Trial to Evaluate Safety and Tolerability of ALN-PCS02 in Subjects With Elevated LDL-Cholesterol (LDL-C)

A Phase 1, Randomized, Single-blind, Placebo-Controlled, Single Ascending Dose, Safety, Tolerability and Pharmacokinetics Study of ALN-PCS02 in Subjects With Elevated LDL-Cholesterol (LDL-C)

The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of a single dose of ALN-PCS02 in subjects with Elevated LDL-Cholesterol (LDL-C).

Inclusion Criteria:

• Elevated LDL-C of >3.0 mmol/L and <5.7 mmol/L
• Fasting triglyceride concentration ≤2.8 mmol/L
• Body weight >60.0 kg; body mass index (BMI) between 19.00 kg/m2 and <35.00 kg/m2
• Adequate blood counts, liver and renal function
• May not received any lipid lowering drug/agent within the 30 days prior to the screening
• Non-smokers for at least 3 months
• Women of child-bearing potential must have a negative pregnancy test, cannot be breast feeding, and must use an adequate method of birth control
• Males agree to use appropriate contraception
• Willing and able to comply with protocol-required visit schedule and visit requirements and provide written informed consent

Exclusion Criteria:

• Known hepatitis B surface antigen (HBsAg), hepatitis B virus (HBV), hepatitis C virus (HCV) or human immunodeficiency virus (HIV) infection
• Multiple drug allergies or know sensitivity to oligonucleotide
• History of drug abuse and/or alcohol abuse
• Receiving an investigational agent within 3 months prior to study drug administration
• Subjects with safety laboratory test results deemed clinical significant by the Investigator;
• Received prescription drugs within 4 weeks of first dosing
• Subjects who have donated more than 500 mL of blood within the 3 months prior to ALN-PCS02 or placebo administration;
• Received megadose vitamin therapy or dietary supplements within 4 weeks prior to screening
• Subjects who have used prescription drugs within 4 weeks of first dosing
• Considered unfit for the study by the Principal Investigator
• Employee or family member of the sponsor or the clinical study site personnel