#Taipei #USA #AnnJi Pharmaceutical #AJ201 #Kennedy's Disease

FDA's Fast Track Designation for AJ201

On October 22, 2025, AnnJi Pharmaceutical Co., Ltd. announced that the U.S. Food and Drug Administration (FDA) has awarded Fast Track Designation to AJ201, marking it as a first-in-class therapy for spinal and bulbar muscular atrophy (SBMA), commonly known as Kennedy's disease. This designation represents a significant advancement, as it aims to expedite the development and review of drugs that have the potential to address serious conditions and fulfill unmet medical needs.

Dr. Wendy Huang, AnnJi's CEO and Chairperson, expressed her enthusiasm, stating that this recognition underscores the importance and promise of AJ201 for patients suffering from SBMA. Alongside having received Orphan Drug Designations from both the FDA and the European Medicines Agency (EMA), AnnJi remains resolute in its commitment to advance AJ201 into Phase 3 development. This progress could lead to one of the first approved treatments for SBMA in over twenty years.

Understanding SBMA and AJ201

SBMA is a rare inherited neuromuscular disorder that predominantly affects males. It is caused by an expansion of CAG repeats in the androgen receptor (AR) gene, leading to the production of a mutant AR protein. This alteration induces muscle and neuronal degeneration through various cellular mechanisms, including toxicity, oxidative stress, and neuroinflammation. Statistics indicate that SBMA affects approximately 1 in 40,000 males globally, with no FDA-approved treatments currently available.

AJ201, also referred to as JM17, is an investigational compound demonstrating promising results in preclinical models of SBMA. It works by reducing the toxicity associated with the mutant AR and improving motor function. Mechanistically, AJ201 promotes the degradation of the pathogenic AR protein and stimulates the expression of antioxidant enzymes, proteasome subunits, and heat shock proteins, all of which may contribute to slowing the pace of disease progression, as referenced in studies by Bott et al. (2016).

About AnnJi Pharmaceutical

Founded with the goal of innovating drug development, AnnJi Pharmaceutical Co., Ltd. (TWSE 7754) specializes in creating novel small molecules for various medical conditions, including neurology, dermatology, and immune-inflammatory diseases. The firm is particularly focused on addressing unmet medical needs in rare disorders such as Kennedy's disease (SBMA) and Idiopathic Pulmonary Fibrosis (IPF).

With a mission to enhance the quality of life for patients afflicted with chronic diseases, AnnJi is engaged in advancing drug candidates through collaborations with global pharmaceutical partners. The company aims for rigorous development phases, and upon achieving proof-of-concept from Phase II clinical trials, it plans to out-license its products for further collaboration and commercialization. Ultimately, AnnJi seeks to bring its therapies to an international market and promote sustainable growth.

The Fast Track Designation for AJ201 is not only a beacon of hope for those affected by Kennedy's disease but also a testament to the ongoing innovations in therapeutic development aimed at tackling rare and debilitating conditions. As AnnJi continues its journey towards a potentially transformative treatment, all eyes will be on the upcoming stages of this critical research.

For further information, contact Ann Ji at the company's investor relations department.