Diagonal Therapeutics Inc.’s DIAG-723 has been awarded orphan drug designation by the FDA for the treatment of hereditary hemorrhagic telangiectasia (HHT). Additionally, the EMA has provided a positive opinion for orphan drug designation, confirming that DIAG-723 meets the criteria for designation as an orphan drug in the E.U.
Biomea Fusion Inc. has released preclinical findings from a 28-day weight loss study in obese nonhuman primates evaluating BMF-650, a next-generation oral small-molecule glucagon-like peptide-1 receptor agonist (GLP-1RA).
About 30%-40% of triple-negative breast cancers (TNBC) show HER2-low status and may benefit from the HER2-directed antibody-drug conjugate trastuzumab deruxtecan (T-DXd). Ataxia-telangiectasia mutated (ATM) kinase plays a crucial role in double-strand break (DSB) repair response, thus inhibitors of ATM, such as AZD-1390, may enhance the effect of DSB inducers, resulting in DNA damage accumulation.
Scientists at Jiangsu Hengrui Pharmaceuticals Co. Ltd. and Shanghai Hengrui Pharmaceutical Co. Ltd. have divulged glucagon-like peptide 1 receptor (GLP-1R) agonists reported to be useful for the treatment of type 1 diabetes, atherosclerosis, type 2 diabetes, obesity, nonalcoholic or metabolic dysfunction-associated steatohepatitis (NASH/MASH), hypertension, diabetic complications and coronary heart disease among others.
Shanghai Fosun Pharmaceutical (Group) Co. Ltd. has synthesized poly(ADP-ribose) glycohydrolase (PARG) inhibitors reported to be useful for the treatment of cancer.
Novartis AG has disclosed huntingtin (HTT; HD) (mutant) splicing modulators reported to be useful for the treatment of familial dysautonomia, Huntington’s disease and spinal muscular atrophy.
B-cell lymphoma represents about 85% of non-Hodgkin lymphoma cases; therapeutic approaches are represented by Bruton tyrosine kinase (BTK) inhibitors, but still some issues, such as drug resistance and off-target toxicity, limit the clinical benefits.
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