Proqr Therapeutics NV has highlighted the continued progression and expansion of its early-stage pipeline of RNA editing therapies, including programs in cholestatic diseases, Hurler syndrome, metabolic dysfunction-associated steatohepatitis and Rett syndrome.
Boehringer Ingelheim GmbH is stopping development of OSE Immunotherapeutics SA’s BI-770371 in metabolic dysfunction-associated steatohepatitis (MASH), after the SIRPα antagonist failed to show efficacy in a phase II study. Codevelopment of BI-770371 will continue for oncology indications, however, which was the initial target of the duo’s €1.4 billion (US$1.6 million) partnership in 2018.
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.
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.
