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AnnJi Pharmaceutical's Breakthrough in AJ201 for SBMA

AnnJi Pharmaceutical Co., Ltd. has recently made headlines at the World Muscle Society (WMS) 2025 international congress held in Vienna. The company presented the Phase 2a clinical trial results for its innovative drug AJ201, designed to combat Spinal and Bulbar Muscular Atrophy (SBMA), a rare and hereditary neuromuscular disorder. This significant event marks a crucial step for not only AnnJi but the millions of patients affected by this challenging condition.

SBMA, also known as Kennedy’s disease, affects primarily men in their 30s to 40s, leading to progressive degeneration of motor neurons and muscle wasting. As the disease advances, patients often face enormous challenges in daily activities, including difficulties in swallowing and an increased risk of recurrent aspiration pneumonia, which unfortunately can lead to a shortened lifespan.

The presentation by Dr. Tahseen Mozaffar, who is an investigator in the clinical study, shed light on the therapeutic potential of AJ201. The drug is an oral suspension of a new curcumin analogue, rosolutamide, specially designed for ease of use, particularly for patients who struggle with swallowing. Unlike typical treatments, AJ201 operates via multiple mechanisms including the activation of Nrf2, modulation of oxidative stress, and induction of heat shock proteins. These mechanisms work synergistically to help clear mutant protein aggregates, thereby positioning AJ201 as a pioneering small molecule therapy for neurodegenerative disorders like SBMA.

The Phase 2a trials yielded positive functional outcomes, highlighting the potential of AJ201 to be the first effective therapy for SBMA in two decades. Dr. Andrew Pai also presented the latest preclinical research on JM17, the active ingredient in AJ201. This compound demonstrated significant benefits in various pathways, improving mitochondrial function, reducing inflammation and fibrosis, and enhancing the structural organization of muscle fibers.

In addition, the data revealed an inhibition of pro-inflammatory mediators, alongside the activation of protective signaling pathways, underscoring JM17’s potential as a multifaceted therapeutic candidate for SBMA.

Dr. Wendy Huang, CEO and Chairperson of AnnJi, expressed her pride in the impactful findings shared at WMS 2025. “We are extremely proud of the late-breaking abstract and preclinical research article presented at WMS 2025, showcasing the significant advancements we've made in bringing AJ201 closer to being the world's first effective therapy for Kennedy's disease,” she stated.

AJ201 has received orphan drug designation from both the U.S. FDA and the European Medicines Agency (EMA), which adds to its credibility and potential market traction. Furthermore, AnnJi has developed a robust global patent portfolio, ensuring competitive advantages and market exclusivity for AJ201.

Looking ahead, AnnJi is committed to advancing the clinical development of AJ201 into Phase II/III trials and is actively pursuing global licensing partnerships. The ultimate goal is to provide a new ray of hope for SBMA patients worldwide, making AJ201 not just a milestone for AnnJi but also a historic achievement for the Taiwanese pharmaceutical industry on the global stage.

About AnnJi Pharmaceutical

AnnJi Pharmaceutical Co., Ltd. (TWSE 7754) is a clinical-stage drug development company focused on creating innovative small molecule therapies. The company aims to address unmet medical needs in neurology, dermatology, and immuno-inflammatory diseases, including rare conditions like SBMA and idiopathic pulmonary fibrosis (IPF). AnnJi is dedicated to developing advanced therapies that enhance the quality of life for patients suffering from chronic diseases. By emphasizing high-quality, differentiated drug candidates, AnnJi strives to collaborate with global pharmaceutical partners to introduce transformative therapies to the market.