The farnesoid X receptor (FXR) is a nuclear receptor predominantly expressed in the liver, intestine and kidney. FXR is crucially involved in regulating bile acid homeostasis, controlling inflammatory responses in the liver, and regulating lipid and glucose metabolism. Therefore, FXR plays a role in regulating metabolic dysfunction-associated steatohepatitis (MASH) and has been proposed as a promising target for MASH drug development.
Continuing to build on the successful launch of Rezdiffra (resmetirom), Madrigal Pharmaceuticals Inc. is adding six preclinical-stage siRNA therapies to its metabolic dysfunction-associated steatohepatitis (MASH) pipeline in a deal with Ribo Life Science Co. Ltd. and its subsidiary, Ribocure Pharmaceuticals AB, that could be worth $4.4 billion if all milestones are achieved.
Madrigal Pharmaceuticals Inc. has signed an exclusive global license agreement with Suzhou Ribo Life Science Co. Ltd. and its subsidiary Ribocure Pharmaceuticals AB for six preclinical small interfering RNA (siRNA) programs.
Metabolic dysfunction-associated steatohepatitis (MASH) involves hepatic steatosis, inflammation, and fibrosis driven in part by hepatic stellate cell (HSC) activation. Dual inhibition of ACLY and ACSS2 restricts complementary hepatic sources of acetyl-CoA, a key driver of lipogenesis and MASH pathology.
As the march toward a new therapy continues in metabolic dysfunction-associated steatohepatitis (MASH), new approaches are drawing Wall Street’s attention. Among them is Inventiva SA’s pan-PPAR approach.
Tangram Therapeutics plc has submitted a clinical trial application (CTA) to the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a phase I/II trial of TGM-312 for metabolic dysfunction-associated steatohepatitis (MASH).
Mursla Bio Ltd. recently entered a partnership with an unnamed large pharmaceutical company to use its AI Precision Medicine platform to help with drug development and ultimately develop companion diagnostics. The collaboration uses Mursla’s platform ability to isolate and analyze extracellular vesicles from a simple blood sample, to provide biologically labelled, multiomics data to improve patient stratification, monitor treatment and develop companion diagnostics.
Opko Health Inc. has recently presented data for their GLP-1/glucagon receptor dual agonist OPK-88006, which is in preclinical development for the treatment of metabolic disease, including metabolic dysfunction-associated steatohepatitis (MASH) and obesity.
Samjin Pharmaceutical Co. Ltd. has disclosed inhibitors of 17-β-hydroxysteroid dehydrogenase 13 (HSD17B13; 17-β-HSD 13) and/or estradiol 17-β-dehydrogenase 1 (HSD17B1; 17β-HSD1) and/or HSD17B2 (17β-HSD2). As such, they are believed to be potentially useful for the treatment of nonalcoholic or metabolic dysfunction-associated steatohepatitis (NASH/MASH).
