PHILADELPHIA, PA — Aro Biotherapeutics said it will present interim clinical data next month from its ongoing Phase 1b trial of ABX1100, an experimental therapy aimed at treating late-onset Pompe disease, offering what the company describes as the first clinical evidence for a new approach targeting glycogen production in muscle.
The data will be unveiled in an oral presentation at the 22nd annual WORLDSymposium in San Diego, a leading international meeting focused on lysosomal diseases. The presentation will include early analyses of safety, tolerability, biomarker changes, and pharmacodynamic activity in patients with late-onset Pompe disease who are already receiving enzyme replacement therapy.
Ozlem Goker-Alpan, M.D., president of the Lysosomal and Rare Disorders Research and Treatment Center in Fairfax, Virginia, will deliver the presentation, scheduled for Thursday, February 5, 2026, at 1:30 p.m. PST.
According to Aro, the interim findings represent the first clinical data supporting a GYS1-targeting substrate reduction therapy, a strategy designed to limit glycogen buildup rather than replace missing enzymes. Excess glycogen accumulation in muscle tissue is a defining feature of Pompe disease and the primary driver of progressive muscle weakness and respiratory decline.
ABX1100 is built around a CD71 receptor-binding Centyrin conjugated to a short-interfering RNA that interferes with expression of GYS1 messenger RNA, reducing activity of the enzyme responsible for glycogen production in muscle. The investigational therapy has received Orphan Drug Designation and Rare Pediatric Disease status from the U.S. Food and Drug Administration.
Pompe disease is a rare inherited neuromuscular disorder caused by a deficiency of acid alpha-glucosidase, leading to toxic glycogen buildup in muscle cells. Late-onset Pompe disease can emerge anytime after infancy and is typically treated with enzyme replacement therapy, which requires lengthy intravenous infusions multiple times each month. Despite its use, many patients continue to experience disease progression, highlighting a significant unmet medical need.
Additional details on the Phase 1b clinical trial are available through ClinicalTrials.gov under identifier NCT06109948.
Aro Biotherapeutics said the forthcoming data will help clarify whether ABX1100 could complement existing treatments and potentially reduce the long-term burden of care for patients living with late-onset Pompe disease.
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