AnnJi Pharmaceutical Company Announces Positive Phase 1/2a Results for AJ201 in Spinal and Bulbar Muscular Atrophy (SBMA) Patients
Taipei, May 21, 2025 – AnnJi Pharmaceutical Co., Ltd., a clinical-stage Taiwanese biotechnology company focused on addressing unmet medical needs in dermatology, neurology, and rare diseases, today announced positive results from its Phase 1/2a randomized, double-blind, placebo-controlled, first-in-patient clinical trial of AJ201 in adults with Spinal and Bulbar Muscular Atrophy (SBMA). Conducted across six U.S. clinical sites, the study evaluated the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of AJ201 (ClinicalTrials.gov Identifier: NCT05517603). While not powered to assess efficacy, exploratory endpoints revealed meaningful treatment-related improvements that support continued clinical development.
Read the Press Release for more information.
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. The distribution sites are listed below. 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. AnnJi has entered a licensing arrangement with Avenue Therapeutics for the development and eventual commercialization of AJ201. For more information, visit Avenue Therapeutics.
Avenue Therapeutics and AnnJi are excited to announce that Mike S. was the first KD patient to participate and be dosed in the clinical trial of AJ201 at the University of California, Irvine. Please see the press release from Avenue Therapeutics for more information. To read the release, click here.
There currently are six AnnJi drug trial distribution sites which are listed below with contact information.
Site 01 (University of California, Irvine)
Principal Investigator: Tahseen Mozaffar, MD
Contact: Pola Gaid, 714-456-6191, polagaid@hs.uci.edu
Site 02 (National Institutes of Health)
Principal Investigator: Christopher Grunseich, MD
Contact: Angela Kokkinis, 301-451-8146, akokkinis@cc.nih.gov
Site 03 (Stanford University)
Principal Investigator: John W. Day, MD, PhD
Contact: Emily Lien, 650-407-7912, emlien@stanford.edu
Site 04 (Mayo Clinic Rochester)
Principal Investigator: Eric J. Sorenson, MD
Contact: Jane Sultze, 507-538-5523, sultze.jane@mayo.edu
Site 05 (Mayo Clinic Jacksonville)
Principal Investigator: Bjorn E. Oskarsson, MD
Contact: Megan Donahue, 904-953-3647, donahue.megan@mayo.edu
Site 06 (University of Washington in St. Louis)
Principal Investigator: Alan Pestronk, MD
Contact: Oliver Doerr/ Jennifer M. Koniak, 314-362-1626, oliver.doerr@wustl.edu, koniak@wustl.edu
