AnnJi Advances AJ201 into Phase 3 for Rare Kennedy's Disease
Taipei – October 05, 2026 -- AnnJi Pharmaceutical Co., Ltd. (TPEx: 7754) is proceeding with the U.S. portion of its pivotal Phase 3 ROMA-KD trial for AJ201, an oral small molecule targeting spinal and bulbar muscular atrophy (SBMA), also known as Kennedy's disease, a rare inherited neuromuscular disorder with no FDA-approved treatment.
AJ201 becomes the only investigational SBMA therapy in Phase 3 development worldwide
AnnJi submitted the Phase 3 protocol to the U.S. FDA under its active Investigational New Drug application and will now move to activate U.S. trial sites. The global, multicenter, randomized, double-blind, placebo-controlled ROMA-KD study is designed to evaluate efficacy and safety in ambulatory patients with symptomatic SBMA, with approximately 200 patients expected to enroll worldwide and the United States serving as a key region.