#Taiwan #Taipei #AJ201 #SBMA #AnnJi

AnnJi Pharmaceutical's Groundbreaking AJ201 Presentation at WMS 2025

On October 13, 2025, AnnJi Pharmaceutical Co., Ltd., traded publicly under TWSE: 7754, captivated the audience at the World Muscle Society (WMS) 2025 Congress in Vienna with the promising results from the Phase 2a clinical trials of AJ201. This new treatment aims to address spinal bulbar muscular atrophy (SBMA), also known as Kennedy's disease, showcasing a significant advance in potential therapeutic options for patients afflicted by this neurodegenerative condition.

Dr. Tahseen Mozaffar, one of the lead investigators of this pivotal study, presented key findings that underscored the beneficial effects of AJ201. The drug, a curcumin analogue known as rosolutamide, is formulated as an oral suspension, making it accessible for patients who face challenges with swallowing. Its multimodal action mechanism includes Nrf2 activation, oxidative stress modulation, and the induction of heat shock proteins to help eliminate mutant protein aggregates. These features position AJ201 as a frontrunner in treating polyglutamine expansion disorders like SBMA, marking what could be the first breakthrough treatment in nearly two decades.

The Phase 2a trials reported positive functional outcomes for SBMA patients, signaling the remarkable potential of AJ201 as a new standard of care. Complementing the main findings, Dr. Andrew Pai also presented preclinical research on JM17, the active component of AJ201. In animal models of SBMA, JM17 demonstrated multiple benefits, notably enhancing mitochondrial function, alleviating inflammation and fibrosis, and improving the structural organization of muscle fibers. An analysis of upstream regulators indicated the inhibition of pro-inflammatory mediators, alongside the activation of protective signaling pathways. This evidence strengthens JM17’s profile as a multifunctional therapeutic candidate that may open new mechanical avenues for treating SBMA.

Wendy Huang, CEO of AnnJi Pharmaceutical, expressed immense pride in presenting these late-breaking results at the 2025 WMS Congress, highlighting the significant strides taken to develop AJ201 into the world’s first effective therapy for Kennedy's disease. Notably, AJ201 has received orphan drug designation from both the U.S. FDA and the European Medicines Agency (EMA), coupled with a robust global patent portfolio ensuring strong commercial exclusivity.

As part of its future plans, AnnJi aims to advance AJ201 through pivotal Phase 2/3 clinical trials and strengthen global licensing partnerships. The company hopes to offer new hope to patients worldwide while establishing AJ201 as a historic success for Taiwan's emerging pharmaceutical industry on an international scale.

Understanding Kennedy’s Disease (SBMA)

Kennedy's disease, or spinal bulbar muscular atrophy (SBMA), is a rare and hereditary neuromuscular degenerative disease characterized by the progressive degeneration of lower motor neurons in the spinal cord, brainstem, and skeletal muscles. It predominantly affects men in their 30s to 40s, with a prevalence rate estimated at 1 in 40,000. As the disease progresses, typically around the age of 50, many patients develop difficulties with chewing and swallowing, and recurrent aspiration pneumonia often becomes a leading cause of mortality. Currently, there are no approved treatments for SBMA, making the development of AJ201 even more significant.

About AnnJi Pharmaceutical

AnnJi Pharmaceutical Co., Ltd. (TWSE: 7754) is a clinical-stage drug development company focused on innovating small molecular compounds to address unmet medical needs in neurology, dermatology, and immune-inflammatory diseases, including rare disorders like SBMA and idiopathic pulmonary fibrosis (IPF). The company is committed to creating innovative treatments that improve the quality of life for patients suffering from neglected chronic diseases. AnnJi's strategic approach revolves around developing differentiated, high-quality drug candidates in collaboration with global pharmaceutical partners. Following principle validation in Phase II clinical trials, it seeks to license its products for co-development and further commercialization, aiming to distribute them globally and create sustainable growth for the company.