Mirum was founded upon a drug treatment (maralixibat) that RiverVest helped develop through Lumena – a Fund II company acquired by Shire in 2014.
After acquiring Lumena, Shire continued to run the randomized withdrawal study on maralixibat, the oral drug being evaluated in children with Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC).
Former Lumena CEO Mike Grey and CMO Niall O’Donnell – a RiverVest Managing Director – remained in touch with the families and physicians. By all accounts, the treatment was working!
But Shire – now part of Takeda – deprioritized the program in 2018 before launching a Phase III clinical study. Undeterred, the Lumena team bought back the asset and brought on Chris Peetz and his team to move maralixibat swiftly through FDA approval (based on additional analyses of the ALGS Phase II data) and a successful commercial launch.
Mirum went public in July 2019, and LIVMARLI was approved by the FDA in September 2021 for the treatment of cholestatic pruritus in patients with ALGS one year of age and older.
LIVMARLI, a minimally absorbed ileal bile acid transporter inhibitor, is the first and only FDA-approved medication for treating this rare liver disease that affects 2,000 to 2,500 children in the United States.
In conjunction with the approval, Mirum received a rare pediatric disease priority review voucher to apply toward speedier FDA review of one of its other products.
Mirum is advancing LIVMARLI and volixibat, also acquired from Shire, across five additional late-stage trials for high-need rare cholestatic liver diseases.
Read the latest Mirum news here.