A study of neoGAA in naïve and alglucosidasealfa treated late-onset Pompe disease patients.

This is a phase I, multi-centre, multinational, open-label study of the drug neoGAA.

NeoGAA will be given in increasing doses and bi-weekly intravenous infusion.

• Group 1 - Late-onset Pompe disease patients naïve to treatment, 3 dose levels
• Group 2 - Late-onset Pompe disease patients previously treated with alglucosidasealfa, 3 dose levels

Objectives:

• the safety and tolerability of neoGAA
• the pharmacokinetic parameters of neoGAA
• the pharmacodynamic effects of neoGAA on skeletal muscle and other exploratory biomarkers
• the effect of neoGAA on exploratory efficacy endpoints