US-based Ionis Pharmaceuticals’ affiliate Akcea Therapeutics has launched a Phase II clinical programme of AKCEA-ANGPTL3-L to treat rare hyperlipidemias.
AKCEA-ANGPTL3-L is being developed to minimise the production of angiopoietin-like 3 (ANGPTL3) for cardioprotective effect and decreased risk of insulin resistance and diabetes mellitus.
The Phase II programme will include three clinical trials for familial chylomicronemia syndrome (FCS), familial partial lipodystrophy (FPL), and homozygous familial hypercholesterolemia (HoFH).
In addition to evaluating safety, tolerability, pharmacokinetics, and pharmacodynamics, the trials will aim to characterise the potential of AKCEA-ANGPTL3-L in specific patient populations.
Akcea Therapeutics chief medical officer Louis O’Dea said: “ANGPTL3 is a key regulator of a number of lipid and metabolic pathways and as such is an important target for the therapeutic management of rare hyperlipidemias, such as FCS, FPL, and HoFH.
“We are committed to developing additional therapeutic options for these patient populations whose significant medical needs are currently greatly underserved.”
The first trial under the programme will include an assessment of subcutaneous, once weekly injection of AKCEA-ANGPTL3-L over a period of 13 weeks in subjects with FCS.
Scheduled to report top-line results next year, this trial will be followed by studies in FPL and HoFH patients.
AKCEA-ANGPTL3-L was found to be well-tolerated during a Phase I/II clinical trial conducted in subjects with increased triglycerides, with an 85% dose-dependent decrease in ANGPTL3 protein after six weeks.
Performed for various doses of the drug candidate, the trial further showed similar reductions in triglycerides; low-density lipoprotein (LDL), very low-density lipoprotein (VLDL), and non-high desnsity lipoprotein (HDL) cholesterol; apolipoprotein B; and apolipoprotein C-III protein.
