Actelion’s PGI2 Receptor Demonstrates Efficacy In PAH Patients

Actelion has obtained positive result in a phase IIa study, with orally active non-prostanoid PGI2 receptor agonist in pulmonary arterial hypertension (PAH) patients.

In the 43 patient placebo-controlled double-blind study to assess efficacy, safety and tolerability, the primary endpoint of change from baseline in pulmonary vascular resistance (PVR) was met with high statistical significance (P<0.01). The compound was well tolerated in the study, said the company.

Jean-Paul Clozel, MD and chief executive officer of Actelion, said: “Together with our partner Nippon Shinyaku, we are very excited by the remarkable results we have seen in this study. I am especially pleased as efficacy was achieved on top of background therapy with either an endothelin receptor antagonist and/or a PDE-5 inhibitor. This efficacy with good tolerability makes us determined to accelerate our development program.”

Actelion will now approach the FDA to finalize the design for a phase III program with a morbidity/mortality endpoint. Consequently, Actelion expects to initiate this program before the end of 2009.