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.
Safety and Pharmacokinetics
The safety and pharmacokinetic profiles in SBMA patients were consistent with prior healthy volunteer data. AJ201 was generally well tolerated, and no systemic drug accumulation was observed.
Positive Clinical Signals
After 12 weeks of oral treatment, AJ201 recipients showed clinically meaningful improvements in physical and muscle function compared to placebo, including a 17.6-meter gain in the 6-Minute Walk Test (6MWT) and a 0.8-point increase in the SBMA Functional Rating Scale (SBMAFRS) on average, while the placebo group experienced slight declines. AJ201 also led to reduction in serum creatine kinase and myoglobin levels—suggesting a positive therapeutic effect. Most responders are in the AJ201 group: 11 of 15 for 6MWT, 6 of 7 for SBMAFRS, 14 of 14 for creatine kinase, and 11 of 12 for myoglobin, further supporting AJ201’s benefit. Additionally, patients in AJ201 group reported significant improvement in physical function component of SF36v2 quality-of-life questionnaire, while placebo group a decline (p=0.026).
Biomarker Findings and RNA Sequencing Reveal Mechanistic Support
Mutant androgen receptor (mAR) levels, a proposed biomarker for SBMA, were assessed via muscle biopsies. Nuclear mAR levels were reduced by more than 50% in 53% of AJ201-treated patients, compared to 17% of placebo, indicating potential therapeutic activity.
RNA sequencing of muscle biopsies from AJ201-treated patients revealed activation of the Nrf2 pathway, along with modulation of several disease-relevant signaling cascades. These changes, absent in the placebo group, offer support for AJ201’s therapeutic mechanism of action.
Conclusion and Expert Commentary
Consistent improvements across functional, biochemical, and molecular markers observed in this study support continued development of AJ201 for SBMA.
“The study results are highly encouraging. AJ201 has shown evidence of clinical benefit, demonstrated through improvements in functional assessments, positive shifts in serum biomarkers, and RNA sequencing data supporting activation of the Nrf2 pathway. Together, these findings reinforce the therapeutic potential of AJ201,” said Dr. Christopher Grunseich, Principal Investigator of the study. Dr. Grunseich is a Lasker Clinical Research Scholar and Head of the Inherited Neuromuscular Diseases Unit at the National Institute of Neurological Disorders and Stroke (NINDS), National Institutes of Health (NIH).
“SBMA is a slowly progressing neuromuscular disorder, and I am greatly encouraged by the positive clinical outcomes observed after a relatively short course of AJ201 treatment,” said Wendy Huang, Ph.D., Chief Executive Officer and Chairperson of the Board at AnnJi. “AnnJi is committed to advancing the program into Phase 3 clinical trials, with the aim of delivering a safe, effective, and much-needed therapeutic option for patients living with SBMA—a disease that currently lacks any FDA-approved treatments.”
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