Jan 22, 2020: Akcea Therapeutics and Ionis Pharmaceuticals announced positive topline results from the Phase 2 study of AKCEA-APOCIII-LRx in the treatment of patients with hypertriglyceridemia, patients who have established cardiovascular disease (CVD).
The objective of the Phase 2 study was to evaluate the safety and efficacy of altered doses and dosing frequencies of AKCEA-APOCIII-LRx.
The multicenter, randomized, double-blind, placebo-controlled, dose-ranging study that includes 114 patients with the clinical diagnosis of CVD or who are at high risk of CVD.
Participants were administered AKCEA-APOCIII-LRx or placebo via SC injection for at least six months with some patients being treated up to a year.
Weekly, bi-weekly, and monthly dosing was explored in the four cohorts with doses ranging from 10 mg to 50 mg of total monthly dose.
Observations from the AKCEA-APOCIII-LRx study included: Statistically considerable dose-dependent reductions in the fasting triglycerides compared to placebo at all dose levels.
Maximum once monthly dose of 50 mg, more than 90% of patients achieved serum triglycerides of ≤ 150 mg/dL, compared to below 5% of patients in the placebo group; mean triglyceride levels of patients at the baseline was 285 mg/dL, Major reductions in multiple additional risk factors, including apoC-III, very low-density lipoprotein (VLDL-C) and remnant cholesterol, compared to placebo.
Statistically considerable increases in the high-density lipoprotein cholesterol (HDL-C) compared to placebo at all dose levels. Around 85% of the patients completed treatment and the rate of treatment discontinuation was comparable between the active and the placebo groups.
About AKCEA-APOCIII-LRx
AKCEA-APOCIII-LRx (ligand conjugated antisense (LICA) drug) was discovered by the Ionis and co-developed by Akcea and Ionis.
It is an antisense drug developed using Ionis’ proprietary LIgand Conjugated Antisense (LICA) technology platform and is designed in order to inhibit production of apolipoprotein C-III (apoC-III), a protein produced in the liver that plays a essential role in the regulation of serum triglycerides. https://ir.akceatx.com/news-releases/news-release-details/akcea-and-ionis-report-positive-topline-phase-2-results-akcea
