ISIS 678354-CS7: An Open-Label Study of AKCEA-APOCIII-LRX Administered Subcutaneously to Patients With Familial Chylomicronemia Syndrome (FCS) Previously Treated With Volanesorsen (ISIS 304801)

Familial Chylomicronemia Syndrome

1. Safety endpoints include a proportion of patients who show the following changes from Baseline to Week 53, Week 105, Week 157 and Week 209 :-Decrease in platelet (PLT) count by>30% -Decrease in PLT count by>50% - PLT count value<50,000mm3 -Major bleeding events -Clinically relevant non-major bleeding events -Decrease in eGFR by>30% -Decrease in eGFR by>50% - UPCR≥1000mg/g -UACR≥500mg/g- ALT or AST>5×ULN- Total bilirubin>2.0mg/dL- ALT or AST>3×ULN and total bilirubin≥2xULN, 2. Tolerability assessments will include adverse events (AEs), clinical laboratory test, and use of concomitant medications

1. PK assessments include determination of trough (pre-dose) and post-treatment plasma ISIS 678354 concentrations, 2. PD endpoints include: 1. Change and percent change from Baseline to Week 53, Week 105, Week 157 and Week 209 in fasting: - Triglycerides (TG) - APOC-III, very low-density lipoprotein (VLDL)-C, chylomicron-TG, total cholesterol (TC), non-HDL-C, low-density lipoprotein (LDL)-C, apoB, apoB48, HDL-C, ApoA-1 2. Event rate of acute pancreatitis (Week 1 through Weeks: 53, 105, and 157)

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