Aro Biotherapeutics Completes Enrollment in Phase 1b Trial of Targeted siRNA Therapy, ABX1100, in Late-Onset Pompe Disease (LOPD)

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8:30 AM on Wednesday, September 24

The Associated Press

PHILADELPHIA--(BUSINESS WIRE)--Sep 24, 2025--

Aro Biotherapeutics, a clinical-stage biotechnology company developing potent and tissue-targeted short-interfering RNA (siRNA) medicines, today announced that it has completed enrollment in its ongoing Phase 1b clinical trial of ABX1100, a novel investigational therapy for the treatment of late-onset Pompe disease (LOPD). The trial is evaluating safety, tolerability, pharmacokinetics, and pharmacodynamics of ABX1100 in patients with LOPD who are currently receiving enzyme replacement therapy (ERT).

“While rapid enrollment in the Phase 1b ABX1100 clinical trial brings us closer to establishing proof of concept for GYS1 inhibition as a therapeutic approach, it also shows the overwhelming physician and patient desire for new treatments driven by the significant unmet clinical need,” said Purnanand Sarma, Ph.D., chief executive officer of Aro Biotherapeutics.

“Pompe disease is a highly debilitating disease that significantly impairs the patient's strength and independence, as available therapies are quite onerous,” added Ozlem Goker-Alpan, M.D., president of Lysosomal and Rare Disorders Research and Treatment Center, and an investigator on the study. “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.”

People living with LOPD have a genetic deficiency in the enzyme that breaks down glycogen, a stored form of sugar used for energy. As a result, glycogen accumulates in muscle tissue, causing cellular damage and organ dysfunction that present as muscle impairment, weakness, and disability that can eventually progress to death from respiratory failure. 1

Aro is developing a pipeline of tissue-targeted siRNA medicines using a proprietary protein technology called Centyrins, which are small proteins engineered for exceptional efficiency, versatility, and safety. ABX1100 is a first-in-class Centyrin–siRNA conjugate that inhibits expression of glycogen synthase 1 (GYS1), the enzyme responsible for glycogen production. This targeted approach is distinctly different from currently used ERT.

When combined with ERT, and as a monotherapy, ABX1100 has shown significant activity in preclinical models. The Phase 1b trial of ABX1100 aims to validate those findings in patients with LOPD, and to evaluate the potential of ABX1100 to improve patient outcomes.

All patients enrolled in the trial received one dose of ABX1100 on Day 1 and a booster dose on Day 29, and will be monitored through 20 weeks to assess safety and preliminary therapeutic activity. Analysis of the results will inform the design of later-phase clinical studies of ABX1100.

More information about the Phase 1b trial is available at ClinicalTrials.gov using the identifier NCT06109948.

About ABX1100

ABX1100, an investigational treatment for Pompe disease, is comprised of a CD71 receptor-binding Centyrin conjugated to a short-interfering RNA (siRNA) that specifically interferes with expression of GYS1 messenger RNA (mRNA), thereby reducing levels and overall activity of the GYS1 enzyme in muscle tissues. ABX1100 has received Orphan Drug Designation and Rare Pediatric Disease status from the United States Food and Drug Administration.

About Pompe disease

Pompe disease is a rare neuromuscular disorder caused by a genetic deficiency of acid alpha-glucosidase, which leads to a toxic buildup of glycogen in the muscle. This buildup leads to progressive loss of muscle function, weakness, and disability that can eventually progress to death from respiratory failure. 1 Late-onset Pompe disease (LOPD) is a subtype of Pompe that has onset after the first year of life. The current standard of care for LOPD is enzyme replacement therapy (ERT), which aims to restore genetically deficient enzymes. Despite the availability of ERT, significant unmet need remains due to limited ERT efficacy and burden of care. ERT requires intravenous infusions that can last as long as 6 hours, multiple times a month.

About Aro Biotherapeutics

Aro Biotherapeutics is a biotechnology company working to develop potent and versatile tissue-targeted genetic medicines with a platform based on a proprietary protein technology called Centyrins. The company is developing a wholly owned pipeline of Centyrin-based therapeutic candidates for tissue-specific targeting of therapeutics for a diverse set of diseases. For more information, visit https://www.arobiotx.com/.

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