PROSPECTUS SUPPLEMENT SUMMARY
This summary highlights certain information about us, this offering and selected information contained elsewhere in or incorporated by reference into this prospectus supplement and the accompanying prospectus. This summary is not complete and does not contain all of the information that you should consider before deciding whether to invest in our common stock. For a more complete understanding of our company and this offering, we encourage you to read and consider carefully the more detailed information in this prospectus supplement and the accompanying prospectus, including the information incorporated by reference into this prospectus supplement and the accompanying prospectus, and the information included in any free writing prospectus that we may authorize for use in connection with this offering, including the information contained in and incorporated by reference under the heading “Risk Factors” beginning on page S-6 of this prospectus supplement, and under similar headings in the other documents that are filed after the date hereof and incorporated by reference into this prospectus supplement.
Unless the context requires otherwise, references in this prospectus supplement to “Mirum,” “the company,” “we,” “us,” “our” or similar terms refer to Mirum Pharmaceuticals, Inc. and, where appropriate, our subsidiaries.
Company Overview
We are a biopharmaceutical company dedicated to transforming the treatment of rare diseases affecting children and adults. We have three approved medications: LIVMARLI® (maralixibat) oral solution, or Livmarli, Cholbam® (cholic acid) capsules, or Cholbam, and Chenodal® (chenodiol) tablets, or Chenodal.
Livmarli is a novel, orally administered, minimally-absorbed ileal bile acid transporter, or IBAT, inhibitor, or IBATi, that is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome, or ALGS, three months of age and older in the United States and for the treatment of cholestatic pruritus in patients with ALGS two months of age and older in Europe. We believe the prevalent 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. ALGS is estimated to impact one out of every 30,000 births globally. We market and commercialize Livmarli in the United States and certain countries in Europe through our specialized and focused commercial team. We have also 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. We have submitted a Supplemental New Drug Application, or sNDA, to the U.S. Food and Drug Administration, or FDA, and a marketing authorization to the European Marketing Agency, or EMA, based on positive data from our Phase 3 study of Livmarli in patients with PFIC.
In July 2023, we entered into an Asset Purchase Agreement, or the Purchase Agreement, with Travere Therapeutics, Inc., a Delaware corporation, or Travere, pursuant to which we acquired Travere’s bile acid product portfolio, including Cholbam and Chenodal, two therapies addressing rare diseases in high-need settings. Cholbam is FDA-approved for the treatment of bile acid synthesis disorders due to single enzyme deficiencies and adjunctive treatment of peroxisomal disorders in patients who show signs or symptoms or liver disease. Chenodal has received medical necessity recognition by the FDA to treat patients with cerebrotendinous xanthomatosis, or CTX. We believe the prevalent patient population in the United States with bile acid synthesis disorder is approximately 200-300 and that the prevalent population with CTX exceeds 1,000.
Our late-stage pipeline includes three investigational treatments for debilitating liver diseases. The Livmarli development program includes the Phase 2b EMBARK clinical trial for BA. We expect to report topline data from the EMBARK trial in the second half of 2023. We are also advancing our product candidate volixibat, a novel, oral, minimally-absorbed agent designed to inhibit IBAT, for the treatment of adult patients with