The results are a disappointment to the Swiss company as it seeks to expand the indication and market for its lead product.
In the idiopathic pulmonary fibrosis (IPF) study, the primary endpoint was defined as change from baseline in six-minute walk distance after twelve months. No improvement was found in this endpoint.
Positive trends were observed in the IPF study for pre-defined secondary endpoints, such as the combined incidence of death or treatment failure at 12 months. The company has revealed that it will now initiate discussions with scientific experts and regulatory authorities worldwide on the design of the phase III mortality-morbidity study based on these positive trends.
In the study with patients suffering from pulmonary fibrosis related to systemic sclerosis, no effect was observed on either primary or secondary endpoints.
“The findings related to the six-minute-walk test in pulmonary fibrosis are disappointing. Nevertheless, I am very impressed by clinically relevant trends in the pre-defined secondary endpoints related to death or disease worsening,” said Dr Jean-Paul Clozel, chief executive officer of Actelion.