Ionis Pharmaceuticals and its affiliate Akcea Therapeutics have reported positive data from the Phase II clinical trial of AKCEA-APOCIII-L conducted in hypertriglyceridemia patients with or at risk of cardiovascular disease (CVD).
AKCEA-APOCIII-L is a ligand conjugated antisense (LICA) drug candidate being developed to minimise apolipoprotein C-III (apoC-III) production.
ApoC-III is associated with the regulation of serum triglycerides and decreased apoC-III levels are linked to low triglycerides levels and lower CVD risk.
The multi-centre, randomised, double-blind, placebo-controlled, dose-ranging Phase II trial investigated the safety and efficacy of the drug at various subcutaneous doses and dosing frequencies in 114 participants.
Top-line results showed that the trial met the primary endpoint with a significant reduction in triglycerides at all dose levels compared to placebo.
The trial also met various secondary goals, including a significant decrease in apoC-III, very low-density lipoprotein (VLDL-C), and a rise in high-density lipoprotein cholesterol (HDL-C) versus placebo.
Further analysis revealed a favourable safety and tolerability profile, with no safety signals and treatment-emergent adverse events (TEAEs) comparable to that of placebo. The most common adverse event reported was reactions at the injection site.
Akcea Therapeutics chief medical officer Louis O’Dea said: “Based on the positive results from this study, we plan to rapidly pursue the development of AKCEA- APOCIII-L for familial chylomicronemia syndrome or FCS.
“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.”
Ionis and Akcea plan to assess AKCEA-APOCIII-L for the treatment of familial chylomicronemia syndrome and may expand to others indications.
In November, the companies launched a Phase III trial of AKCEA-TTR-L to treat polyneuropathy in patients with hereditary TTR (hATTR) amyloidosis.