The Phase 1/2 trial is an open-label study designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamic and clinical activity of BMN 701.
In the study, BMN 701 will be administered as an intravenous infusion every two weeks at doses of 5mg/kg, 10mg/kg and 20mg/kg.
The company plans to enroll approximately 30 patients between 13 and 65 years of age, with late-onset Pompe disease for a treatment period of 24 weeks.