Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Participants With Lysosomal Acid Lipase Deficiency

The study was composed of a screening period, a treatment period, and a post-treatment
follow-up period (including an End of Study visit). Participants who successfully completed
screening assessments to determine study eligibility were allocated to 3 sequential cohorts
(0.35, 1, or 3 milligrams/kilogram [mg/kg]). Within each cohort, one participant was
initially dosed and, if sebelipase alfa was deemed safe and well tolerated in this
participant (based on at least 24 hours of monitoring), dosing was allowed to be initiated
for the remaining participants in the cohort. Initiation of dosing in the next cohort
occurred only after all participants in the preceding cohort had been monitored for at least
5 days after the second infusion, without any evidence of significant safety signals, and an
independent Safety Committee had reviewed the cumulative safety data and provided their
recommendation on the acceptability of beginning dosing in the next cohort.

Cholesteryl Ester Storage Disease (CESD) is the late onset phenotype for LAL Deficiency, a
lysosomal storage disorder, which also has an early onset phenotype known as Wolman disease
that primarily affects infants. CESD can present in childhood but often goes unrecognized
until adulthood when the underlying pathology is advanced. Many of the signs and symptoms are
common to participants with other liver conditions.

CESD is an autosomal recessive genetic condition and is characterized by hepatomegaly,
persistently abnormal liver function tests and type II hyperlipidemia. Splenomegaly and
evidence of mild hypersplenism may affect some participants. Untreated, CESD may lead to
fibrosis, cirrhosis, liver failure and death.

Inclusion Criteria:

- Male or female participants ≥ 18 and ≤ 65 years of age

- Documented decreased LAL activity

- Evidence of liver involvement

Exclusion Criteria:

- Clinically significant concurrent disease, serious inter-current illness, concomitant
medications or other extenuating circumstances

- Clinically significant abnormal values on laboratory screening tests, other than liver
function or lipid panel tests

- Aspartate aminotransferase and/or alanine aminotransferase persistently elevated > 3x
upper limit of normal at screening

- Previous hemopoietic bone marrow or liver transplant

- Current history of alcohol abuse