Manufacturing issues are the latest problem for Ultragenyx Pharmaceutical Inc. to solve after last week’s disappointment in a phase III study to treat brittle bones. The U.S. FDA gave the company a complete response letter (CRL) regarding the BLA for its gene therapy to treat Sanfilippo syndrome type A, saying it needs more details and improvements made about CMC after having finished manufacturing facility inspections.
Diabetic retinopathy (DR) is a leading cause of vision loss in patients with diabetes, with limited therapeutic options. While current treatments focus on the proliferative stage, there is an urgent need to understand the underlying mechanisms of the early stages of DR to halt progression. Growing research suggests that activated microglia are key drivers of inflammation in DR.
Single agonists of the glucagon-like peptide-1 receptor (GLP-1R) have been a success in the treatment of obesity, but monomeric dual or triple agonists have demonstrated improved efficacy on energy intake, appetite or metabolic function.
Oxyntomodulin (OXM) is a peptide hormone released by intestinal L cells after food intake. It acts as a dual agonist of glucagon-like peptide 1 (GLP-1) and glucagon receptors, regulating appetite, energy expenditure and glucose metabolism. However, its short plasma half-life limits its therapeutic potential.
Eli Lilly & Co. has divulged relaxin receptor 1 (RXFP1; LGR7) agonists reported to be useful for the treatment of diabetes, stroke, atherosclerosis, pulmonary hypertension, chronic obstructive pulmonary disease, hypercholesterolemia, chronic kidney disease and diabetes nephropathy, among others.
Adipose tissue is essential for energy balance, but its dysfunction due to excessive lipid accumulation contributes to metabolic disorders such as insulin resistance, dyslipidemia and type 2 diabetes. Thus, regulating adipogenesis represents a promising strategy for combating obesity-related complications.
Neurocrine Biosciences Inc. has divulged cyclic peptides acting as corticotropin-releasing factor 1 and 2 (CRF1 and CRF2) antagonists reported to be useful for the treatment of congenital adrenal hyperplasia, cancer, osteoporosis, myopathy, hyperglycemia, Cushing syndrome, peptic ulcers and hypertension, among others.
Rhythm Pharmaceuticals Inc. will be sitting down with regulators in the U.S. and Europe to discuss phase III plans in the wake of positive phase II data from the study testing bivamelagon (formerly LB5-4640), an oral melanocortin-4 receptor agonist, in patients with acquired hypothalamic obesity.
Guangdong Zhongsheng Ruichuang Pharmaceutical Co. Ltd. has described glucagon-like peptide 1 receptor (GLP-1R) agonists reported to be useful for the treatment of type 2 diabetes, obesity, atherosclerosis, nonalcoholic or metabolic dysfunction-associated steatohepatitis (NASH/MASH), hyperglycemia, cerebral infarction, hypertension and Parkinson’s disease.
Metabolic dysfunction-associated steatohepatitis (MASH) is a progressive liver disease that has limited available therapies. Shanghai Minwei Biotechnology Co. Ltd. has developed and presented data for MWN-105, a GLP-1/GIP/FGF21 triple agonist aimed at controlling metabolic dysfunction and fibrosis during MASH.
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