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AnnJi Pharmaceutical Co., Ltd., clinical-stage drug development company focused on the development of innovative small molecules, announced that the US 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 US 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.”
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).
AnnJi Pharmaceutical Co., Ltd. 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).
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