The I148M mutation in the PNPLA3 gene, which encodes patatin-like phospholipase domain-containing protein 3, is known to confer risk of fatty liver, cirrhosis and hepatic inflammation, which may lead to hepatocellular carcinoma or metabolic dysfunction-associated steatohepatitis (MASH).
Ibio Inc. has announced data from a non-GLP, nonhuman primate (NHP) pharmacokinetic (PK) study of IBIO-600, the company’s lead asset and a potentially best-in-class long-acting anti-myostatin antibody designed for subcutaneous administration. Results suggest the antibody could provide a significantly extended half-life in humans and a weight loss treatment option while preserving and promoting muscle growth.
Although the causes of polycystic ovary syndrome (PCOS) are unclear, researchers know this condition leads to endometrial dysfunction in women who have hormonal imbalances, and insulin resistance. Now, a study led by scientists at the Karolinska Institutet in Sweden has revealed the cellular and genetic differences that distinguish this disorder in the first atlas of the human endometrium during the proliferative phase of the menstrual cycle.
In type 2 diabetes patients, inadequate hyperglycemic control can lead to insulin-secreting β-cell exhaustion, dedifferentiation and eventual loss. Since adult β cells have limited proliferative capacity, this final stage cannot be reversed.
Fibroblast activation protein (FAP) is a serine protease, the expression of which increases with pathogenic fibroblasts in the fibrotic liver during metabolic dysfunction-associated steatohepatitis (MASH) and might induce fibrosis by cleaving several proteins that regulate extracellular matrix turnover and metabolism, including α2-antiplasmin (α2-AP) and fibroblast growth factor 21 (FGF21). Astrazeneca plc recently presented new results on their research regarding their oral small-molecule FAP inhibitor, AZD-2389, as a candidate drug for treating MASH.
As companies continually search for next-generation obesity prospects, one of the leaders in the disease space, Novo Nordisk A/S, has obtained an exclusive license to a preclinical-stage, first-in-class, small-molecule inhibitor of Acyl-CoA synthetase 5 (ACSL5) developed by Lexicon Pharmaceuticals Inc.
Maze Therapeutics Inc. has disclosed new sodium-dependent neutral amino acid transporter B(0)AT1 (SLC6A19) inhibitors reported to be useful for the treatment of phenylketonuria, metabolic syndrome, hyperphenylalaninemia, tyrosinemia, isovaleric acidemia, propionic acidemia, maple syrup urine disease and chronic kidney disease, among others.
Agios Pharmaceuticals Inc. has patented new 4-pyrazolo[1,5-a]pyridin-2-yl-4,5,6,7-tetrahydro-1h-imidazo[4,5-c]pyridine derivatives acting as phenylalanine hydroxylase (PAH) R408W mutant stabilizers reported to be useful for the treatment of phenylketonuria.
Palatin Technologies Inc. has obtained U.S. orphan drug designation for PL-7737 for leptin receptor (LEPR) deficiency, including obesity caused by this condition.
The liver is a key at maintaining glucose and lipid homeostasis, crucial processes for metabolic health. When these processes are disrupted, a series of metabolic disorders may occur, including type 2 diabetes and metabolic dysfunction-associated steatotic liver disease (MASLD).
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