AnnJi Pharmaceutical Co., Ltd. (AnnJi, TPEx: 7754) today announced the submission of an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) to initiate a pivotal Phase 3 clinical trial of AJ201 (rosolutamide) for spinal and bulbar muscular atrophy (SBMA, or Kennedy’s disease). The submission represents a major regulatory milestone in AnnJi’s planned global registration program for AJ201. Unless the FDA requests additional information or otherwise places the study on clinical hold, AnnJi may proceed with initiation of the U.S. portion of the planned global Phase 3 trial following the standard 30-day FDA review period. This milestone positions AJ201 as the only known therapeutic candidate for SBMA currently advancing toward Phase 3 development globally.

The submission builds on encouraging results from the completed Phase 1/2a study of AJ201 announced in May 2025 and subsequent regulatory interactions supporting the planned late-stage development program. AJ201 has received Orphan Drug Designation from the U.S. FDA and the European Medicines Agency (EMA) and Fast Track Designation from the FDA. The planned Phase 3 study is expected to enroll approximately 200 patients globally. The timing of study initiation and execution may be adjusted in coordination with AnnJi's global development and partnering strategy.

“The Phase 3 submission for AJ201 marks a major milestone toward delivering potentially the first disease-modifying treatment for people living with SBMA,” said Dr. Wendy Huang, Chairperson and CEO of AnnJi. “We will continue to advance international partnerships and global development, with the goal of bringing an effective therapy to patients with SBMA as quickly as possible and creating long-term value for our shareholders.”
 

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SBMA, or Kennedy's disease, is a rare X-linked inherited neuromuscular disorder caused by CAG repeat expansion in the androgen receptor (AR) gene. The resulting mutant AR protein contributes to muscle and neuron degeneration through mechanisms involving cellular toxicity, oxidative stress, and neuroinflammation. SBMA affects ~1 in 40,000 males globally and currently has no FDA-approved treatment. AJ201, also known as JM17, is an investigational oral small molecule designed to address multiple disease mechanisms implicated in SBMA. AJ201 promotes clearance of pathogenic mutant androgen receptor (mAR) protein and activates cellular pathways involved in antioxidant defense, protein quality control, and stress response, including Nrf2, Nrf1, and HSF1. Through these complementary mechanisms, AJ201 is being developed as a potential disease-modifying therapy for SBMA (Bott et al., 2016).

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