TAIPEI, March 4, 2026 /PRNewswire/ -- AnnJi Pharmaceutical Co., Ltd. (AnnJi, TWSE: 7754) announced its participation in the 2026 International Patient and Scientific Conference hosted by the Kennedy's Disease Association (KDA). Held between February 27 and March 2, the conference served as a premier forum bringing together patients with Spinal and Bulbar Muscular Atrophy (SBMA), caregivers, clinicians and leading researchers to discuss advances in disease science, therapeutic development, and clinical care.
Building on the recent U.S. FDA Fast Track Designation for AJ201(Rosolutamide) and encouraging Phase 2 clinical results, AnnJi presented two scientific abstracts spanning translational transcriptomic findings through highly anticipated Phase 3 trial. These presentations underscored AJ201's multi-modal mechanism of action and its potential as a first-in-class therapy within AnnJi's strategic roadmap paving the way for global Phase 3 trial.
Scientific Highlights and Abstract Presentations of AJ201 during the KDA Conference
- Transcriptomic Insights: Research titled "Transcriptomic Evidence of AJ201 Target Engagement and Biological Response in SBMA Muscle" utilized RNA-seq to demonstrate that AJ201 engages the Nrf2 antioxidant pathway. The data confirmed that treatment modulates multiple biological axes rather than a single pathway, reinforcing AJ201's drug-specific biological engagement.
- Phase 3 Trial Design: AnnJi unveiled the preliminary design of its global, multicenter Phase 3 trial. This proposed randomized, double-blind, placebo-controlled trial is designed to evaluate the efficacy and safety of AJ201 in ambulatory individuals with symptomatic SBMA. The trial represents a critical step toward potential global regulatory registration and aims to further validate AJ201's therapeutic benefit in this underserved patient population.
Additionally, Dr. Christopher Grunseich, MD, the study's Principal Investigator, led a featured session titled "Safety and Preliminary Efficacy Demonstrated in a Phase 1/2a Clinical Trial of AJ201 in Spinal and Bulbar Muscular Atrophy." During the presentation, he shared data highlighting AJ201's favorable safety profile, early signals of efficacy observed in the Phase 2 study, and key updates on the overall clinical development program.
Steadfast Commitment to the SBMA Community
"We are honored to stand alongside patients and advocates as we work to translate scientific progress into tangible hope," said Dr. Wendy Huang, CEO and Chairperson of AnnJi. "The positive clinical findings, together with the recent FDA Fast Track Designation, strengthen our confidence in AJ201's potential and affirm that we are advancing in the right direction."
Amid recent clinical setbacks of other candidates in the field, AnnJi continues to lead SBMA drug development. The company has intensified preparations to initiate its multinational Phase 3 trial, with participant enrollment anticipated to commence in late 2026. This momentum reflects AnnJi's unwavering commitment to advancing AJ201 until it reaches the patients who need it most.
To further this mission, AnnJi also hosted a "Patient & Family Forum" during the 2026 KDA Conference. Recognizing that patients and caregivers bring invaluable lived expertise beyond what scientific publications alone can capture, this forum provided a platform for open dialogue and transparent engagement. The event introduced the mechanism and development of AJ201, explored potential partnership opportunities, and announced the formation of a Patient Advisory Council in collaboration with KDA. AnnJi aims to partner with SBMA patients and families to help optimize the Phase 3 trial design, address potential challenges proactively, and accelerate the path toward delivering new hope to SBMA families worldwide.
About Kennedy's Disease (SBMA)
Kennedy's disease, also known as Spinal and Bulbar Muscular Atrophy (SBMA), is a rare and serious inherited neuromuscular degenerative disorder. It is characterized by the progressive degeneration of lower motor neurons in the spinal cord, brainstem, and skeletal muscles. The disease primarily affects males between the ages of 30 and 40, with an estimated prevalence of 1 in 40,000. As the disease progresses—typically by age 50—patients often develop difficulties with chewing and swallowing, and ultimately, recurrent aspiration pneumonia becomes a common cause of death. Currently, there are no approved treatments for SBMA.
About AnnJi Pharmaceutical
AnnJi Pharmaceutical Co., Ltd. (TWSE: 7754) is a clinical-stage drug development company focused on the development of innovative small molecules (NCEs). The company is dedicated to addressing high unmet medical needs in the fields of neurology, dermatology, and immune-inflammatory diseases, including rare diseases, such as Kennedy's disease (SBMA) and Idiopathic Pulmonary Fibrosis (IPF).
AnnJi is committed to developing innovative therapies that improve the quality of life for patients suffering from neglected chronic diseases. We focus on advancing high-quality, differentiated, and innovative drug candidates, and collaborating with global pharmaceutical partners. Upon achieving proof-of-concept in Phase 2 clinical trials, we aim to out-license our products for further co-development and commercialization, with the goal of bringing them to global markets and driving sustainable growth for the company.
Contact:
Anne Yu; anne.yu@ajpharm.com
Sylvia Lin; sylvia.lin@ajpharm.com
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Empowering the SBMA Community: AnnJi Highlights Patient & Family Forum and Shares AJ201 Scientific Advancement at the 2026 KDA Conference
