A recent study published in Acta Neuropathologica Communications sheds light on the gender disparity in the incidence of ACTH-secreting pituitary neuroendocrine tumors (PitNETs), which occur more frequently in females than in males.
Though it’s been used off-label for more than three decades to treat cerebrotendinous xanthomatosis, Mirum Pharmaceuticals Inc.’s chenodiol gained an official U.S. FDA nod Feb. 21 as the first drug approved specifically for treating the rare autosomal recessive lipid storage disease.
CSPC Zhongnuo Pharmaceutical (Shijiazhuang) Co. Ltd. has described glucagon-like peptide 1 receptor (GLP-1R) agonists reported to be useful for the treatment of diabetes, obesity, Alzheimer’s disease, overweight and nonalcoholic or metabolic dysfunction-associated steatohepatitis (NASH/MASH).
Jiangsu Hansoh Pharmaceutical Group Co. Ltd. and Shanghai Hansoh Biomedical Co. Ltd. have identified glucagon-like peptide 1 receptor (GLP-1R) agonists reported to be useful for the treatment of diabetes, obesity and nonalcoholic or metabolic dysfunction-associated steatohepatitis (NASH/MASH).
Researchers from MBX Biosciences Inc. presented preclinical data on MBX-4291, a prodrug developed as a long-acting GLP-1/GIP co-agonist through MBX’s proprietary Precision Endocrine Peptide (PEP) platform.
Research seems to be gaining ground in Wilson disease, a rare inherited condition that causes copper levels to accumulate in the liver, brain and eyes. Most people are diagnosed as children or around middle age, but the age spectrum can be wide. Monopar Therapeutics Inc. has drawn Wall Street’s attention in the space.
Septerna Inc. has announced its decision to discontinue a healthy volunteer phase I trial of SEP-786, an oral small-molecule agonist of the parathyroid hormone 1 receptor being developed for the treatment of hypoparathyroidism.
In a recent publication in Cell, researchers from the National University of Singapore and collaborators have proposed using commensal bacteria in the nasal cavity as a delivery vector for precision therapy targeting the OE and brain.
Dealmaking in the Asia Pacific (APAC) region took off this week, with the latest showcasing Genome & Co.’s licensing deal with Ellipses Pharma Ltd. for GENA-104, a phase I-ready immuno-oncology asset, under undisclosed terms Feb. 11.
Septerna Inc.’s stock plunged as much as 68% throughout the day Feb. 18 on news that the company was stopping a phase I trial of SEP-786 in healthy volunteers following two severe events of elevated unconjugated bilirubin in the highest dose cohort of the multiple ascending-dose portion of the study.
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