• AJ201 (rosolutamide) is an investigational oral small molecule designed to address multiple disease mechanisms of spinal and bulbar muscular atrophy (SBMA, or Kennedy's disease)
• Following submission of the Phase 3 protocol to the U.S. FDA under AnnJi's active Investigational New Drug (IND) application, AnnJi will proceed with U.S. site activation for the global Phase 3 ROMA-KD trial
• AJ201 has received Fast Track Designation from the U.S. FDA and Orphan Drug Designation in both the U.S. and the European Union

AnnJi Pharmaceutical Co., Ltd. (AnnJi, TPEx: 7754), a clinical-stage drug development company focused on innovative small molecules, today announced that it is proceeding with the U.S. portion of its pivotal Phase 3 ROMA-KD trial of AJ201 (rosolutamide) in patients with spinal and bulbar muscular atrophy (SBMA, or Kennedy's disease), a progressive, inherited neuromuscular disease with no FDA-approved treatment.

"For people living with SBMA, the disease is felt in everyday moments: a staircase that becomes harder to climb, a meal that becomes harder to swallow, a voice that becomes harder to use," said Dr. Wendy Huang, Chairperson and CEO of AnnJi. "It affects whole families, often across generations, and there is still no FDA-approved treatment. Advancing AJ201 into Phase 3 brings us one step closer to potentially changing that. We are now focused on activating U.S. trial sites, enrolling patients and completing this study as efficiently and responsibly as possible, with patients and families at our side."

AJ201 is an investigational oral small molecule that promotes clearance of the pathogenic mutant androgen receptor (mAR) protein and activates cellular stress-response pathways, including Nrf1, Nrf2 and HSF1. A potential first-in-class treatment for SBMA, AJ201 has received Orphan Drug Designation and Fast Track Designation from the U.S. FDA, as well as Orphan Drug Designation in the European Union.

The Phase 3 program builds on encouraging results from the completed Phase 2 study of AJ201, announced in May 2025, and was informed by subsequent interactions with regulatory authorities.

The ROMA-KD trial is a global, multicenter, randomized, double-blind, placebo-controlled study designed to evaluate the efficacy and safety of AJ201 in ambulatory individuals with symptomatic SBMA. Approximately 200 patients are expected to enroll globally, with the United States as a key region. The trial is intended to support potential global regulatory submissions for AJ201. AnnJi will coordinate site activation across regions in line with its global development and partnering strategy.

Patient and Care Partner Advisory Council

AnnJi has established its 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. The Council brings together people living with SBMA and their care partners, whose lived experience offers insights that clinical data alone cannot capture.

Serving in an advisory capacity, the Council provides a structured forum for ongoing, two-way dialogue between AnnJi and the SBMA community. Members share perspectives that may help inform AnnJi's patient-centered research and clinical development, including considerations for study design and the development of clear, accessible patient-facing materials. The Council operates independently of AnnJi's clinical trials, and participation has no bearing on trial eligibility or enrollment.

Commitment to the SBMA Community

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.