Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease

Inclusion criteria:

- Patient has been diagnosed with Pompe Disease prior to or during the screening period
based on 2 GAA gene mutations and either: endogenous GAA activity <75% of the lower
limit of the normal adult range reported by the testing laboratory, as assessed in
cultured skin fibroblasts -or- endogenous GAA activity <75% of the lower limit of the
normal adult range reported by the testing laboratory, as assessed by dried blood spot
or whole blood assay;

- Patient is male or female and 13 years of age or older at the time of enrollment in
the study;

- Sexually active patients must be willing to use an acceptable method of contraception
while participating in the study and for at least 4 months following the last dose of
BMN 701;

- If patient is female and not considered to be of childbearing potential, she is at
least 2 years post-menopausal or had tubal ligation at least 1 year prior to
screening, or who have had total hysterectomy;

- If patient is female and of childbearing potential, she has negative urine pregnancy
tests during the Screening Period and at the Baseline visit and be willing to have
additional pregnancy tests during the study;

- Patient has ≥30% predicted upright FVC and either <80% predicted upright FVC, or >10%
reduction in supine FVC compared to upright FVC during the Screening Period;

- Patient is naïve to Enzyme Replacement Therapy (ERT) with rhGAA;

- Patient must be able to ambulate at least 40 meters (131.2 feet) on the 6MWT conducted
at the Screening visit (use of assistive devices such as walker, cane, or crutches, is
permitted); and

- If subject was female, she was not lactating

Exclusion criteria:

- Patient has a history of diabetes or other disease known to cause hypoglycemia and is
currently receiving, or might anticipate receiving, hypoglycemic agents during the
course of the study;

- Patient has been on any immunosuppressive medication other than glucocorticosteroids
within 1 year prior to enrollment into this study;

- Patient requires invasive ventilatory assistance at the time of enrollment into the
study;

- Patient has received any investigational medication within 30 days prior to the first
dose of study drug or is scheduled to receive any investigational drug other than BMN
701 during the course of the study;

- Patient has previously been admitted to the study;

- Patient is breastfeeding at screening or planning to become pregnant (self or partner)
at any time during the study;

- Patient has a medical condition or extenuating circumstance that, in the opinion of
the Investigator, might compromise the patient's ability to comply with the protocol
requirements or compromise the patient's well being or safety;

- Patient has any condition that, in the view of the Investigator, places the patient at
high risk of poor treatment compliance or of not completing the study.