Novel antisense drug 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-APOCIII-LRx for the treatment of patients with hypertriglyceridemia at risk for or with established CVD met its primary endpoint of significant triglyceride lowering.
The study also met several secondary endpoints with a positive safety and tolerability profile, according to a press release from the companies.
AKCEA-APOCIII-LRx is an antisense drug that inhibits apolipoprotein C-III production.
The phase 2 study aimed to evaluate the efficacy and safety of the drug in 114 patients (mean triglyceride levels, 285 mg/dL) with CVD or who were at high risk for CVD. Patients were assigned AKCEA-APOCIII-LRx or placebo and were treated for at least 6 months, according to the release. Four cohorts were formed to assess weekly, biweekly and monthly dosing, in addition to doses ranging from 10 mg to 50 mg per month.
Significant dose-dependent reductions in fasting triglycerides occurred at all dose levels compared with placebo, according to the release. Among patients assigned 50 mg per month, more than 90% achieved serum triglyceride levels of less than 150 mg/dL vs. less than 5% in the placebo group.
There were also significant reductions in VLDL, ApoC-III and remnant cholesterol with increases in HDL, according to the release.
“We are very encouraged that patients achieved a significant reduction in ApoC-III and triglyceride levels in this study, with the majority reaching triglyceride levels below the recognized threshold for cardiovascular risk,” Louis O’Dea, MD, chief medical officer for Akcea Therapeutics, said in the release. “These data further validate the consistent efficacy and safety profile that we have seen across Ionis’ [ligand-conjugated antisense] technology platform.”
Both groups had similar rates of treatment-emergent adverse events. The most commonly seen event being injection site reactions, which were mostly infrequent, mild and mainly occurred in patients assigned the weekly dose, according to the release. No safety signals were observed such as those associated with liver function, platelet counts and renal function.
“Based on the positive results from this study, we plan to rapidly pursue development of AKCEA-APOCIII-LRx for familial chylomicronemia syndrome,” O’Dea said in the release. “Because we were able to achieve substantial triglyceride lowering with this investigational medicine, we are also considering developing it for other rare and common diseases associated with elevated triglycerides.”
Disclosure: O’Dea is an employee of Akcea Therapeutics.
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