PHILADELPHIA, PA — Aro Biotherapeutics reported interim data from a Phase 1b study of ABX1100, an investigational therapy for late-onset Pompe disease, showing the drug was generally well tolerated and achieved sustained target engagement in patients.
The data were presented during an oral session at the 22nd annual WORLDSymposium in San Diego, California. A session poster has been made available on the company’s website.
ABX1100 was evaluated in 29 normal healthy volunteers and nine patients with late-onset Pompe disease. The therapy was administered as a 20-minute infusion on Days 1 and 29, followed by 20 weeks of monitoring. In the patient cohort, ABX1100 was given as an add-on to enzyme replacement therapy, the current standard of care. The patients had a mean age of 54 years at study initiation and had been receiving enzyme replacement therapy for one to 15 years.
Muscle biopsies were collected at designated intervals to assess the persistence of glycogen synthase 1 messenger RNA knockdown. According to interim findings from the first four patients, ABX1100 was detected in muscle tissue at 10 weeks and demonstrated sustained knockdown of GYS1 mRNA in quadriceps muscle from Week 6 through Week 10, achieving the targeted level of suppression. Company officials said the data suggest the potential for quarterly or less frequent dosing.
“The sustained knockdown of GYS1 mRNA in muscle, as demonstrated in this phase 1b trial in patients, and in the earlier phase 1 trial in healthy volunteers, provides proof of principle that GYS1 inhibition may be a viable therapeutic approach in patients with late-onset Pompe disease, and a source for hope for the Pompe community,” said Purnanand Sarma, Ph.D., chief executive officer of Aro Biotherapeutics.
ABX1100 was generally well tolerated, with no dose interruptions, discontinuations, withdrawals, or serious adverse events reported in the study.
The trial also examined exploratory biomarkers. Interim data from the first four patients showed reductions in creatine kinase, a marker of muscle damage, by Week 10. Three of the four patients also experienced reductions in the Pompe disease biomarker Hex4, or glucose tetrasaccharide, by the same time point. Additional patient evaluations are ongoing to better understand the durability of the observed mRNA knockdown.
Ozlem Goker-Alpan, M.D., president of the Lysosomal and Rare Disorders Research and Treatment Center in Fairfax, Virginia, who presented the data, said the findings represent an early step toward alternative treatment approaches.
“Our study is the first to demonstrate the effect of a substrate reduction therapy in patients with late-onset Pompe disease,” Goker-Alpan said, adding that the safety profile, pharmacokinetics and biomarker data support further development of ABX1100 as a potential addition or alternative to enzyme replacement therapy.
More information about the Phase 1b trial is available at ClinicalTrials.gov under identifier NCT06109948.
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