Drug Development

Ionis reports a positive PhIII for volanesorsen in rare metabolic disorders

Louis O'Dea, Akcea

Louis O’Dea, Akcea

Investigators for Ionis Pharmaceuticals $IONS say they gathered positive evidence of efficacy for their late-stage drug volanesorsen in rare cases of metabolic diseases.

The drug produced a mean reduction of 71.2% in triglycerides among the 113 patients in the study who took the treatment over 13 weeks. That compares to a negligible reduction among the placebo arm of the study.

In addition, 82% of patients treated with volanesorsen achieved triglyceride levels less than 500 mg/dl after 13 weeks of treatment, compared to 14% of placebo-treated patients (p<0.0001).

There was one case of serum sickness recorded in the study, which may have been drug related. Ionis, though, says it isn’t likely that the drug sparked that case.

This is one of several late-stage studies underway for this drug, says Ionis, which plans to review its progress in early January. The study recruited patients with rare metabolic disorders: familial chylomicronemia syndrome and familial partial lipodystrophy by targeting apolipoprotein C-III with the antisense drug.

“The findings in COMPASS reinforce the efficacy and safety of volanesorsen observed in Phase 2 studies across multiple patient populations, including FCS,” said Dr. Louis O’Dea, chief medical officer for Akcea, a subsidiary of Ionis.  “No drug available today has demonstrated the magnitude of the triglyceride reductions observed with volanesorsen.  These results confirm the potential value of targeting ApoC-III to lower triglycerides in patients who have high unmet need with potentially life-threatening consequences.”


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RAPS Regulatory Convergence 2017