Actelion Lung Drug Meets Main Goal in Pivotal Study

Actelion Ltd. CEO Clozel
Jean-Paul Clozel, chief executive officer of Actelion Ltd. Source: Actelion Pharmaceuticals Ltd/copyright 2011 via Bloomberg

Actelion Ltd.’s experimental lung drug macitentan met the main goal of a late-stage clinical trial, giving the company a possible successor to a treatment that accounts for 89 percent of sales. The shares rose the most since the company went public in 2000.

Patients with pulmonary arterial hypertension given 10-milligram doses of macitentan were 45 percent less likely to experience worsening of the disease than those who got a placebo, Actelion said in a statement today. The drug showed a trend toward reducing premature deaths, though the result wasn’t statistically significant, the Allschwil, Switzerland-based company said.

The results will help sustain sales growth when Tracleer, the company’s top-selling drug, starts losing patent protection in 2015, said Adrian Howd, an analyst at Berenberg Bank in London. It’s also likely to make Actelion a takeover target, he said.

“A lot of companies would be interested in looking at Actelion,” Howd said by phone. “They have a specialty franchise in a disease area with a poor survival rate and a lot of scope for future therapies. This actually increases the chance of an acquisition in the future.”

Seeking Approval

Actelion jumped 14 percent to 38.40 Swiss francs in Zurich, the biggest gain since 2000, giving the company a market value of 5 billion francs ($5.5 billion). The stock has lost 23 percent in the past year including reinvested dividends, compared with an 18 percent gain in the Bloomberg Europe Pharmaceutical Index of 18 companies.

Actelion plans to submit macitentan to regulators by the fourth quarter, the company said.

Bayer AG or Amgen Inc. may be interested in buying Actelion, Peter Welford, an analyst at Jefferies International Ltd. in London, said by phone today. Spokesmen for Bayer and Actelion, and a spokeswoman for Amgen, declined to comment.

Bayer has its own experimental pulmonary hypertension drug, riociguat, which is in late-stage trials. Phase-three data on the drug is due by the second half of this year, the company said.

The results bolster Actelion’s Chief Executive Officer Jean-Paul Clozel, who said last year that macitentan’s prospects justified keeping the company independent. He fought off an attempt by a hedge fund to oust board members and force the company to consider a sale. The board would have had to consider “all options” if macitentan failed, Clozel said Jan. 10.

‘Extensive Effect’

“I was not expecting such an extensive effect,” Clozel said on a call with analysts today. “Our previous evaluation might have to be revisited,” he said in response to a question about comments he made previously about macitentan and a follow-up drug, selexipag, being bigger earners than Tracleer.

Clozel co-founded Actelion with his wife, Martine Clozel, and three other executives in 1997 to develop medicines that had been abandoned by Roche Holding AG. Martine Clozel is an Actelion senior vice president, head of drug discovery and chief scientific officer.

Tracleer had revenue of 1.52 billion francs last year, or 89 percent of product sales. Macitentan may garner as much as 1.5 billion francs in peak sales, Andrew Weiss, an analyst at Bank Vontobel in Zurich, wrote in a note today.

Tracleer is losing market share to Gilead Sciences Inc.’s Letairis after U.S. regulators allowed the Foster City, California-based company to remove a reference to the risk of liver damage from its drug’s label in March 2011. Sales of Tracleer fell 7 percent last year, while Letairis rose 22 percent to $293 million. GlaxoSmithKline Plc sells Letairis outside the U.S. under the name Volibris.


Macitentan was well tolerated by patients in the trial, Actelion said. Full results from the study of 742 people will be made public at a medical meeting or in journal articles.

“This is indicative of a best-in-class label,” Berenberg’s Howd said, referring to the macitentan results. “Everyone who says that the label’s not important, look what Letairis did to Tracleer sales. It’s clear the market is sensitive to label claims.”

Tracleer and macitentan are designed to treat pulmonary arterial hypertension, in which the arteries that move blood from the heart to the lungs narrow, making the heart work harder and causing elevated blood pressure.

Over time the heart muscle can weaken and fail, causing death. The drugs block a chemical called endothelin that causes blood vessels to constrict.

Actelion has no plans to do a trial comparing macitentan directly with Tracleer or Letairis, Clozel said.

‘Brighter’ Future

The trend toward preventing premature death was better than expected, said Michael Leuchten, an analyst at Barclays Capital in London who described the result as a “kiss of life” for Actelion. He had predicted macitentan would prevent patients’ disease worsening but thought it may not show a so-called mortality benefit.

“We were not convinced the Seraphin trial results would be robust enough to convince physicians, yet the trend to mortality may prove this to be too conservative,” Leuchten wrote. “We believe the future looks a lot brighter for Actelion than last week.”

Leuchten rates Actelion overweight/neutral and is the top-ranked analyst on the stock over the past 12 months, according to data compiled by Bloomberg.

Macitentan failed in a trial for idiopathic pulmonary fibrosis last year, one of a number of setbacks for Actelion in drug development. Actelion scrapped development of an insomnia pill in January 2011, after abandoning another drug called clazosentan that didn’t help patients with bleeding in the brain. The company also failed in 2010 to have Tracleer approved for wider use.


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