AnnJi Pharmaceutical Announces Breakthrough Results of AJ201 for Spinal and Bulbar Muscular Atrophy

AnnJi Pharmaceutical's Clinical Breakthrough on AJ201

On October 13, 2025, AnnJi Pharmaceutical Co., Ltd. (TWSE: 7754) announced a significant milestone at the World Muscle Society (WMS) 2025 Congress in Vienna. The company revealed the Phase 2a results of AJ201, a novel treatment for Spinal and Bulbar Muscular Atrophy (SBMA), also known as Kennedy's disease. These findings were recognized as one of the seven late-breaking abstracts during the international event.

Dr. Tahseen Mozaffar, one of the principal investigators of the study, presented the results, underscoring the therapeutic potential of AJ201 for those suffering from SBMA. AJ201 is an oral suspension based on a new curcumin analog—Rosolutamid—offering an accessible administration method for patients with swallowing difficulties. The drug operates through multiple signaling pathways, including the activation of Nrf2, managing oxidative stress, and inducing heat shock proteins to facilitate the degradation of mutated protein aggregates. This multimodal action positions AJ201 as a groundbreaking small-molecule therapy for neurodegenerative PolyQ diseases like SBMA.

In the Phase 2a trials, patients treated with AJ201 exhibited positive functional outcomes, highlighting its promise as the first effective treatment for SBMA in over 20 years. The clinical trials utilized a rigorous scientific approach, yielding results that indicate a strong efficacy signal.

Preclinical Research Enhancements

Adding to the momentum, Dr. Andrew Pai shared recent preclinical research findings on Rosolutamid (JM17), the active component in AJ201. The research demonstrated notable benefits in an SBMA mouse model, specifically enhanced mitochondrial function, reduced inflammation and fibrosis, and improved structural organization of muscle fibers. Furthermore, the analysis of upstream regulators confirmed the inhibition of pro-inflammatory mediators (TNF-α, IL-6, TGF-β) and the activation of protective signaling pathways. These promising results point toward JM17 as a multifunctional therapeutic candidate, contributing valuable mechanistic insights for treating SBMA.

Dr. Wendy Huang, CEO and Chairperson of AnnJi, expressed pride in their presentation and the significant progress made in developing AJ201 as a globally effective therapy for Kennedy’s disease. Notably, AJ201 has received Orphan Drug Designation status from both the American FDA and the EMA, alongside a strong global patent portfolio that ensures market exclusivity.

Looking ahead, AnnJi continues to advance the clinical development of AJ201 into Phase 2/3 trials, aiming to forge global licensing partnerships. Their goal is to provide hope to patients worldwide while establishing AJ201 as a monumental success for Taiwan's pharmaceutical industry.

Understanding Kennedy's Disease (SBMA)

Kennedy’s disease, or Spinal and Bulbar Muscular Atrophy (SBMA), is a rare genetic neuromuscular degenerative disorder characterized by the progressive degeneration of lower motor neurons in the spinal column, brain stem, and skeletal muscles. Primarily affecting males aged 30 to 40, the condition's prevalence is estimated at 1 in 40,000. As the disease progresses, especially around the age of 50, patients often experience severe challenges with chewing and swallowing, leading to recurrent aspiration pneumonia—a common cause of mortality.

Currently, there are no approved treatments available for SBMA, making the advances presented by AnnJi at WMS 2025 a beacon of hope for patients suffering from this debilitating condition.

About AnnJi Pharmaceutical

AnnJi Pharmaceutical Co. Ltd. (TWSE 7754) operates in the clinical phase of pharmaceutical development, focusing on innovative small molecules (NCEs). The company aims to address unmet medical needs in neurology, dermatology, and inflammatory immune diseases, including rare conditions like Kennedy’s disease and idiopathic pulmonary fibrosis (IPF). AnnJi is dedicated to developing innovative therapies to improve the quality of life for patients with neglected chronic illnesses. The company prioritizes high-quality, differentiated drug candidates and collaborates with pharmaceutical partners worldwide, aiming to license products for further development and marketing.