Mirum(US:MIRM)2022-05-06 16:35LIVMARLI for Alagille Syndrome (ALGS) - LIVMARLI is FDA-approved for cholestatic pruritus in ALGS patients [7], with a potential market opportunity exceeding $500 million in the US [7, 9] - A 6-year analysis demonstrated a 70% reduction in clinical outcomes (liver transplantation, biliary diversion surgery, hepatic decompensation, or death) with LIVMARLI compared to an untreated cohort [21] - Q1 2022 total product net revenue for LIVMARLI was $10.9 million, with approximately 75% of dispenses reimbursed [9] Pipeline and Development - The company has 5 late-stage indications under development [7, 12], including Progressive Familial Intrahepatic Cholestasis (PFIC), Biliary Atresia, Primary Sclerosing Cholangitis (PSC), Intrahepatic Cholestasis of Pregnancy (ICP), and Primary Biliary Cholangitis (PBC) [12] - MARCH Phase 3 data for PFIC is expected in Q4 2022 [12, 47, 80] - EMBARK study data for Biliary Atresia is expected in 2023 [12, 55, 80] - Interim data for VISTAS (PSC) and OHANA (ICP) are expected in Q4 2022, with VANTAGE (PBC) interim data expected in 2023 [12, 59, 80] Volixibat for Adult Cholestasis - Volixibat is being targeted for adult cholestasis, including ICP, PSC, and PBC [58, 59] - There are approximately 40,000 ICP cases per year in the US [59] - There are approximately 130,000 PBC cases per year in the US [59] Financial Status - The company reported $12.9 million net revenue in Q1 2022, with $10.9 million in net product revenue [78] - The company has a strong balance sheet with $239.9 million as of March 31, 2022 [78]