Apolipoprotein (ApoC-III) is a protein target for therapies that Akcea is developing to lower levels of triglycerides in the blood. The protein ApoC-III attaches to particles in the blood that contain triglycerides, and it is thought to act like a braking system on the body’s process for removing triglycerides from the bloodstream. High levels of ApoC-III slow down the metabolism and/or removal of triglycerides from the bloodstream, which can lead to high levels of triglycerides in the blood.

Akcea’s lead program, volanesorsen, is an antisense drug designed to reduce ApoC-III protein production and to lower triglycerides to treat patients with select dyslipidemias, including those with familial chylomicronemia syndrome (FCS) and familial partial lipodystrophy (FPL).

Four global trials have formed the Phase 3 program for volanesorsen. Two of these studies — the APPROACH Study in patients with FCS and the COMPASS Study in patients with severe hypertriglyceridemia — both met their primary endpoints of reducing triglycerides in the respective patient populations and showed a statistically significant decrease in pancreatitis attacks in FCS patients with a history of high frequency pancreatitis. Two additional Phase 3 studies — the BROADEN Study in patients with FPL and the APPROACH Open Label Study are ongoing.

Read more about the results of the APPROACH Study.

Following completion of the APPROACH clinical trial, the pivotal phase 3 study of volanesorsen in FCS, Akcea is now conducting the APPROACH Open Label Study, a multi-center study to further evaluate the efficacy of volanesorsen measured by percent change in fasting triglyceride levels from baseline and to assess the durability of the efficacy with extended administration of volanesorsen.

Akcea is conducting the BROADEN Study, a randomized, placebo-controlled, double-blind Phase 2/3 clinical trial for volanesorsen in the treatment of FPL, that is currently enrolling patients. The main purpose of the BROADEN Study is to assess the effects of volanesorsen on triglyceride levels and other metabolic derangements in people with FPL.

The U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) have granted orphan drug designation to volanesorsen for the treatment of patients with FCS. The EMA has also granted orphan drug designation to volanesorsen for the treatment of patients with FPL.

We are in the process of preparing the regulatory filing as well as commercial build-out to support the anticipated launch of this program.

Expanded Access / Compassionate Use

At this time, Akcea does not have an expanded access program or compassionate use program.