Leigh syndrome is a rare neurometabolic disorder that causes the CNS to degenerate. Investigators from the University of California, Davis, and Myto Therapeutics Inc. noted that the NDUFS4-knockout (KO) mouse is a well-characterized model for this disease and shared results on the effects of monomethyl fumarate prodrug MYT-109 in this murine model.
Genescience Pharmaceuticals Co. Ltd. has divulged indoline-containing spiro derivatives acting as growth hormone secretagogue receptor (GHSR) agonists reported to be useful for the treatment of growth hormone deficiency.
The liver X receptor (LXR) is a nuclear hormone receptor that is one of the most important regulators of cholesterol homeostasis and plays a key role in triglyceride (TG) regulation. Researchers from Orsobio Inc. and Phenex Pharmaceuticals AG have presented preclinical data on TLC-2716, a potent, small-molecule LXR inverse agonist for the treatment of lipid disorders.
Scientists from Orsobio Inc. and affiliated organizations have described preclinical data for the novel liver-targeted mitochondrial protonophore TLC-6740, being developed for the treatment of metabolic disease. In vitro, mild mitochondrial uncoupling caused by TLC-6740 had pleotropic metabolic benefits in multiple cell lines. TLC-6740 increased mitochondrial potential, oxygen consumption rate and tricarboxylic acid (TCA) cycle flux, and it also inhibited de novo lipogenesis with EC50 values of 6.9 µM.
Gilead Sciences Inc. has synthesized carboxy-benzimidazoles acting as glucagon-like peptide 1 receptor (GLP-1R) agonists reported to be useful for the treatment of arthritis, diabetes, eating disorders, macular degeneration, myocardial infarction, nonalcoholic steatohepatitis, sleep apnea and Parkinson's disease, among others.
Neurons are specialized cells with a high metabolic demand to fulfill their function, survive or keep a healthy half-life. In this sense, the anabolism and catabolism of proteins and lipids could be associated to different neurodegenerative diseases. At the 2022 annual meeting of the Society for Neuroscience, scientists reported the latest discoveries on neuron metabolic needs at a session on 'Powering Thoughts: The Regulation of Neuronal Energy Metabolism and Mitochondria.'
Ardelyx Inc. could have a rocky row to hoe when it makes its case for tenapanor, as a hyperphosphatemia therapy in adults with chronic kidney disease, before the U.S. FDA’s Cardiovascular and Renal Drugs Advisory Committee Nov. 16. The big question facing the adcom is whether the change in baseline serum phosphorous levels achieved by the drug is clinically meaningful. Clearly, FDA reviewers don’t think so, as that question already has resulted in delayed PDUFA dates, a complete response letter and two formal dispute resolution requests.