Efficacy and Safety of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia

Key Inclusion Criteria:

1. Diagnosis of functional HoFH

2. If undergoing LDL apheresis, must have initiated LDL apheresis at least 3 months prior
to screening and must have been on a stable weekly or every other week schedule and/or
stable settings for at least 8 weeks

3. Willing to consistently maintain his/her usual low fat or heart-healthy diet for the
duration of the study

Key Exclusion Criteria:

1. LDL-C level <70 mg/dL (1.81 mmol/L) at the screening visit

2. Background medical Lipid Modifying Therapy (LMT) (if applicable) that has not been
stable before the screening visit

3. Lipid-apheresis schedule /apheresis settings (if applicable) that have not been stable
for at least 8 weeks before the screening visit

4. Use of nutraceuticals or over-the-counter therapies known to affect lipids, at a
dose/amount that has not been stable for at least 4 weeks prior to the screening visit

5. Presence of any clinically significant uncontrolled endocrine disease known to
influence serum lipids or lipoproteins

6. Newly diagnosed (within 3 months prior to randomization visit) diabetes mellitus or
poorly controlled (HbA1c >9%) diabetes

7. History of a MI, unstable angina leading to hospitalization, coronary artery bypass
graft surgery, percutaneous coronary intervention, uncontrolled cardiac arrhythmia,
carotid surgery or stenting, stroke, transient ischemic attack, valve replacement
surgery, carotid revascularization, endovascular procedure or surgical intervention
for peripheral vascular disease within 3 months prior to the screening visit

8. Pregnant or breastfeeding women

9. Sexually active women of child bearing potential (WOCBP), who are unwilling to
practice a highly effective birth control method prior to the initial dose, during the
study, and for 24 weeks after the last dose of study drug

10. Sexually active men who are unwilling to use forms of medically acceptable birth
control during the study drug treatment period and for 24 weeks after the last dose of
study drug

Note: Other protocol defined inclusion/exclusion criteria may apply.