AnnJi Pharmaceutical Co., Ltd.

AnnJi Pharmaceutical announced it is proceeding with the U.S. portion of its pivotal Phase 3 ROMA-KD trial of AJ201, also known as rosolutamide, in patients with spinal and bulbar muscular atrophy, or SBMA, also called Kennedy's disease. The company said it submitted the Phase 3 protocol to the U.S. FDA under its active Investigational New Drug application and will now activate U.S. sites for the global trial. The ROMA-KD study is a global, multicenter, randomized, double-blind, placebo-controlled trial expected to enroll approximately 200 ambulatory patients with symptomatic SBMA worldwide, with the United States as a key region, and is intended to support potential global regulatory submissions. AJ201, an investigational oral small molecule and a potential first-in-class treatment for SBMA, has received Fast Track Designation from the U.S. FDA and Orphan Drug Designation in both the United States and the European Union. AnnJi said the Phase 3 program builds on encouraging results from its completed Phase 2 study announced in May 2025, and the company also noted its SBMA Patient and Care Partner Advisory Council, first announced in collaboration with the Kennedy's Disease Association at the 2026 KDA International Patient and Scientific Conference.