FDA Grants Fast Track Designation to AnnJi’s AJ201 for Kennedy Disease

AnnJi Pharmaceutical Co., Ltd. (TWSE: 7754) announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to AJ201, a first-in-class investigational therapy for spinal bulbar muscular atrophy (SBMA), also known as Kennedy disease.

The Fast Track designation is intended to expedite the development and review of drugs that address serious conditions with unmet medical needs.

Company Statement

Dr. Wendy Huang, CEO and Chair of the Board of AnnJi, commented:

"Receiving Fast Track designation for AJ201 marks an important milestone for AnnJi. Along with orphan drug designations from the U.S. FDA and the European Medicines Agency (EMA), this recognition reinforces AJ201’s potential to address the unmet needs of patients with SBMA. We remain committed to advancing AJ201 into Phase 3 development and working closely with the FDA to potentially deliver the first approved treatment for SBMA in over twenty years."

About Spinal Bulbar Muscular Atrophy (SBMA)

SBMA is a rare, inherited, X-linked neuromuscular disorder caused by an expanded CAG repeat in the androgen receptor (AR) gene. The mutant AR protein contributes to muscle and neuron degeneration through mechanisms including cellular toxicity, oxidative stress, and neuroinflammation.

• Prevalence: ~1 in 40,000 men worldwide
• Current treatments: No FDA-approved therapies

About AJ201

AJ201, also known as JM17, is a novel investigational compound showing potential to:

• Reduce mutant AR toxicity
• Improve motor function in preclinical SBMA models
• Promote degradation of pathogenic AR protein
• Induce expression of antioxidant enzymes, proteasome subunits, and heat shock proteins, potentially slowing disease progression

About AnnJi Pharmaceutical

AnnJi Pharmaceutical Co., Ltd. (TWSE: 7754) is a clinical-stage drug development company focused on innovative small molecules for neurology, dermatology, and immuno-inflammatory diseases, including rare diseases such as SBMA and idiopathic pulmonary fibrosis (IPF).

The company’s strategy includes:

• Developing high-quality, differentiated drug candidates
• Collaborating with global pharmaceutical partners
• Licensing products for co-development and commercialization after proof-of-concept in Phase II trials
• Expanding access to global markets while generating sustainable growth

AnnJi is committed to improving quality of life for patients with neglected chronic diseases through innovative therapies.

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