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Empowering the SBMA Community: Insights from the KDA 2026 Conference

Introduction
AnnJi Pharmaceutical Co., Ltd. recently participated in the International Patient and Scientific Conference hosted by the Kennedy’s Disease Association (KDA), held from February 27 to March 2, 2026. This pivotal event brought together patients affected by spinal and bulbar muscular atrophy (SBMA), caregivers, leading clinicians, and researchers to discuss scientific advancements and therapeutic developments related to this rare condition.

Deepening Connections
During the conference, Dr. Wendy Huang, the CEO and Chairperson of AnnJi, facilitated the "Patient & Family Forum." This initiative fostered meaningful dialogue between patients, their families, and healthcare providers, further strengthening relationships and enhancing confidence in the clinical advancements of AJ201, particularly its clinical trials.

Scientific Presentations
Building upon the recent Fast Track designation by the U.S. FDA for AJ201 (Rosolutamide) and promising Phase 2 clinical results, AnnJi delivered two significant scientific presentations. These presentations included translational transcriptomic results and updates on the eagerly anticipated Phase 3 trial.

1. Transcriptomic Evidence: One of the highlights was the research titled, “Transcriptomic Evidence of AJ201 Target Engagement and Biological Response in SBMA Muscle.” This study utilized RNA sequencing to demonstrate AJ201's engagement with the Nrf2 antioxidant pathway, confirming that the treatment influences multiple biological axes rather than a single pathway, showcasing AJ201's specific biological engagement.
2. Phase 3 Trial Design: AnnJi also presented a preliminary plan for their global multicenter Phase 3 clinical trial. This randomized, double-blind, placebo-controlled trial is designed to evaluate the efficacy and safety of AJ201 in ambulant patients suffering from symptomatic SBMA. This trial is crucial for potential global regulatory approval and aims to validate AJ201's therapeutic benefits for this underserved patient population.

Key Findings from Clinical Studies
Moreover, Dr. Christopher Grunseich, the principal investigator of the study, led a session entitled, “Safety and Preliminary Efficacy Demonstrated in a Phase 1/2a Clinical Trial of AJ201 in Spinal and Bulbar Muscular Atrophy.” His presentation highlighted AJ201's favorable safety profile and early efficacy signals observed during the Phase 2 study, offering updates on the entire clinical development program.

Commitment to the SBMA Community
Dr. Wendy Huang expressed, “We are honored to be alongside patients and advocates to translate scientific progress into tangible hope.” She emphasized the positive clinical results and FDA’s Fast Track designation, which strengthens their confidence in AJ201 and their commitment to advancing it to those in dire need. Despite recent clinical setbacks faced by other candidates in this field, AnnJi is determined to lead drug development for SBMA. They are ramping up preparations for the multinational Phase 3 trial, with participant recruitment expected to commence by the end of 2026.

Engaging Patients and Families
To reinforce this mission, AnnJi held the “Patient & Family Forum” at the KDA 2026 Conference. Recognizing that the lived expertise of patients and caregivers surpasses what scientific literature can reflect, this forum was a platform for open dialogue and transparent engagement. The event illuminated AJ201's mechanism and development while exploring partnership opportunities and announcing the creation of a patient advisory board in collaboration with the KDA. AnnJi aims to collaborate with SBMA patients and families to optimize the Phase 3 trial design, proactively tackle potential challenges, and accelerate the path towards delivering new hope to SBMA families worldwide.

About Kennedy's Disease (SBMA)
Kennedy's Disease, also known as Spinal Bulbar Muscular Atrophy (SBMA), is a rare hereditary degenerative neuromuscular disorder characterized by the progressive degeneration of lower motor neurons in the spinal cord, brainstem, and skeletal muscles. Primarily affecting men between the ages of 30 and 40, its prevalence is approximately 1 in 40,000. As the disease progresses, typically into the 50s, patients often face difficulties with chewing and swallowing, and recurrent aspiration pneumonia can become a frequent cause of death. Currently, there is no approved treatment for SBMA.

About AnnJi Pharmaceutical
AnnJi Pharmaceutical Co., Ltd. (TWSE: 7754) is a clinical-stage drug development company focused on developing innovative small molecules. The company aims to address significant unmet medical needs in neurology, dermatology, and immuno-inflammatory diseases, including rare disorders like Kennedy's Disease (SBMA) and idiopathic pulmonary fibrosis (IPF). AnnJi is committed to creating innovative treatments that enhance the quality of life for patients suffering from neglected chronic illnesses. They prioritize developing differentiated and innovative drug candidates in collaboration with global pharmaceutical partners, intending to sublicense their products for further co-development and commercialization, ultimately distributing them to global markets and ensuring sustainable growth for the company.