Antisense oligonucleotide therapy for triglyceride reduction shows positive top-line results
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Akcea Therapeutics and Ionis Pharmaceuticals announced that a phase 2 study of AKCEA-ANGPTL3-LRx for the treatment of patients with hypertriglyceridemia, type 2 diabetes and nonalcoholic fatty liver disease met its primary endpoint of significant triglyceride lowering.
The study also met several secondary endpoints with a favorable tolerability and safety profile, according to a press release from the companies.
AKCEA-ANGPTL3-LRx is an antisense oligonucleotide therapy that reduces production of angiopoietin-like 3 (ANGPTL3) protein in the liver.
The phase 2 study aimed to evaluate the efficacy and safety of AKCEA-ANGPTL3-LRx in 105 patients with hypertriglyceridemia, type 2 diabetes and nonalcoholic fatty liver disease. Patients were assigned AKCEA-ANGPTL3-LRx or placebo for 6 months. Three cohorts were formed to assess doses from 40 mg per month up to 80 mg per month, according to the release.
Significant dose-dependent reductions in fasting triglycerides were observed in patients assigned AKCEA-ANGPTL3-LRx compared with placebo. Other dose-dependent reductions were observed in apolipoprotein C-III, ANGPTL3, non-HDL, VLDL and total cholesterol. Patients assigned AKCEA-ANGPTL3-LRx did not have reductions in HbA1c or liver fat vs. placebo. Both groups had similar changes in platelets. AKCEA-ANGPTL3-LRx was well tolerated and had a favorable safety profile. Injection site reactions were the most common adverse event, although most were mild, according to the release.
“Results from the phase 2 study showed that antisense-mediated reduction of ANGPTL3 has the potential to address unmet needs in patients with cardiovascular diseases,” Louis O’Dea, chief medical officer at Akcea Therapeutics, said in the release.
Results from this study will be presented at a future meeting, according to the release.
Disclosure: O’Dea is an employee of Akcea Therapeutics.