The bad news keeps piling up for Sage Therapeutics Inc. Having absorbed other study stumbles in the past few months, the company now has halted development of dalzanemdor in treating Huntington’s disease after top-line phase II data showed it missed a statistically significant difference compared to placebo on the primary endpoint.
Aeovian Pharmaceuticals Inc. has prepared tricyclic fused imidazole compounds acting as ADP-ribosyl cyclase/cyclic ADP-ribose hydrolase 1 (CD38) inhibitors reported to be useful for the treatment of neurodegeneration, inflammation, fibrosis, systemic scleroderma and more.
Ractigen Therapeutics Co. Ltd.’s RAG-21, a novel siRNA therapy targeting the FUS gene, has been awarded orphan drug designation by the FDA for the treatment of amyotrophic lateral sclerosis (ALS).