AJ201

AJ201 (rosolutamide) is an investigational, orally administered small molecule curcumin analog being developed by AnnJi Pharmaceutical for the treatment of Spinal and Bulbar Muscular Atrophy (SBMA), also known as Kennedy's disease. SBMA is a rare, X-linked inherited neuromuscular disorder characterized by the expansion of CAG repeats in the androgen receptor (AR) gene, which leads to the accumulation of toxic mutant AR protein aggregates. AJ201 functions by activating the Nrf2, Nrf1, and Hsf1 signaling pathways, which collectively enhance antioxidant responses, promote the proteasome-mediated degradation of mutant AR, and improve protein folding. Preclinical data have shown that AJ201 can reduce mutant AR toxicity and improve motor function. The drug is currently being evaluated in Phase 1/2a clinical trials to assess its safety, tolerability, and pharmacodynamics in patients with SBMA.

Other names

rosolutamide