The drug: Macitentan
The disease: Pulmonary arterial hypertension
The developer: Actelion
Peak sales potential: $1.4 billion to $3 billion

Talk about your high-risk development programs. Facing the loss of patent protection for its blockbuster lung drug Tracleer, Switzerland's Actelion had all of its eggs in the next-gen basket for macitentan. So when the Phase III SERAPHIN readout came at the end of April demonstrating that the drug worked as hoped, company execs could breathe a big sigh of relief.

According to investigators, a 10-mg dose of macitentan reduced the risk of mortality and morbidity 45% compared with a placebo, while the 3-mg dose cut the same risks 30%. It managed those results without evidence of liver toxicity, giving it an edge on Tracleer. And in this heated M&A environment, the promising Phase III trial quickly triggered fresh speculation of a possible buyout. 

That data allowed Actelion to restructure its R&D efforts and shake off any lingering questions about investor criticisms, and it once again revived the persistent buyout rumors that always seem to linger about this company.

When Tracleer goes generic in 2015, macitentan should be in place on the market, provided regulators follow through with an approval. Applications are slated for the end of the year.

For more:
Actelion will slash 135 staffers, refocus on rare diseases in R&D shakeup
Actelion surges as positive PhIII macitentan data reignites buyout buzz
Actelion braces for looming pivotal data on blockbuster macitentan program
Actelion drug flunks PhII but safety data bolsters confidence in blockbuster PhIII


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