Akcea Therapeutics closes enrolment in Phase lll trial of volanesorsen to treat hypertriglyceridemia

17th May 2016 (Last Updated May 17th, 2016 18:30)

Akcea Therapeutics, a subsidiary of Ionis Pharmaceuticals, has completed patient enrolment in a Phase lll trial (COMPASS study) of volanesorsen in patients with severe hypertriglyceridemia.

Akcea Therapeutics, a subsidiary of Ionis Pharmaceuticals, has completed patient enrolment in a Phase lll trial (COMPASS study) of volanesorsen in patients with severe hypertriglyceridemia.

The randomised, double-blind, placebo-controlled COMPASS study has enrolled 115 patients.

Currently under development, volanesorsen is an antisense drug that expects to treat patients with severely high triglycerides, either as a single agent or in combination with other triglyceride-lowering agents.

The drug is designed to reduce the production of ApoC-III, a protein produced in the liver that plays a central role in the regulation of plasma triglycerides.

"Our success in reaching this important milestone ahead of schedule demonstrates our ability to successfully conduct a complex, multi-centre global clinical trial."

The COMPASS study is one of three ongoing global trials that shape the Phase lll programme for volanesorsen and is designed to provide safety data in support of future regulatory filings in the US, EU and Canada for volanesorsen in two indications, familial chylomicronemia syndrome (FCS) and familial partial lipodystrophy (FPL).

FCS is a rare, genetic disorder and may also be called familial chylomicronemia or Fredrickson Type 1 hyperlipoproteinemia, or familial lipoprotein lipase deficiency.

FPL is a rare lipid disorder characterised by abnormal fat distribution across the body and a range of metabolic abnormalities, such as severe insulin resistance, dyslipidemia and hypertriglyceridemia, hepatic steatosis and, in affected women, features of hyperandrogenism.

Akcea's two other ongoing global, multi-centre Phase lll trials of volanesorsen include the APPROACH study in patients with FCS and the BROADEN study in patients with FPL.

The APPROACH Phase lll trial in FCS patients completed enrolment last year and data from this study are expected in the first half of next year.

Data from the BROADEN Phase lll study in FPL is aimed to be received by the first half of 2018.

Akcea Therapeutics chief medical officer Dr Louis O'Dea said: "The COMPASS study was designed to yield important data to strengthen our robust safety database for volanesorsen beyond what we will collect in our ongoing APPROACH and BROADEN trials.

"Our success in reaching this important milestone ahead of schedule demonstrates our ability to successfully conduct a complex, multi-centre global clinical trial.

"Most importantly, we expect the safety data available from the 115 patients enrolled in this trial to bolster what we anticipate will be a compelling case in support of timely access to volanesorsen for people with FCS and FPL."