FDA Grants Fast Track Designation for AJ201, a First-in-Class Therapy for Kenndy's Disease

TAIPEI, Oct. 23, 2025 /PRNewswire/ -- AnnJi Pharmaceutical Co., Ltd. (AnnJi, TWSE: 7754) today announced that the U.S. Food & Drug Administration (FDA) has granted Fast Track Designation for AJ201, the first-in-class for therapy for spinal and bulbar muscular atrophy (SBMA, also known as Kennedy's disease).

"Receiving the Fast Track Designation for AJ201 marks a significant milestone for AnnJi," said Dr. Wendy Huang, CEO and Chairperson of the Board of AnnJi. "Alongside the Orphan Drug Designations granted by both the U.S. FDA and EMA, this recognition reinforces AJ201's potential to address the unmet needs of patients with SBMA. Backed by a strong global patent portfolio, we remain committed to advancing AJ201 into Phase 3 development and to working closely with the FDA to potentially deliver the first approved treatment for SBMA in more than two decades."

About SBMA and AJ201

SBMA, or Kennedy's disease, is a rare X-linked inherited neuromuscular disorder caused by CAG repeat expansion in the androgen receptor (AR) gene. The resulting mutant AR protein contributes to muscle and neuron degeneration through mechanisms involving cellular toxicity, oxidative stress, and neuroinflammation. SBMA affects ~1 in 40,000 males globally and currently has no FDA-approved treatment.

AJ201, also known as JM17, is a novel investigational compound that has shown potential in reducing mutant AR toxicity and improving motor function in preclinical SBMA models. At the molecular level, it promotes degradation of pathogenic mAR protein, induces expression of antioxidant enzymes, proteasome subunits, and heat shock proteins, all of which may slow disease progression (Bott et al., 2016).

About AnnJi Pharmaceutical

AnnJi Pharmaceutical Co., Ltd. (TWSE: 7754) is a clinical-stage drug development company focused on the development of innovative small molecules (NCEs). The company is dedicated to addressing high unmet medical needs in the fields of neurology, dermatology, and immune-inflammatory diseases, including rare diseases, such as Kennedy's disease (SBMA) and Idiopathic Pulmonary Fibrosis (IPF).

AnnJi is committed to develop innovative therapies that improve the quality of life for patients suffering from neglected chronic diseases. We focus on advancing high-quality, differentiated, and innovative drug candidates, and collaborating with global pharmaceutical partners. Upon achieving proof-of-concept in phase II clinical trials, we aim to out-license our products for further co-development and commercialization, with the goal of bringing them to global markets and driving sustainable growth for the company.

Contact:

Anne Yu; anne.yu@ajpharm.com