BALANCE is a clinical study of an investigational drug for people living with Familial Chylomicronemia Syndrome (FCS).

The purpose of the BALANCE study is to determine if a potential new antisense treatment, or “investigational antisense drug,” is safe and effective in breaking down dietary fat that can lower triglycerides in the blood and potentially help improve a patient’s quality of life for people living with FCS.

• Familial Chylomicronemia Syndrome (FCS) is a genetic, life-threatening disease, in which the patient is unable to digest fats, which often results in high triglycerides (TGs). Sustained high TG levels can lead to pancreatitis.
• The investigational antisense drug (or placebo) will be randomly administered via subcutaneous injection beneath the skin. The Familial Chylomicronemia Syndrome (FCS) treatment is designed to target and reduce the activity of a protein in the body. Targeting this protein may help break down dietary fat and triglycerides.

To pre-qualify for BALANCE, you must:

• Be at least 18 years of age
• Have very high triglyceride levels (over 880 mg/dL or 10 mmol/L)
• Have a medical diagnosis of Familial Chylomicronemia Syndrome (FCS)
• A preferred history of pancreatitis
• Be willing to follow a diet of 20 grams of fat or less per day
• In addition to the requirements above, other tests and information will be required to determine your eligibility.

• If you qualify for and participate in the study, you will be randomly assigned to receive either the investigational drug or placebo via subcutaneous injection into the skin. (You have a greater chance of receiving the study therapy compared to the placebo).
• To help keep the study results as accurate as possible, you, the doctor, and staff conducting the study will not know if you are being given the investigational drug or the placebo (unless there is an emergency).
• Your total participation in the study will be for approximately 16 months, (22 visits total over 70 weeks) with only two visits needed onsite including screening for eligibility, treatment, and follow-up.
• Over 53 weeks, you will receive 13 doses of the study therapy or placebo.