Aro Biotherapeutics has announced encouraging early results from a Phase 1b clinical study of ABX1100, an investigational treatment being developed for adults with late-onset Pompe disease.
ABX1100 is designed as a substrate reduction therapy, meaning it works by lowering the production of glycogen in muscle cells rather than trying to remove it after it accumulates. The therapy uses a targeted delivery system to send small interfering RNA (siRNA) directly into muscle tissue, where it reduces the activity of glycogen synthase 1 (GYS1), a key enzyme involved in glycogen production.
In this early-stage study, ABX1100 was given alongside standard enzyme replacement therapy (ERT) to a small group of patients. The results showed clear biological activity, including reductions in markers linked to glycogen production and muscle damage. These findings indicate that the treatment is successfully acting on its intended target within the body.
Researchers also observed early indications of potential clinical benefit, such as improvements in measures related to lung function and overall muscle health. Although these findings are preliminary, they suggest that ABX1100 could offer added benefit beyond current standard treatment alone.
The therapy was generally well tolerated, with no serious safety concerns reported during the study. This favourable safety profile, combined with evidence of target engagement, supports further clinical development.
Further research in larger groups of patients will be needed to confirm these early results, better understand long-term effects, and determine whether ABX1100 can meaningfully slow disease progression.
Press release: https://www.businesswire.com/news/home/20260316177283/en/Aro-Biotherapeutics-Reports-Positive-Phase-1b-Topline-Results-for-ABX1100-a-Muscle-targeted-GYS1-siRNA-in-Patients-with-Late-Onset-Pompe-Disease-LOPD
Clinical trial: clinicaltrials.gov/study/NCT06109948