2024 was another banner year for GLP-1 receptor agonists (GLP-1RAs) on multiple fronts. They continued to expand into new indications, and provide their developers with both rich remuneration and scientific acclamation. There are now seven approved GLP-1RAs. Commercially, the most successful one so far is semaglutide, sold under the brand name Wegovy or Ozempic depending on the indication.
The first acellular tissue engineered vessel for adults with vascular trauma in a bodily extremity has been approved by the U.S. FDA. Symvess, which comes from regenerative tissue developer Humacyte Inc., can be used to repair an arterial injury that could be life threatening and require immediate attention.
Ionis Pharmaceuticals Inc. CEO Brett Monia predicted that diagnoses of familial chylomicronemia syndrome (FCS) will “accelerate fairly quickly” now that the firm has gained U.S. FDA clearance for Tryngolza (olezarsen) as an adjunct to diet to reduce triglycerides in adults with the rare form of severe hypertriglyceridemia (sHTG) that can lead to life-threatening acute pancreatitis (AP).
Merck & Co. Inc. has turned to Asia for a second time to get into the GLP-1 market, this time to Shanghai-based Hansoh Pharmaceutical Group Co. Ltd. for its investigational preclinical oral small-molecule GLP-1 receptor agonist. Hansoh is getting $112 million up front and could bring in another $1.9 billion in milestone payments. Merck said the addition to its GLP-1 arsenal is “to provide additional cardiometabolic benefits beyond weight reduction.”
Wall Street must wait until 2025 for data from the higher-dose cohort in Tenaya Therapeutics Inc.’s phase Ib/II study with gene therapy TN-201 for MYBPC3-linked hypertrophic cardiomyopathy (HCM), but shares (NASDAQ:TNYA) closed Dec. 17 at $1.41, down $1.47, or 51%, after results from the first cohort rolled out.
After Keros Therapeutics Inc.’s voluntary halt of dosing in two arms of its phase II study in pulmonary arterial hypertension, the company’s stock crumpled after a year of muscular performance and its recent and massive deal with Takeda Pharmaceutical Co. Ltd.
China’s National Medical Products Administration (NMPA) granted new approvals to several Chinese biopharmaceuticals this week, including expanding indications of four different cancer drugs and clearing one sublingual tablet for stroke.
Novartis AG lost its bid, at least for now, to delay generic competition to its blockbuster heart drug, Entresto (sacubitril, valsartan), on the basis of patent infringement.
At the Breakthroughs in Muscular Dystrophy special meeting held in Chicago Nov. 19-20, 2024, and organized by the American Society of Gene & Cell Therapy (ASGCT), multiple interventions at the RNA level were among the approaches that were presented to fight muscular dystrophies.
Since the isolation of the gene that causes Duchenne muscular dystrophy (DMD), scientists have progressed in understanding the mechanisms that lead to muscular diseases that can be evident from the early stages of childhood. This has led to the development of diagnostics and therapeutics, some approved by the FDA.
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