In a stunner, Merck lands positive PhIII for its CETP outcast anacetrapib

Long after many analysts had given Merck’s CETP heart drug anacetrapib up for dead following a slate of expensive burials for the first three drugs in the class, the pharma giant managed to shock the industry this morning with news that their drug came through a 30,000-patient study with convincing data of its effectiveness.

In a brief statement on the topline results, the pharma giant noted that its drug significantly reduced the risk of a coronary event – a composite of coronary death, myocardial infarction, and coronary revascularization – among high-risk patients who had already been through LDL-lowering therapy effectively.

Merck’s shares $MRK jumped 3% in early trading but then quickly flattened as skeptics zeroed in on the pharma giant’s tepid remarks and skeptics quickly began to offer their own assessments on Twitter.

Heart disease R&D has become a much changed field over the last few years, as new PCSK9 cholesterol drugs have proved. Amgen recently tried to grab a market lead with its positive outcomes data, but so far has gained little traction among payers who were unconvinced by the numbers. And significantly, Merck’s short statement notes that the giant player is still considering whether to file for an approval, leaving everyone eager to see the hard data at the European Society of Cardiology meeting August 29. The announcement notably is also free of the usual quote expected from a top R&D exec heralding success and the potential for helping patients.

But what a surprise this is.

“Buyside was NOT expecting this MRK trial to “work” …,” noted Evercore ISI’s Umer Raffat. “My most recent investor survey had 20% odds for this trial.” Raffat, though, also wants to see the hard data to evaluate the commercial potential, especially considering Merck’s hesitancy. And Leerink’s Seamus Fernandez spent some time reading between the lines of Merck’s release, noting that the “cryptic language in the press release suggests a less than definitive risk/reward profile of the drug.”

Merck, however, proved that in this field, it ain’t over until the data arrive — even though the first huge gambles on CETP at Roche, Pfizer and Eli Lilly had all gone bust.

“In baseball you don’t get four strikes,” noted the Cleveland Clinic’s Steve Nissen in 2015. He was ready to call game over after Eli Lilly killed their drug when it failed to pass muster in a futility test. At the time, Bernstein’s Tim Anderson was one of the few analysts still willing to consider that Merck’s decision to go with the biggest Phase III in the bunch still left the door open to success.

But he didn’t have much company.

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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