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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).

May 21, 2025

AnnJi Pharmaceutical Company Announces Positive Phase 1/2a Results for AJ201 in Spinal and Bulbar Muscular Atrophy (SBMA) Patients

AnnJi Pharmaceutical Co., Ltd., a clinical-stage Taiwanese biotechnology company focused on addressing unmet medical needs in dermatology, neurology, and rare diseases, today announced positive results from its Phase 1/2a randomized, double-blind, placebo-controlled, first-in-patient clinical trial of AJ201 in adults with Spinal and Bulbar Muscular Atrophy (SBMA).  Conducted across six U.S. clinical sites, the study evaluated the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of AJ201 (ClinicalTrials.gov Identifier: NCT05517603). While not powered to assess efficacy, exploratory endpoints revealed meaningful treatment-related improvements that support continued clinical development. 

Read the news release from AnnJi: Anji Biotech's New Drug AJ201 Has Achieved Impressive Clinical Phase 1b/2a Trial Results, Demonstrating Positive Therapeutic Efficacy and Potential.

Read the KDA News article: First-In-Patient Trial of a Drug to Treat Kennedy’s Disease 

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

Follow-Up News 

Read the KDA News article: FDA Grants Fast Track Designation for AJ201 

Read the KDA News article: AnnJi Showcases Promising Results for AJ201 in Kennedy’s Disease Clinical Trial

 

 

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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