In 2006, Pfizer (PFE), the world's biggest drugmaker, shelved a cholesterol pill it spent $1 billion to develop after a study found the drug, called torcetrapib, raised blood pressure and actually caused more heart attacks. Pfizer's decision left rival Merck (MRK) with a tough call. Merck had also spent years pursuing a new drug that would boost the good cholesterol that ferries fat out of the bloodstream while at the same time reducing bad cholesterol.
Its own treatment, anacetrapib, was still unproven; the company risked throwing good money after bad. "There was a huge debate at every level from the research teams to the top levels of leadership," recalls Luciano Rossetti, Merck's senior vice-president for global scientific strategy.
For six months, Merck's researchers tested and retested anacetrapib. The results came up positive; the drug didn't raise blood pressure in animals. Chief Executive Officer Richard T. Clark decided that Merck would press ahead.
That gamble paid off: On Nov. 17, Merck told thousands of heart doctors gathered in Chicago for the American Heart Assn. annual meeting that new data showed that anacetrapib reduced bad cholesterol by 40 percent while raising good cholesterol by an unprecedented 138 percent in a study of 1,623 patients. "If what we are seeing now is borne out in larger studies, this could be the next big thing that could benefit hundreds of millions of people," said Christopher P. Cannon, the study's lead researcher and a cardiologist at Brigham and Women's Hospital in Boston. "This is totally unprecedented territory."
Pfizer and Merck have been locked in a 25-year battle to dominate the market for cholesterol drugs, which last year topped $35 billion worldwide, according to researcher IMS Health. The standard treatments for high cholesterol, called statins, work by reducing levels of LDL, the bad cholesterol that clogs arteries and causes heart attacks. In 1987, Merck received approval to market the first statin, called Mevacor. Pfizer followed a decade later with Lipitor, a drug in the same family that soon became the world's best-selling medicine, with $11.4 billion in sales last year.
Lipitor has been hard to beat, so pharmaceutical companies in search of new blockbuster drugs have been shifting their focus to areas where breakthroughs may be easier to come by. Even Pfizer: In January the company said it would narrow its drug-development pipeline to 500 projects in six disease areas; cardiovascular wasn't one of them.
That tips the balance in favor of Merck, which boasts a promising pipeline of cardiovascular drugs in late-stage trials involving some 120,000 patients. Anacetrapib is one of four treatments in final testing to prevent heart clogs and blood clots that each have the potential for at least $1 billion in yearly sales by 2016, according to Robert Hazlett, an analyst at BMO Capital Markets. Two of the drugs target cholesterol, and the other two aim to prevent clots. Merck researchers are particularly bullish on vorapaxar, which they believe may achieve the ultimate goal of a blood thinner: to lower the risk of blood clots without causing excessive bleeding. The company plans to seek regulatory approval next year, pending results of a 40,000-patient study. That means the drug could hit the market around the time Bristol-Myers Squibb's (BMY) Plavix, the leading blood thinner with $10 billion in worldwide sales, is set to go off patent in the U.S.
Vorapaxar and anacetrapib each have the potential to exceed $5 billion a year in sales if they aren't sidelined by side effects, figures BMO's Hazlett. Anacetrapib may even accomplish what no other drug in history has been able to do: outsell Lipitor. A rival experimental drug from Switzerland's Roche has not performed as well in studies. Says Hazlett: "Other organizations have notably pulled back, and to their credit, Merck has continued to focus, making good, well-calculated bets."
The bottom line: In the 25-year battle for dominance of the $35 billion cholesterol market, the balance of power is now tipping to Merck.