Study has Commenced for a New Potential Familial Chylomicronemia Syndrome Treatment


Hypertriglyceridemia is the term used to describe abnormally high numbers of triglycerides in the blood. People with Hypertriglyceridemia have an increased risk of heart disease, stroke, and heart attack.

Familial chylomicronemia syndrome (FCS) is a rare form of hypertriglyceridemia caused by a gene mutation which results in severely high levels of triglycerides. FCS is genetic and people with the condition usually have more than 900mg of triglycerides per deciliter of blood. Patients with FCS represent the top .1% of measured triglycerides. It can cause type II diabetes mellitus, cognitive issues, chronic abdominal pain, hepatic steatosis, and acute pancreatitis (which can be fatal) among other symptoms.

Unfortunately, there are no current treatments which are effective for treating FCS.

Thankfully, researchers are working to change this.


Arrowhead Pharmaceuticals has just recently announced that the first patient in their Phase 1 trial for hypertriglyceridemia and FCS has been dosed. The trial is called AROAPOC31001 and it is examining an RNAi based therapy called ARO-APOC3 which targets apolipoprotein C-III, otherwise known as apoC-III.

ApoC-III works to regulate triglycerides which makes it a valuable target in FCS research. Previous studies have shown that people with apoC-III loss-of-function mutations have reduced triglyceride levels and reduced risk for developing cardiovascular disease.

This Phase 1 study has two parts. The first is a single-ascending dose examination including 4 cohorts which each has 10 healthy, adult volunteers who have elevated triglyceride levels. Each participant will be given one dose of ARO- APOC3 or placebo. Dose levels of ARO-APOC3 will range from 25mg to 200mg. The second component of this study is a multiple-dose segment. It will include 3 different patient cohorts of individuals with hypertriglyceridemia and 1 patient cohort of individuals with FCS. These cohorts will all receive two monthly doses of APOC3. In total, this Phase 1 trial will enroll up to 63 participants.

The ultimate goal of this study is to examine the pharmacokinetics, pharmacodynamics, tolerability, and overall safety of ARO-APOC3. It will evaluate these effects in health volunteers, FCS patients, and hypertriglyceridemia patients.

Hopefully we will see positive results published from this study soon, leading us closer to better treatment options for FCS and hypertriglyceridemia patients.

You can read more about this Phase 1 study here.

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