AnnJi Pharmaceutical Advances AJ201 into Phase 3 Trial for SBMA

TAIPEI, Oct. 5, 2026 /CourierPR/ -- AnnJi Pharmaceutical Co., Ltd., a clinical-stage drug development company based in Taiwan (AnnJi, TPEx: 7754), has advanced its investigational therapy, AJ201 (rosolutamide), into the pivotal Phase 3 ROMA-KD trial for the treatment of spinal and bulbar muscular atrophy (SBMA), also known as Kennedy's disease. This marks a significant step in the development of a potential first-in-class treatment for this rare, X-linked inherited neuromuscular disorder.

AJ201, also known as JM17, is an oral small molecule designed to address multiple disease mechanisms of SBMA. It promotes the clearance of the pathogenic mutant androgen receptor (mAR) protein and activates cellular stress-response pathways, including Nrf1, Nrf2, and HSF1. According to AnnJi, these mechanisms make AJ201 a promising candidate for a disease-modifying therapy.

The Phase 3 ROMA-KD trial is a global, multicenter, randomized, double-blind, placebo-controlled study aimed at evaluating the efficacy and safety of AJ201 in ambulatory individuals with symptomatic SBMA. Approximately 200 patients are expected to enroll globally, with the United States serving as a key region. The trial is designed to support potential global regulatory submissions for AJ201.

AnnJi has received Fast Track Designation from the U.S. Food and Drug Administration (FDA) and Orphan Drug Designation in both the U.S. and the European Union for AJ201. The company has also established an SBMA Patient and Care Partner Advisory Council, first announced in collaboration with the Kennedy's Disease Association (KDA) at the 2026 KDA International Patient and Scientific Conference. This council brings together people living with SBMA and their care partners to provide insights that clinical data alone cannot capture.

SBMA, or Kennedy's disease, is a rare, X-linked inherited neuromuscular disorder caused by a CAG repeat expansion in the androgen receptor (AR) gene. The resulting mutant AR protein contributes to motor neuron and muscle degeneration through mechanisms involving cellular toxicity, oxidative stress, and neuroinflammation. SBMA is believed to be substantially underdiagnosed, in part because its symptoms are often mistaken for other neuromuscular conditions, such as amyotrophic lateral sclerosis (ALS), and because the absence of an approved treatment has offered little incentive for genetic testing.

The Phase 2 study of AJ201, announced in May 2025, showed encouraging results. The Phase 3 program was informed by subsequent interactions with regulatory authorities. The ROMA-KD trial is expected to support potential global regulatory submissions for AJ201.

AnnJi remains committed to advancing AJ201 for people living with SBMA and their families, and to working in transparent partnership with patients, care partners, clinicians, and advocacy organizations worldwide.