Aro Biotherapeutics has reported positive topline results from its phase 1b clinical trial of ABX1100, an investigational therapy for adults with late-onset Pompe disease (LOPD). ABX1100 is a substrate reduction therapy (SRT) designed to reduce glycogen production in muscle cells, addressing a key driver of disease progression.
ABX1100 uses Aro’s Centyrin platform to deliver small interfering RNA (siRNA) directly to muscle tissue via the CD71 receptor. The therapy targets glycogen synthase 1 (GYS1), the enzyme responsible for glycogen production. By lowering GYS1 activity, ABX1100 aims to reduce glycogen accumulation in muscle cells, which contributes to muscle weakness and respiratory impairment in Pompe disease.
In the phase 1b study, ABX1100 was administered as an add-on to standard enzyme replacement therapy (ERT) in a small group of patients with LOPD. The results showed successful target engagement, with meaningful reductions in biomarkers associated with glycogen production and muscle damage. These findings suggest that ABX1100 is effectively impacting the underlying disease biology.
Early signs of clinical benefit were also observed, including improvements in functional measures and indicators of muscle health. While these results are preliminary, they support the potential of ABX1100 to enhance treatment outcomes beyond what is achieved with ERT alone.
ABX1100 was generally well tolerated, with no serious safety concerns reported in the study. The favorable safety profile, combined with evidence of biological activity, supports continued clinical development.
These results build on earlier phase 1a data in healthy volunteers, which demonstrated durable GYS1 knockdown in muscle tissue following a single dose. Together, the findings reinforce the promise of substrate reduction therapy as a novel approach for treating Pompe disease.
Aro Biotherapeutics continues to advance ABX1100 as part of its broader pipeline of targeted genetic therapies designed to address the root causes of rare diseases.
Further studies with larger patient populations will be needed to confirm clinical benefit, evaluate long-term safety, and determine the therapy’s impact on disease progression.
Source
Clinical trial: clinicaltrials.gov/study/NCT06109948