Actelion Pharmaceuticals has reported positive outcomes from the Portico trial after meeting the primary endpoint of the study.
Portico is a Phase IV, prospective, randomised, placebo-controlled, double-blind, multicentre, parallel-group trial that examined the efficacy and safety of Opsumit (macitentan) against placebo for the treatment of patients with portopulmonary hypertension (PoPH).
During the trial, 85 patients were randomised in a 1:1 ratio into two treatment groups to receive 10mg of macitentan or placebo for a 12-week double-blind treatment period.
After 12 weeks of treatment, the trial met its primary endpoint by showing a reduction of 35% in pulmonary vascular resistance (PVR) for macitentan compared with placebo.
Macitentan also significantly improved mean pulmonary arterial pressure (mPAP) and cardiac index in comparison with placebo.
In addition, no significant difference between macitentan and placebo groups was observed in six-minute walk distance (6MWD) or WHO Functional Class (FC).
The most common adverse events were reported to be peripheral edema and headache.
The trial also found that the safety of macitentan was similar to those observed in previous clinical trials of PoPH.
Portico trial lead investigator Olivier Sitbon said: “The findings of PORTICO are relevant because if patients with PoPH can be treated to successfully lower pulmonary vascular pressure and resistance, more patients may be eligible for liver transplant as they will potentially have a better prognosis for this surgery.
“The fact that the hepatic safety profile of macitentan in patients with PoPH was consistent with that observed in previous trials is particularly reassuring, as PoPH patients are typically excluded from PAH clinical trials on safety grounds.”
Opsumit is an orally active endothelin receptor antagonist (ERA) that is currently being used for the treatment of pulmonary arterial hypertension (PAH) in the US and Europe.