Zacks_Analysts' Blog : Actelion Candidate Misses Goal - Analyst Blog

European biotechnology company Actelion Ltd. (ALIOF) recently announced disheartening data from a mid stage trial (MUSIC; n=178) of its pipeline candidate, macitentan, evaluating its use in the treatment of idiopathic pulmonary fibrosis (IPF). The double-blind, randomized, placebo controlled, multicenter study failed to meet the primary end-point of forced vital capacity (a measure of lung function).

The study, however, showed that the safety and tolerability of macitentan was acceptable, particularly the liver enzyme elevations were no different in patients treated with macitentan from patients on placebo. Actelion will not initiate a late stage trial for the candidate for the IPF indication.

Macitentan is an endothelin receptor antagonist (ERA) designed to protect tissues from the damaging effect of elevated endothelin, specifically in the cardiovascular system.  Macitentan is a follow-on compound to Actelion’s lead drug Tracleer. Tracleer is one of the three products which Actelion currently markets for the treatment of pulmonary arterial hypertension (PAH). The other two are Ventavis and Veletri. Actelion also markets another drug Zavesca for the treatment of Gaucher’s disease.

Actelion is currently conducting a late stage morbidity/mortality PAH trial for macitentan (SERAPHIN). Enrollment of 742 patients for the study was completed in December 2009.  Actelion is expected to present data from the study in the first half of 2012. This data presentation will be an important clinical catalyst for Actelion in the near term.

Our Take

The shares of Actelion carry a Zacks #3 Rank (short-term Hold rating). Actelion has a dominant position in the multi-billion dollar PAH market with drugs like Tracleer and Veletri in its product suite. We also like the company’s strong pipeline. We however, feel Actelion needs to diversify its product portfolio beyond the PAH franchise to sustain growth.