Ionis achieves successful Phase 3 trial to treat rare diseases

pharmafile | December 19, 2016 | News story | Manufacturing and Production, Research and Development Akcea, Ionis, rare disease 

Ionis Pharmaceutial announced that the results of a Phase 3 trial into its treatment for hypertriglyceridemia met its primary endpoint. The treatment, developed by Ionis’ subsidiary Akcea, showed a significant reduction in triglycerides found in the blood as compared with those who were treated solely by placebo.

The antisense drug, volanesorsen, brought about a mean reduction of 71.2% in triglycerides after a period of 13 weeks of treatment; those who received a placebo only saw a reduction of 0.9%. The success of the trial was labelled as representing “an important milestone towards our planned regulatory filings for volanesorsen in the U.S., Europe and Canada in 2017,” by Paula Soteropoulos, president and chief executive officer of Akcea Therapeutics.

The trial saw patients suffering from the rare conditions familial partial lipodystrophy (FPL) and familial chylomicronemia syndrome (FCS) that both cause extremely high incidence of triglycerides in the patients’ blood. Both sets of patients are unable to effectively clear the triglycerides in the blood and therefore need help through medication. Patients are at high risk of suffering from problems with the pancreas, including acute pancreatitis in the long-term.

Data from the clinical trial indicated that the medication saw an effective reduction in the level of triglycerides but also had a safe profile. The most common side-effect of the medication was injection site reactions, which were mostly mild; though it did cause 13% to discontinue the trial. The only serious monitored event was a case of serum sickness but, after examination, it was concluded that it was unlikely to have been caused by the medication.

“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.  “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.”

The good news for Ionis comes on the back of successfully earning a milestone payment from AstraZeneca, from the successful development of a separate antisense drug.

Ben Hargreaves

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