Not hitting the primary endpoint of a phase III study caused the U.S. FDA to say it would not review Biohaven Ltd.’s NDA for troriluzole to treat spinocerebellar ataxia (SCA), an ultra-rare disorder.
Nanjing Zhihe Pharmaceutical Technology Co. Ltd. has described nucleotide derivatives acting as prodrugs reported to be useful for the treatment of viral infections.
Therabest Co. Ltd. has identified glutathione (GSH) or hydrogen peroxide (H2O2)-activated prodrugs comprising obeticholic acid dimers conjugated via linkers and their nanogels coated with fucoidan reported to be useful for the treatment of liver diseases.
Nicorandil is a potent K(ATP) channel activator and a sulfonylurea receptor 2 (SUR2) selective agonist launched for the treatment of angina pectoris. Nicorandil binds to ATP-sensitive KATP channels in the mitochondrial inner membrane to preserve mitochondrial function during ischemia.
A frustrating lack of detail left analysts with little to do but speculate on the odds for a 2023 U.S. FDA approval of Ascendis Pharma A/S’s Transcon PTH (palopegteriparatide), a parathyroid hormone prodrug for hypoparathyroidism, after the company disclosed a letter from the agency citing unspecified deficiencies that preclude further discussions about labeling and postmarketing requirements.
Protego Biopharma Inc. has identified prodrugs of transthyretin (TTR; PALB) stabilizers acting as TTR aggregation and/or amyloid formation inhibitors reported to be useful for the treatment of familial amyloid polyneuropathy and familial amyloid cardiomyopathy.
Researchers from Shasqi Inc. have reported the development of a new monomethyl auristatin E (MMAE)-based therapeutic, SQ-2270, within its proprietary CAPAC (Click Activated Protodrugs Against Cancer) platform for the treatment of cancer.