MIRM vs COGT Comparison

Mirum Pharmaceuticals Inc. is a biopharmaceutical company focused on developing and commercializing therapies for rare and orphan diseases. Its main product, Livmarli (maralixibat), is an orally administered IBAT inhibitor approved to treat cholestatic pruritus in patients with Alagille syndrome. The company is also developing treatments such as maralixibat for PFIC and ALGS, and volixibat for adult cholestatic liver diseases. Mirum currently has three approved medicines: Livmarli, Cholbam, and Ctexli.

Cogent Biosciences Inc a biotechnology company focused on developing precision therapies for genetically defined diseases. It designs rational precision therapies that treat the underlying cause of disease and improve the lives of patients. Its program CGT9486, is a selective tyrosine kinase inhibitor designed to potently inhibit the KIT D816V mutation as well as other mutations in KIT exon 17. In the vast majority of cases, KIT D816V is responsible for driving Systemic Mastocytosis (SM), a serious disease caused by the unchecked proliferation of mast cells. Exon 17 mutations are also found in patients with advanced gastrointestinal stromal tumors (GIST), a type of cancer with dependence on oncogenic KIT signaling.