PHILADELPHIA, PA — Aro Biotherapeutics announced positive topline results from the Phase 1b portion of a clinical trial evaluating its investigational therapy ABX1100 for late-onset Pompe disease, showing reductions in a key enzyme linked to glycogen production and early signs of functional improvement, the company said.
The study found that ABX1100 achieved sustained knockdown of glycogen synthase 1 messenger RNA, a target associated with glycogen buildup in muscle tissue, in all evaluable patients.
The Phase 1b portion included nine patients treated across four sites in the United States and Canada, with participants receiving two infusion doses on Days 1 and 29 as an add-on to enzyme replacement therapy, according to the company.
Patients were followed for 20 weeks, and muscle biopsies were collected at multiple intervals to assess the persistence of the treatment’s effect.
Aro said the therapy achieved approximately 62% reduction in the targeted mRNA in quadriceps muscle through Week 10, with data at Week 16 suggesting the potential for dosing intervals of three months or longer.
The study also measured lung function using forced vital capacity percent predicted, with seven patients showing an average improvement of about 2.5% at five months compared to baseline.
Patients with more advanced disease showed greater improvement, according to the company.
The trial further evaluated biomarkers including creatine kinase and urinary Hex4, which in most patients declined from baseline at Week 10, indicating potential reductions in muscle damage and glycogen accumulation.
ABX1100 was well tolerated, with no serious adverse events, infusion reactions, treatment discontinuations or clinically meaningful laboratory abnormalities reported, the company said.
“This study provides clinical proof of concept for the use of RNA-based substrate reduction therapy to treat late-onset Pompe disease,” said Dr. Ozlem Goker-Alpan, the study’s lead investigator.
Aro Biotherapeutics Chief Executive Officer Purnanand Sarma said the results will inform discussions with regulators about advancing the therapy into later-stage development, citing improvements in lung function as a key clinical measure.
The company said full results will be presented at a future scientific conference.
More information about the study is available at ClinicalTrials.gov under identifier NCT06109948.
Pompe disease is a rare genetic disorder caused by deficiency of an enzyme responsible for breaking down glycogen, leading to progressive muscle weakness and respiratory complications.
ABX1100 is an RNA-based therapy designed to reduce glycogen production in muscle tissue and has received orphan drug and rare pediatric disease designations from the U.S. Food and Drug Administration.
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