Drug intelligence / Profile preview

rosolutamide

Development stage
Phase 2
Lead developer
AnnJi Pharmaceutical
Modality
Small Molecules
Administration
Oral
01

Overview

Rosolutamide (AJ-201) is an orally administered small-molecule curcumin analog developed by AnnJi Pharmaceutical for the treatment of spinal and bulbar muscular atrophy (SBMA), also known as Kennedy's disease. The drug acts as a multi-pathway modulator that activates the Nrf2, Nrf1, and Hsf1 pathways. This activation enhances antioxidant responses, induces heat shock proteins to facilitate protein folding, and promotes proteasome-mediated degradation of mutant androgen receptor (AR) aggregates. By clearing these toxic protein aggregates and reducing oxidative stress, rosolutamide aims to mitigate the neurodegeneration associated with SBMA, a condition for which there are currently no approved disease-modifying treatments. The drug has received Fast Track Designation from the U.S. FDA.

Other names
rosolutamide
02

Targets

NFE2L1 (Nuclear respiratory factor 1)NFE2L2 (Nuclear factor (erythroid-derived 2)-like 2)HSF1 (Heat shock factor protein 1)

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