AnnJi Pharmaceutical Co., Ltd. has launched a first-in-patient Phase 1/2a study of AJ201, a curcumin-based compound, to evaluate its safety, tolerability, and related biochemical and pharmacological activities in Kennedy’s Disease patients. The study will be conducted at six sites in the United States in 2023. AJ201 is designed to activate the Nrf1 and Nrf2 cellular pathways to promote degradation of the mutant androgen receptor (AR) protein that is responsible for Kennedy’s Disease. AJ201 has been granted orphan drug status by the FDA and EMA for the treatment of Kennedy’s Disease.

The six test sites are: Stanford University; University of California, Irvine (UCI); Mayo Clinic, Jacksonville (FL); Mayo Clinic, Rochester (MN); Washington University in St. Louis; and the National Institutes of Health near Washington, D.C. Enrollment has begun at UCI and is expected to commence at the other sites in late Spring 2023. For additional information, visit A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Of AJ201 In Patients – Full Text View – ClinicalTrials.gov.

AnnJi has entered a licensing arrangement with Avenue Therapeutics for the development and eventual commercialization of AJ201. For more information, visit Avenue Therapeutics.

 

You can search the NIH Clinical Trial Website for results of past Kennedy’s Disease trials. To go to their website, click here.

 

The site wcgCenterWatch is a website where you can search for clincal trials. To access the data base, click here.

 

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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).