Akcea and Ionis report positive topline phase 2 study results of AKCEA-ANGPTL3-LRx
[caption id="attachment_9277" align="aligncenter" width="747"] Press Release[/caption]
Favorable safety and tolerability were observed
Primary and multiple secondary endpoints were met with significant reduction in triglyceride levels, additional lipid parameters and ANGPTL3
BOSTON?and?CARLSBAD, Calif.,?Jan. 28, 2020?(GLOBE NEWSWIRE) --?Akcea Therapeutics, Inc.?(NASDAQ: AKCA), a majority-owned affiliate of?Ionis Pharmaceuticals, Inc., and?Ionis Pharmaceuticals, Inc.?(NASDAQ: IONS), today announced positive topline results from the Phase 2 study of AKCEA-ANGPTL3-LRx?in patients with hypertriglyceridemia, type 2 diabetes and non-alcoholic fatty liver disease (NAFLD). The study met the primary endpoint of significant triglyceride lowering and multiple secondary endpoints with a favorable safety and tolerability profile. AKCEA-ANGPTL3- LRx?is an investigational antisense oligonucleotide therapy being developed to treat patients with certain cardiovascular and metabolic diseases. The objective of the dose-ranging Phase 2 study was to evaluate the safety and efficacy of AKCEA-ANGPTL3-LRx. The multicenter, randomized, double-blind, placebo-controlled study included 105 patients with hypertriglyceridemia, type 2 diabetes and NAFLD. Participants were administered AKCEA-ANGPTL3-LRx?or placebo via subcutaneous injection for six months. Weekly and monthly dosing was explored in three cohorts with doses ranging from 40 mg to 80?mg of total monthly dose. Observations from the AKCEA-ANGPTL3-LRx?study included:- Statistically significant dose-dependent reductions in fasting triglycerides compared to placebo at all dose levels
- Dose-dependent reductions in ANGPTL3, apoC-III, very low-density lipoprotein (VLDL-C), non-HDL cholesterol and total cholesterol compared to placebo
- No reductions in liver fat or hemoglobin A1C compared to placebo
- AKCEA-ANGPTL3-LRx?was generally well-tolerated and demonstrated a favorable safety and tolerability profile. The most common adverse event was injection site reactions, which were mostly mild
- Changes in platelets were similar between placebo and treated groups
- JAMA Cardiol. 2018?Oct 1;3(10):957-966.
- N Engl J Med. 2017 Jul 20;377(3):222-232.