AnnJi Pharmaceutical Co Ltd (TPEx:7754), a Taiwan-based, clinical-stage drug development company focused on innovative small molecules, announced on Monday that it is proceeding with the US arm of its pivotal Phase 3 ROMA-KD trial of AJ201 (rosolutamide) in patients with spinal and bulbar muscular atrophy (SBMA, or Kennedy's disease), a progressive, inherited neuromuscular disease with no FDA-approved treatment.

AJ201 is an investigational oral small molecule that promotes clearance of the pathogenic mutant androgen receptor (mAR) protein and activates cellular stress-response pathways, including Nrf1, Nrf2 and HSF1. A potential first-in-class treatment for SBMA, AJ201 has received Orphan Drug Designation and Fast Track Designation from the US FDA, as well as Orphan Drug Designation in the European Union.

According to AnnJi, the Phase 3 programme builds on encouraging results from the completed Phase 2 study of AJ201, and was informed by subsequent interactions with regulatory authorities.

ROMA-KD is a global, multicentre, randomised, double-blind, placebo-controlled study designed to evaluate the efficacy and safety of AJ201 in ambulatory individuals with symptomatic SBMA. Approximately 200 patients are expected to enrol globally, with the United States as a key region. The trial is intended to support potential global regulatory submissions for AJ201.