Pompe Therapy Shows Early Hope in Trial

PHILADELPHIA, PA— Aro Biotherapeutics shared preliminary results from a Phase 1b trial of ABX1100, an experimental treatment for late-onset Pompe disease, indicating the medication was mostly well-tolerated and maintained consistent target activity in participants.

The information was shared during an oral presentation at the 22nd annual WORLDSymposium held in San Diego, California. A poster from the session is available on the company's website.

ABX1100 was tested in 29 healthy individuals and nine patients suffering from late-onset Pompe disease. The treatment was delivered as a 20-minute infusion on Days 1 and 29, with a 20-week follow-up period. For the patient group, ABX1100 was used alongside enzyme replacement therapy, which is the existing standard of care. The participants had an average age of 54 when the study began and had been undergoing enzyme replacement therapy for between one and 15 years.

Muscle biopsies were taken at specific time points to evaluate the ongoing reduction of glycogen synthase 1 messenger RNA. Based on preliminary results from the first four patients, ABX1100 was found in muscle tissue after 10 weeks and showed continuous suppression of GYS1 mRNA in the quadriceps muscle from week 6 to week 10, reaching the desired level of inhibition. Company representatives stated that the data indicate the possibility of dosing every three months or less frequently.

"The continuous reduction of GYS1 mRNA in muscle, as shown in this phase 1b study involving patients and a previous phase 1 trial with healthy participants, offers evidence that targeting GYS1 could be a promising treatment for individuals with late-onset Pompe disease, and serves as a beacon of hope for the Pompe community," said Purnanand Sarma, Ph.D., chief executive officer of Aro Biotherapeutics.

ABX1100 was typically well accepted, with no instances of dose interruptions, study dropouts, or severe side effects observed during the research.

The trial also looked into exploratory biomarkers. Preliminary results from the first four patients indicated decreases in creatine kinase, a sign of muscle damage, by week 10. Three out of the four patients also saw drops in the Pompe disease biomarker Hex4, also known as glucose tetrasaccharide, at the same point. Further assessments of the patients are being conducted to gain a better understanding of how long the observed reduction in mRNA levels lasts.

Dr. Ozlem Goker-Alpan, president of the Lysosomal and Rare Disorders Research and Treatment Center in Fairfax, Virginia, who shared the information, stated that the results mark a preliminary move toward new methods of treatment.

"Our research is the initial to show the impact of a substrate reduction therapy in individuals with late-onset Pompe disease," Goker-Alpan stated, noting that the safety record, pharmacokinetics, and biomarker information indicate continued exploration of ABX1100 as a possible supplement or substitute for enzyme replacement therapy.

Additional details regarding the Phase 1b study can be found on ClinicalTrials.gov using the identifier NCT06109948.