Mirum Pharmaceuticals Inc.

• Mirum Pharmaceuticals Inc.
• Health-Care

MIRM

We are a biopharmaceutical company focused on the development and commercialization of a late-stage pipeline of novel therapies for debilitating liver diseases. We focus on diseases for which the unmet medical need is high and the biology for treatment is clear. Our pipeline consists of two clinical-stage product candidates with mechanisms of action that have potential utility across a wide range of orphan liver diseases. We are initially developing maralixibat for the treatment of pediatric patients with progressive familial intrahepatic cholestasis, or PFIC, and Alagille syndrome, or ALGS.

Based on improvements in pruritus, or itching, and other disease markers observed in Phase 2 clinical trials, we commenced enrollment in the Phase 3 MARCH clinical trial in PFIC in the second quarter of 2019 and are planning to initiate a Phase 3 clinical trial in ALGS in the first half of 2020. We are developing volixibat for the treatment of adult patients with cholestatic liver diseases and expect to initiate Phase 2 clinical trials in 2020. In addition to data from ongoing clinical trials of maralixibat, we expect to complete enrollment in the MARCH clinical trial in the second quarter of 2020 and announce topline Phase 3 data in late-2020 and clinical data for volixibat in 2022.

Our initial focus is on rare cholestatic liver diseases, including PFIC, ALGS, biliary atresia, or BA, primary sclerosing cholangitis, or PSC, and intrahepatic cholestasis of pregnancy, or ICP. Cholestasis is characterized by impaired flow of bile resulting in accumulation of toxic bile acids in the liver. This leads to liver injury, progressive liver disease and, if left untreated, can result in fibrosis, cirrhosis, liver failure and death. Approximately half of PFIC patients will need a transplant by the age of ten, and ALGS patients can have significantly reduced life expectancy.

Patients with cholestatic liver disease typically experience debilitating pruritus, which dramatically impacts quality of life and can lead to scarring, sleep deprivation, significant fatigue and psycho-social effects. In some patients, intractable pruritus alone can justify liver transplantation. Other general manifestations of cholestatic liver diseases include jaundice, progressive liver disease and growth delay. Cholestatic liver diseases represent a significant unmet medical need, with no therapies approved by the U.S. Food and Drug Administration, or FDA, for treatment of the indications we are currently pursuing.

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