in the third quarter, all three commercial medicines saw continued growth, with net product sales of $90.3 million, an 89% increase from the third quarter of 2023. This very strong quarter was driven by the continued underlying trends across the brands, with increased PFIC and international uptake for Lipmarlin. With this strong execution from our commercial team, we are increasing our 2024 guidance to $330 to $335 million in full-year net product revenue. We have also made excellent progress with our pipeline. The list of that has been granted breakthrough designation for cholestatic pruritus and PVC based on the Vantage study interim results. As a reminder to qualify for this designation, the FDA requires preliminary clinical evidence that indicates that the drug may demonstrate substantial improvement over existing therapy on at least one clinically significant endpoint. This designation emphasizes the continued unmet need in PBC and the strong early treatment effects seen in the interim results, which have now been accepted as a late-breaking abstract at the upcoming American Association for the Study of Liver Diseases meetings. Our clinical studies in PBC and PSC remain on track with our enrollment guidance, and the Phase III EXPAND studies evaluating lipmarly in additional settings of cholestatic gyritis is now underway. For kinadiol, I'm pleased to announce we received priority review for our NDA and CTX with the PDUFA date of December 28th. And finally, I'm also happy to share that we've expanded our early development pipeline with the addition of MRM3379, a candidate for the treatment of patients with Fragile X syndrome. Fragile X syndrome is a rare genetic disorder that is the leading cause of intellectual disability that affects approximately 50,000 male patients across the U.S. and Europe with no approved therapies. Strategically, this program aligns well with the capabilities we are building in rare genetic neurology to support kinadiol on CTX. We will continue to look for opportunities to leverage our industry-leading rare disease expertise in patient settings where there's a clear need for high-impact medicines. I'm proud of all of our progress to date in 2024 and the potential ahead from here. And I'll now turn the call over to Peter to go over the commercial business.