AnnJi Reports Positive Phase I/IIa Trial Results for SBMA Therapy

Taiwanese company AnnJi Pharmaceutical has announced promising results from a Phase I/IIa randomized, placebo-controlled trial of AJ201, an experimental treatment for spinal and bulbar muscular atrophy (SBMA). Conducted at six US clinical sites, the double-blind study assessed AJ201’s safety, tolerability, pharmacodynamics, and pharmacokinetics.

Though not primarily designed to test efficacy, the trial showed significant improvements in muscle and physical function after 12 weeks. Treated patients improved by 17.6 meters in the 6-Minute Walk Test and gained 0.8 points on the SBMA Functional Rating Scale, while placebo participants declined slightly. AJ201 also reduced serum creatine kinase and myoglobin levels and improved quality of life scores.

Muscle biopsies revealed a 50% reduction in mutant androgen receptor levels in 53% of treated subjects. RNA sequencing indicated activation of the Nrf2 pathway, absent in placebo patients. SBMA is a rare X-linked neuromuscular disease.

26-05-2025