2020-12-06| Trials & Approvals

Ionis Initiates Phase 3 Trials for Familial Chylomicronemia Syndrome Treatment

by Daniel Ojeda
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On December 1st, Ionis Pharmaceutical announced the start of Phase 3 clinical trial to determine the efficacy of AKCEA-APOCIII-LRx for the treatment of adults with familial chylomicronemia syndrome (FCS). FCS is characterized by high levels of triglycerides, and the risk of debilitating and potentially fatal acute pancreatitis. This treatment aims to reduce the levels of a protein involved in triglyceride production. If successful, it would be the first treatment for this disease.

Familial chylomicronemia syndrome (FCS) is a disease caused by mutations in the lipoprotein lipase (LPL) gene. The protein coded by this gene oversees the breaking down of triglycerides in the blood. Mutations in the LPL gene result in the reduced or impaired activity of the LPL protein, which results in an accumulation of chylomicrons. These are composite lipids formed by 90% triglycerides. In turn, the accumulation of chylomicrons reduces blood flow through the pancreas, which can result in acute pancreatitis, permanent organ damage, and pancreatogenic diabetes. Currently, there is no treatment available for FCS.

In order to address this unmet medical need, IONIS pharmaceutical developed the investigational antisense medicine AKCEA-APOCIII-LRx. It reduces the levels of the protein apolipoprotein C-III, which regulates the triglycerides in the blood. By genetically reducing the levels of this protein, IONIOS aims to reduce the levels of triglycerides and consequently reduce the levels of chylomicrons.


BALANCE Clinical Trial

The efficacy of AKCEA-APOCIII-LRx will be evaluated in the BALANCE trial, a global, multi-center, double-blind, placebo-controlled Phase 3 clinical trial. The 60 participants will be divided into a 2:1 ratio receiving AKCEA-APOCIII-LRx or placebo. They will receive a subcutaneous injection every four weeks during the 53-week treatment period. At six months, the percent change from baseline in fasting triglyceride levels will be compared to placebo to see if there is a statistical difference with AKCEA-APOCIII-LRx treatment.

“Initiation of the Phase 3 BALANCE study is a significant milestone for the FCS patient community and for our company. This study also reflects our continuing commitment to develop novel treatment options for patients with unmet medical needs such as those living with FCS,” said Richard S. Geary, Ph.D., executive vice president of development. “AKCEA-APOCIII-LRx is the second Ionis antisense medicine that we are developing with FCS patients in mind. We are hopeful about the prospect of bringing forward a new, safe, and effective treatment since these patients have limited available options.”



Reducing the levels of triglycerides by targeting the Apolipoprotein C-III protein is a promising approach. ARO-APOC3 is an RNAi therapeutic developed by Arrowhead Pharmaceutical is currently in Phase 1 clinical trials aimed at reducing the expression levels of the protein.

Additionally, IONIS is developing a second antisense medicine for the treatment of FCS. AKCEA-ANGPTL3-LRx targets the Angiopoietin-like 3 gene, which codes for a protein involved in the regulation of triglycerides, cholesterol, and glucose. AKCEA-ANGPTL3-LRx finished Phase 2 clinical trials in March 2020. All of these offer the possibility to address the unmet needs of people with FCS.

By Daniel Ojeda, Ph.D.

Related Article: Ascentage Pharma’s Bcl-2 Inhibitor Enters Clinical Trials in Europe



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