The drug: Anacetrapib
The disease: Cardiovascular
The developer: Merck
Peak sales potential: Estimates range anywhere from $3 billion to $10 billion, but skepticism about its ultimate success seems to be growing.

Merck's anacetrapib, a CETP inhibitor that promises to boost HDL, or "good," cholesterol, represents the last of three big programs that once fascinated the industry. Roche ($RHHBY) tried the same thing with dalcetrapib--before it blew up--and Pfizer ($PFE) threw in the towel on torcetrapib long ago. There's also some deep-seated skepticism about the whole approach, in which Eli Lilly ($LLY) has also been engaged with its Phase III trial of evacetrapib.

None of the doubts, though, has slowed Merck ($MRK), which continues to invest heavily in the program. The pharma giant launched its Phase III trial for anacetrapib last year, enrolling 20,000 patients to provide conclusive data for regulators. That program is expected to run to 2017, unless Merck experiences the same kind of setback that afflicted its one-time rivals.

The idea here is that blocking CETP, which turns good cholesterol into bad cholesterol, can rebalance the system. If it works, Merck will have a blockbuster that can prevent heart attacks, coronaries and more. Phase III is powered to prove it.

In Merck's DEFINE study, investigators tracked a 138% spike in HDL cholesterol. But the trial team has revised the drop seen in LDL cholesterol from an initial report of 40% to 25% to 30%, opting for what they see as a better measure.

For more:
Big Pharma betting billions in high-stakes hunt for the next Lipitor
New cloud hovers over class of 'good cholesterol' drugs
Merck trumpets loaded late-stage pipeline, big biosimilar R&D plans
Merck ready to spotlight potential mega-blockbuster
Merck cites 'jaw dropping' data on cholesterol blockbuster contender


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