After two decades of work with Translarna (ataluren) for nonsense mutation Duchenne muscular dystrophy, PTC Therapeutics Inc. withdrew its bid for approval of the compound upon hearing from the U.S. FDA. Regulators told Warren, N.J.-based PTC that the data package for Translarna is “unlikely to meet the agency's threshold of substantial evidence of effectiveness.”
A circuit formed by tumor, immune and nervous systems triggers cancer cachexia and anorexia, the excessive loss of weight, muscle and fat experienced in some cancer types. A new study is the first showing these three actors of a triangle interaction that initiates and feeds the process.
Viking Therapeutics Inc. said after-market hours Feb. 11 that it plans to advance its oral dual GLP-1/GIP receptor, VK-2735, into a phase III trial for obesity in the third quarter of 2026. VK-2735 is a novel dual glucagon-like peptide-1 and glucose-dependent insulinotropic polypeptide (GLP-1/GIP) receptor agonist.
A circuit formed by tumor, immune and nervous systems triggers cancer cachexia and anorexia, the excessive loss of weight, muscle and fat experienced in some cancer types. A new study is the first showing these three actors of a triangle interaction that initiates and feeds the process.
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.
Ascletis Pharma Inc. raised HK$843.53 million (US107.93 million) in a placement on the Hong Kong Stock Exchange to advance its lead program, ASC-30, a small-molecule glucagon-like peptide-1 (GLP-1) receptor agonist that can be dosed once monthly subcutaneously and once daily orally for treating obesity.
The malfunction of lymphatic drainage that occurs in lymphedema leads to excessive cholesterol accumulation in the affected skin and lymphatic vessels, causing inflammation and fibrosis. However, surgery and the chemical reduction of accumulated cholesterol with cyclodextrin reduce inflammation and regenerate lymphatic vessels. Cholesterol is a potential therapeutic target for treating lymphedema, according to this study published on Feb. 11, 2026, in Nature by Veronique Angeli and her colleagues.
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.
Novo Nordisk A/S’ television ad introducing the tablet form of its weight-loss drug, Wegovy (semaglutide), to American consumers didn’t pass regulatory muster.
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