PHILADELPHIA, PA — Aro Biotherapeutics announced it has completed enrollment in its Phase 1b clinical trial of ABX1100, an investigational therapy for late-onset Pompe disease (LOPD). The study is designed to evaluate safety, tolerability, pharmacokinetics, and pharmacodynamics of the therapy in patients currently receiving enzyme replacement therapy (ERT).
ABX1100 is a first-in-class Centyrin–siRNA conjugate targeting glycogen synthase 1 (GYS1), the enzyme responsible for glycogen production. Unlike standard ERT, which replaces the missing enzyme, ABX1100 aims to reduce glycogen accumulation at its source, potentially offering a new therapeutic pathway for patients.
“While rapid enrollment brings us closer to establishing proof of concept for GYS1 inhibition, it also shows the overwhelming physician and patient desire for new treatments,” said Purnanand Sarma, Ph.D., chief executive officer of Aro Biotherapeutics.
Pompe disease, a rare genetic disorder, results from a deficiency in the enzyme that breaks down glycogen. This leads to progressive muscle weakness and organ damage, often culminating in respiratory failure. Current therapies can be burdensome and only partially effective.
“ABX1100 is designed to potentially reduce the burden of Pompe disease and deliver meaningful improvements to patients and their families, using a completely novel treatment modality,” said Ozlem Goker-Alpan, M.D., president of the Lysosomal and Rare Disorders Research and Treatment Center and an investigator in the trial.
In the trial, patients received a single dose of ABX1100 on Day 1, followed by a booster on Day 29, with monitoring extending through 20 weeks. Results will help guide the design of later-stage studies and clarify ABX1100’s role alongside or independent of ERT.
Aro Biotherapeutics is advancing a pipeline of targeted siRNA medicines leveraging its proprietary Centyrin protein platform, which is engineered for efficiency, versatility, and safety.
Trial details are available at ClinicalTrials.gov under identifier NCT06109948.
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