Taiwan-based biotech AnnJi Pharmaceutical (TPEx: 7754) is proceeding with the US portion of the pivotal Phase III ROMA-KD trial of rosolutamide (AJ201) in spinal and bulbar muscular atrophy (SBMA, also known as Kennedy's disease), following submission of the Phase III protocol to the US FDA under its active investigational new drug application.
The global, multicenter, randomized, double-blind, placebo-controlled study will enroll approximately 200 ambulatory individuals with symptomatic SBMA and is intended to support potential global regulatory submissions. Participant enrollment is expected to begin in late 2026.
The Phase III program builds on a completed Phase I/II study that was not powered for efficacy. Exploratory endpoints after 12 weeks showed a 17.6-meter gain in the 6-Minute Walk Test and a 0.8-point increase on the SBMA Functional Rating Scale versus placebo, while nuclear mutant androgen receptor levels were reduced by more than 50% in 53% of AJ201-treated patients compared with 17% of placebo recipients.
AJ201 is designed to promote clearance of pathogenic mutant androgen receptor protein and activate cellular stress-response pathways including Nrf1, Nrf2, and HSF1. AnnJi said oxidative stress is among the mechanisms implicated in SBMA, a rare, X-linked inherited neuromuscular disease with no US FDA-approved treatment.