Prospectus supplement summary
This summary highlights information contained elsewhere or incorporated by reference in this prospectus supplement. This summary does not contain all of the information you should consider before investing in our common stock. Before you decide to invest in our common stock, you should carefully read the prospectus supplement and the accompanying prospectus and any related free writing prospectus, including the section titled “Risk factors” contained in this prospectus supplement, as well as the information included in any free writing prospectus that we have authorized for use in connection with this offering. You should also carefully read the information incorporated by reference into this prospectus supplement and the accompany prospectus, including our consolidated financial statements, and the exhibits to the registration statement of which this prospectus supplement and the accompany prospectus are a part.
Company overview
We are a biopharmaceutical company focused on the identification, acquisition, development and commercialization of novel therapies for debilitating rare and orphan diseases. We focus on diseases for which the unmet medical need is high and the biology for treatment is clear.
Our lead product LIVMARLI (maralixibat) oral solution, or Livmarli, a novel, oral, minimally-absorbed agent designed to selectively inhibit apical sodium bile acid transporter, or ASBT, also known as the ileal bile acid transporter, or IBAT, is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome, or ALGS, one year of age and older in the United States. We market and commercialize Livmarli in the United States through our specialized and focused commercial team. We believe the initial addressable patient population in the United States is approximately 2,000 to 2,500 pediatric ALGS patients, which, based on our current expectations and beliefs, represents a greater than $500.0 million market opportunity. We have also filed for approval in Europe of Livmarli for the treatment of cholestatic liver disease in patients with ALGS and plan to commercialize Livmarli in Western Europe with our international team based in Switzerland. We have entered into license and distribution agreements with several rare disease companies for the commercialization of Livmarli in additional countries.
We are also developing Livmarli for progressive familial intrahepatic cholestasis, or PFIC, and biliary atresia, or BA. Livmarli has been granted breakthrough designation for ALGS and PFIC type 2. We expect to report topline data from our MARCH-PFIC Phase 3 clinical trial in the fourth quarter of 2022 and topline data from our EMABARK Phase 2b clinical trial in BA in the second half of 2023.
We are advancing our second product candidate, volixibat, a novel, oral, minimally-absorbed agent designed to inhibit IBAT, for the treatment of adult patients with cholestatic liver diseases. We are developing volixibat for the treatment of intrahepatic cholestasis of pregnancy, or ICP, primary sclerosing cholangitis, or PSC, and primary biliary cholangitis, or PBC. Volixibat has been studied in over 400 adults for up to 48 weeks. Clinical trials of volixibat have shown significant activity on ASBT and bile acid markers such as 7aC4, fecal bile acids and cholesterol, demonstrating potent biological activity. We expect to report interim data from our VISTAS Phase 2b clinical trial in PSC in mid-2023, interim data from our OHANA Phase 2b clinical trial in the first half of 2023 and interim data from our VANTAGE Phase 2b clinical trial in PBC in the second half of 2023.
Corporate information
We were incorporated in Delaware in May 2018. Our principal executive offices are located at 950 Tower Lane, Suite 1050, Foster City, California 94404, and our telephone number is (650) 667-4085. Our corporate website address is www.mirumpharma.com. Our website and the information contained on, or that can be accessed through, our website will not be deemed to be incorporated by reference in, and are not considered part of, this prospectus supplement or the accompanying prospectus. Our design logo, “Mirum,” and our other registered and common law trade names, trademarks and service marks are the property of Mirum Pharmaceuticals, Inc.