Patients Updates

Pompe Disease

Clinical Research Study:  VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Patients With Late-Onset Pompe Disease.  For more information on the study please see entry (

VAL-1221 for Pompe Disease

Valerion’s lead program, VAL-1221, is in clinical development for Pompe Disease. VAL-1221 is uniquely designed to
target delivery of therapeutic enzymes into skeletal and cardiac muscle, and unlike other therapies, VAL-1221’s unique
mechanism of action clears glycogen from both the lysosome and the cytoplasm.
• Pompe Disease, a rare multi-system genetic disorder with an approximate incidence of up to 1 in 9,000 births in
the U.S., is characterized primarily by skeletal muscle weakness causing problems with ambulation and respiratory
function. Patients with Pompe disease have a deficiency in a protein enzyme (acid alpha-glucosidase or GAA)
which is responsible for the degradation of a complex sugar called glycogen. This enzyme deficiency leads to
an accumulation of glycogen in skeletal, smooth and cardiac muscle tissue, causing damage to tissue structure
and function.


VAL-1221 Pompe Disease: Ongoing Phase 1/2 Study

Study Design:

12 ambulatory and ventilator-free patients
• 18 yrs previously treated with Myozyme or Lumizyme for at least 6 months

• 3 dosing cohorts: VAL-1221 at 3, 10, 30 mg/kg via IV every 2 weeks
• Control: Myozyme/ Lumizyme at usual dose

Study duration:
3 months with extension of VAL-1221 treatment for up to 1 year
• Myozyme/ Lumizyme patients can roll-over to VAL-1221 after 3 months




• Safety, tolerability and immunogenicity

• Pharmacokinetics (PK) and pharmacodynamics (PD)
• Six minute walk test
• Pulmonary function testing (MIP, MEP, FVC)
• Quantitative & qualitative muscle testing
• Patient-reported outcomes/quality of life/disability

Initial Clinical Results & Next Steps

Initial findings from first dosing cohort of Phase 1/2 study*
• VAL-1221 has been well-tolerated to date
– Safety profile similar to approved treatments
• No serious or unexpected adverse events or safety concerns observed
– After 3 months treatment with VAL-1221 at 3mg/kg
• No discontinuations from the study
• All patients from cohort 1 enrolled in the open-label extension phase

Next Steps:
• Dose escalation in cohort 2 complete
• Cohort 3 enrollment scheduled to complete in March
• Topline results expected Q3 2018
• Planned registration study Q4 2018

*Presented at 2018 WORLDSymposium February 2018