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Clinical Trials

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Researchers around the world are trying to find the treatment or cure for Kennedy's Disease (KD).

October 27, 2025

AnnJi Showcases Promising Results for AJ201 in Kennedy’s Disease Clinical Trial

Exciting news for the Kennedy’s Disease community! At the 2025 World Muscle Society International Congress in Vienna, AnnJi Pharmaceutical Co., Ltd. presented groundbreaking Phase 2a results for AJ201, an oral treatment candidate for Kennedy’s Disease, also known as spinal and bulbar muscular atrophy (SBMA).

The study, presented by Dr. Tahseen Mozaffar, leader of a participating trial site, showed positive functional outcomes and strong therapeutic potential. The presentation given by Dr. Mozaffar was chosen as one of only seven Late-Breaking Abstracts, a special recognition reserved for research with important new findings.

AJ201 offers hope as the first potential disease-modifying therapy for Kennedy’s Disease in over two decades.

Read the news release on AnnJi's website: Anji Biotech's AJ201 Clinical Data is Impressive and Was Selected as the Latest Breakthrough Research at the "World Muscle Society 2025 International Annual Conference."

Read the press release on PR Newswire: AnnJi Showcases Clinical Breakthrough of AJ201 at WMS 2025

Learn more about AJ201 and  AnnJi Pharmaceutical Co., Ltd.


 


 

Resources

  • Clinical trials are a key step in the evolution of disease research from the lab to an approved drug or therapy. The Muscular Dystrophy Association (MDA) has a very useful guide to the clinical trial process, including definitions, types or phases of clinical trials, and frequently asked questions. MDA has graciously approved a direct link to their clinical trials page, which you can access by following this link.

  • Search the NIH National Library of Medicine, National Center for Biotechnology Information, for results of past Kennedy's Disease clinical trials. Visit their website.

  • CenterWatch is a website where you can search for clinical trials. Visit their website to access their database.

Research Updates

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