First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221

Inclusion Criteria

• •Adults with Diagnosis of Pompe disease
• •Cohort 1: Enzyme Replacement Therapy (ERT)-experienced subject (ambulatory):
• •Male and female subjects between 18 and 65 years of age, inclusive
• •Received ERT with alglucosidase alfa (Myozyme/Lumizyme) for the previous 2-6 years, inclusive
• •Was receiving alglucosidase alfa at a frequency of once every other week
• •Must have been able to walk 200-500 meters on the 6-Minute Walk Test (6MWT)
• •Had upright Forced Vial Capacity (FVC) 30-80% of predicted normal value
• •Cohort 2: ERT-experienced subjects (non-ambulatory):
• •Male and female subjects between 18 and 65 years of age, inclusive
• •Had been receiving ERT with alglucosidase alfa for ≥2 years at a regular or set frequency
• •Was wheelchair-bound
• •Cohort 3: ERT-naïve subjects (ambulatory):
• •Male and female subjects between 18 and 65 years of age, inclusive
• •Must have been able to walk 200-500 meters on the 6MWT
• •Had upright FVC 30-80% of predicted normal value
• •Cohort 4: ERT-experienced subject (ambulatory):
• •Male and female subjects between 18 and 75 years of age, inclusive
• •Had been receiving ERT with alglucosidase alfa for ≥7 years, inclusive
• •Was receiving alglucosidase alfa at a frequency of once every other week
• •Must have been able to walk 75-600 meters on the 6MWT
• •Had upright FVC 30-85% of predicted normal value