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AnnJi Pharmaceutical's Groundbreaking AJ201 Result at WMS 2025

On October 13, 2025, AnnJi Pharmaceutical Co., Ltd. made headlines at the World Muscle Society (WMS) 2025 International Congress held in Vienna, with the Phase 2a clinical results of their compound AJ201. This innovative treatment, aimed at tackling spinal and bulbar muscular atrophy (SBMA), also known as Kennedy's disease, was proudly presented as one of the seven Late-Breaking Abstracts of the event. The presentation was led by Dr. Tahseen Mozaffar, a key investigator in the study, who underscored the significant therapeutic potential AJ201 holds for patients suffering from this rare genetic disorder.

Understanding AJ201 and Its Mechanism

AJ201 is an oral suspension derived from Rosolutamide, a new curcumin analog designed to be easy to ingest for patients facing difficulties with swallowing—one of the common challenges associated with conditions like SBMA. The mechanism behind AJ201 is multifaceted: it activates Nrf2, modulates oxidative stress, and induces the production of heat shock proteins that assist in clearing harmful mutant protein aggregates. This makes AJ201 a pioneering candidate for treatment of PolyQ neurodegenerative disorders, marking a potentially significant advance in therapies for SBMA over the past two decades.

During the Phase 2a trials, researchers noted promising functional outcomes in patients treated with AJ201, paving the way for its recognition as a first-in-class therapy.

Dr. Andrew Pai also took the podium to share insights from the latest preclinical research on Rosolutamide (JM17), the active ingredient in AJ201. His findings indicated that JM17 could yield multiple benefits in SBMA mouse models, such as improved mitochondrial function, decreased inflammation, and enhanced structural integrity within muscle fibers. Furthermore, regulatory upstream analyses revealed JM17's ability to suppress pro-inflammatory agents, including TNF-α, IL-6, and TGF-β, while simultaneously activating protective signaling pathways. This reinforces JM17's potential as a versatile therapeutic agent offering new avenues for SBMA treatment.

AnnJi’s Commitment to SBMA Patients

Dr. Wendy Huang, the CEO and Chairperson of AnnJi, expressed immense pride in being selected for the Late Breaking Abstract at WMS 2025. She noted that the ongoing advancements with AJ201 signify a monumental step toward developing the world's first effective treatment for Kennedy's disease.

Notably, both the U.S. FDA and the European Medicines Agency (EMA) have granted Orphan Drug Designation to AJ201, further highlighting its potential. AnnJi has established a robust global patent portfolio, ensuring strong market exclusivity for its innovative treatment.

With a clear vision of progressing AJ201 into Phase 2/3 clinical development coupled with the pursuit of global licensing partnerships, AnnJi stands on the brink of delivering a new lease on life to patients suffering from SBMA worldwide. This commitment aligns with their mission to position AJ201 as a landmark success for Taiwan's emerging drug industry on the international stage.

About Kennedy's Disease (SBMA)

Kennedy's disease, or SBMA, is a rare inherited condition characterized by the degeneration of lower motor neurons within the spinal cord, brainstem, and muscles. Primarily affecting males aged 30 to 40, it shows a prevalence rate of about 1 in 40,000, with symptoms often worsening by the age of 50. As the disease progresses, those affected face increased difficulties with functions such as chewing and swallowing, which can lead to recurrent aspiration pneumonia and consequent demise, revealing the urgent need for effective treatments. Currently, no approved therapies exist for SBMA, highlighting the significance of Aj201's potential impact.

About AnnJi Pharmaceutical

AnnJi Pharmaceutical Co., Ltd. (TWSE 7754) is a biotechnology firm dedicated to the development of innovative small molecules aimed at addressing significant unmet medical needs, with a focus on neurology, dermatology, and immune-inflammatory disorders. The company aims to create transformative therapies that enhance the lives of patients suffering from chronic diseases that often go overlooked. Upon validating proof of concept through Phase 2 clinical trials, AnnJi seeks to license their products for further development and commercialization on a global scale, fostering sustainable growth for the company.