Taiwan-based AnnJi Pharmaceutical Co Ltd, a clinical-stage biotechnology company, on Wednesday announced positive results from its Phase 1/2a trial of AJ201 in adults with Spinal and Bulbar Muscular Atrophy (SBMA).

AJ201, also known as JM17, is a novel investigational compound that has shown potential in reducing mutant androgen receptor toxicity and improving motor function in preclinical SBMA models.

The study evaluated the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of AJ201 across subjects at six clinical sites in the USA.

After 12 weeks of oral treatment, AJ201 recipients showed clinically meaningful improvements in physical and muscle function compared to placebo, including a 17.6-metre 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.