Akcea Therapeutics, Inc., declared positive topline outcomes from Phase 2 trial of AKCEA-ANGPTL3-LRx in patients suffering from non-alcoholic fatty liver disease (NAFLD), type 2 diabetes, and hypertriglyceridemia. The clinical trial achieved the primary endpoint of considerable triglyceride lowering and several secondary endpoints with a positive tolerability and safety profile.
AKCEA-ANGPTL3- LRx is an under-development investigational antisense oligonucleotide treatment for treatment of patients enduring certain metabolic and cardiovascular diseases.
The dose-ranging Phase 2 trial is aimed at evaluating the efficacy and safety of AKCEA-ANGPTL3-LRx. This is a randomized, multicenter, placebo-controlled, double blind trial comprising around 105 patients suffering from type 2 diabetes, NAFLD, and hypertriglyceridemia. The patients involved in the study were given placebo and AKCEA-ANGPTL3-LRx via subcutaneous injection for six months. Weekly and monthly medication was studied in three cohorts with dosage between 40-80 mg of the overall monthly dose.
AKCEA-ANGPTL3-LRx study observed no decrease in hemoglobin A1C and liver fat in comparison with the placebo. It was detected that AKCEA-ANGPTL3-LRx was mostly well-tolerated and exhibited a favorable tolerability and safety profile. The most common adverse event in the study was injection site reactions, which were usually mild.
Louis O’Dea, Chief Medical Officer, Akcea Therapeutics, reportedly stated that outcomes from the Phase 2 trial revealed that antisense-mediated minimization of ANGPTL3 has the capability to address unmet requirements in patients with cardiovascular disorders. The company is grateful to the physicians, patients, and families, who partook in the trial and are ready to disclose the data with Pfizer to establish strategies for potential future advancement of AKCEA-ANGPTL3-LRx, he further added.
The AKCEA-ANGPTL3-LRx Phase 2 study validates the benefits of the LICA technology platform for large symptoms, such as cardiovascular disease. Akcea has been continuously observing consistent performance of therapy with low volume monthly doses throughout the LICA programs in terms of safety, patient tolerability, and efficacy.