Taiwanese company AnnJi Pharmaceutical has reported encouraging outcomes from its randomised, placebo-controlled Phase I/IIa trial of AJ201, an investigational compound for treating the adult population with spinal and bulbar muscular atrophy (SBMA).
The first-in-patient, double-blind trial was carried out across six clinical sites in the US, focusing on the compound’s tolerability, safety, pharmacodynamics (PD), and pharmacokinetics (PK).
Although not designed to evaluate efficacy, the study indicated significant treatment-related improvements that advocate for AJ201's further clinical development.
AJ201’s PK and safety profiles in SBMA patients aligned with previous data from healthy volunteers, showing good tolerability and no signs of systemic drug accumulation.
Following a 12-week oral treatment period, subjects receiving the therapy demonstrated clinically meaningful enhancements in physical and muscle function compared to those on placebo.
This included an average gain of 17.6m in the 6 Minute Walk Test (6MWT) and a 0.8-point increase observed in the SBMA Functional Rating Scale.
In contrast, slight declines in these areas are observed in the placebo group.
According to the company, the AJ201 group also exhibited a decrease in levels of serum creatine kinase and myoglobin, suggesting a positive therapeutic impact.
Furthermore, AJ201-treated subjects showed improvements in the physical function component of the SF36v2 quality-of-life questionnaire.
Muscle biopsies showed that nuclear mutant androgen receptor levels were decreased by more than 50% in 53% of therapy-treated subjects, versus 17% in the placebo group.
RNA sequencing from muscle biopsies of AJ201-treated subjects indicated the Nuclear factor erythroid 2-related factor 2 (Nrf2) pathway activation and modulation of several disease-relevant signalling cascades. These changes were not observed in the placebo group.
SBMA is an X-linked inherited neuromuscular disorder, also known as Kennedy’s disease.
AnnJi Pharmaceutical board chairman and CEO Wendy Huang said: “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.
“AnnJi is committed to advancing the programme into Phase III 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.”
