nido-361

NIDO-361 is a novel small molecule drug in clinical development for the treatment of Spinal and Bulbar Muscular Atrophy (SBMA), also known as Kennedy’s disease, a rare inherited X-linked neuromuscular disorder caused by mutations in the androgen receptor (AR) gene. NIDO-361 acts as an androgen receptor modulator, binding to a distinct site on the AR protein (the BF3 site) that differs from typical AR-targeting molecules. By binding this unique site, NIDO-361 regulates co-factor interactions and corrects transcriptional dysregulation associated with mutant AR, thereby aiming to restore healthy cell function and reverse muscle weakening characteristic of SBMA. The drug is administered orally and has received orphan drug designation for SBMA. It is currently being evaluated in Phase 2 clinical trials to assess its safety and efficacy in patients with SBMA[1][2][3][4][5][6][7].