Otsuka Pharmaceutical Co. Ltd. reaped positive results from an open-label phase II study of repinatrabit (JNT-517) in adolescents with phenylketonuria (PKU), a rare disease marked by the inability to break down the amino acid phenylalanine.
Drugs that mimic GLP-1 are widely used to treat diabetes and obesity, but it is not fully understood exactly how they produce many of their beneficial effects. Investigators from The Salk Institute for Biological Studies published a paper in Proceedings of the National Academy of Sciences shedding some light on the mechanisms. “Understanding this process more clearly could help researchers design the next generation of GLP-1-based treatments that are even more precise and effective,” first author Sam Van de Velde told BioWorld.
Work at Avioris Bio Inc. has led to the discovery of proprotein convertase subtilisin/kexin-type 9 (PCSK9) inhibitors reported to be useful for the treatment of hypercholesterolemia.
GM1 gangliosidosis is a lysosomal storage disease caused by mutations in the human GLB1 gene, encoding the ubiquitous lysosomal β-galactosidase. GM1 causes a rapidly progressing neurodegeneration, which can be lethal in the first 2 years of life in the most severe cases.
Researchers from Sab Biotherapeutics Inc. presented data generated in the development and validation of a pharmacokinetic (PK) assay for SAB-142 and the evaluation of its toxicology and immunologic effects in a juvenile cynomolgus monkey model.
China’s National Medical Products Administration (NMPA) has accepted for review Hightide Therapeutics Inc.’s NDA for HTD-1801 for type 2 diabetes, marking the Shenzhen-based company’s first NDA submission and a major step toward commercialization.
Terns Pharmaceuticals Inc. has synthesized glucagon-like peptide 1 receptor (GLP-1R) agonists reported to be useful for the treatment of diabetes, obesity, liver diseases and cardiometabolic syndrome.
Eli Lilly and Co. will invest $500 million to support South Korea’s biopharmaceutical industry over the next five years, following high-level talks March 9 between Prime Minister Kim Min-seok and Lilly Executive Vice President Patrik Jonsson.
Rapport Therapeutics Inc. added $20 million to its cash runway for its lead phase III oral seizure drug, RAP-219, through a potential $328 million license deal signed with Tenacia Biotechnology Co. Ltd.
Pfizer Inc. has identified 4-{4-[(1-{[4-(propan-2-yl)phenyl]carbamoyl)-D-prolyl)amino]cyclohexyl}benzoic acid derivatives acting as gastric inhibitory polypeptide receptor (GIPR) antagonists. As such, they are described as potentially useful for the treatment of type 2 diabetes and obesity.
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