A new drug to treat the lethal condition pulmonary arterial hypertension (PAH) has shown impressive phase III trial results for efficacy and safety.
Actelion’s macitentan has the potential to replace its current PAH treatment, Tracleer, as the company’s leading product – and even to become the standard treatment for PAH.
The trial results boost the Swiss biotech company’s prospects of being acquired by a major pharma company.
Tracleer currently accounts for 90% of Actelion’s sales, but will lose patent protection in 2015 and is facing competition from Gilead’s PAH drug Letairis.
The SERAPHIN phase III study with 742 patients – the largest ever clinical study of a PAH treatment – showed that macitentan reduced the incidence of morbidity and mortality by 45% relative to placebo over three and a half years.
In addition, the drug did not show the negative side-effects on liver function associated with Tracleer, enabling it to compete more effectively with Letairis.
PAH is a chronic, life-threatening disease in which high blood pressure in the pulmonary arteries affects both lung and heart function.
Finance analyst UBS has suggested that macitentan could not only dominate the PAH market but increase its size, with potential annual sales of up to $2.8bn.
Actelion expects to apply for EMA and FDA marketing authorisation for the product by the end of 2012.
Macitentan, a dual endothelin receptor antagonist, was created through a tailored drug discovery process. It has superior sustained receptor binding and tissue penetration properties relative to other ERAs, and its propensity for drug-drug interactions has been shown to be low.
Two senior advisors on the SERAPHIN trial highlighted the study’s importance. Lewis J. Rubin, Emeritus Professor at the University of California, San Diego, said: “With this well-designed PAH study, Actelion pursued an ambitious goal to focus on outcome benefits as the primary endpoint. The impressive results of this landmark study are setting a new standard in how to conduct studies in this devastating disease.”
Gerald Simonneau, Head of the Department of Pulmonary Disease and Intensive Care Unit at the Hospital Antoine Beclere-Clamart in France, added: “These results represent an important milestone in the history of clinical trials in PAH and show that macitentan has the potential to offer a new treatment paradigm for these patients.”