Study in Participants With Homozygous Familial Hypercholesterolemia (HoFH)

Note: The information listed below is not intended to contain all considerations relevant
to a patient's potential participation in this clinical trial, therefore not all
inclusion/exclusion criteria are listed.

Key Inclusion Criteria

1. Diagnosis of HoFH by at least 1 of the following genotype or clinical criteria (all
patients on LDL apheresis must be diagnosed based on genotype):

1. Documented homozygous or compound heterozygous mutations in both low-density
lipoprotein receptor (LDLR) alleles

2. Presence of homozygous or compound heterozygous mutations in Apo B or PCSK9

3. Presence of double heterozygous mutations, i.e, mutations on different genes in
the LDLR, Apo B or PCSK9 alleles

4. Untreated TC >500 mg/dL (12.93 mmol/L) and TG <300 mg/dL (3.39 mmol/L) AND Both
parents with history of TC >250 mg/dL (6.46 mmol/L) OR cutaneous or tendinous
xanthoma before age 10

2. Receiving a stable dose of a statin at the screening visit (documentation if statin
ineffective or patient unable to tolerate statin)

Key Exclusion Criteria:

1. Documented evidence of a null mutation in both LDLR alleles

2. Use of a PCSK9 inhibitor within 10 weeks from screening visit

3. Background medical lipid modifying therapy (LMT) that has not been stable for at least
4 weeks (6 weeks for fibrates, 24 weeks for mipomersen, 12 weeks for maximum tolerated
dose of lomitapide) before the screening visit.

4. LDL apheresis schedule/apheresis settings that have not been stable for at least 8
weeks before the screening visit or an apheresis schedule/settings that is not
anticipated to be stable over the next 24 weeks.

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

6. Chronic use of systemic corticosteroids, unless on a stable regimen of 10 mg daily
prednisone equivalent or less for at least 6 weeks prior to randomization. Note:
topical, intra-articular, nasal, inhaled and ophthalmic steroid therapies are not
considered as 'systemic' and are allowed

7. Systolic blood pressure >160 mmHg or diastolic blood pressure >100 mmHg at the
screening visit (1 repeat measurement is allowed).

8. History of a myocardial infarction (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.