The drug: Darapladib
The disease: Atherosclerosis 
The developer: GlaxoSmithKline
Peak sales potential: Multibillion-dollar market

Back in April when former Human Genome Sciences CEO Tom Watkins was scoffing at GlaxoSmithKline's lowball offer for the company, he cited darapladib as one big reason for turning down the bid. The cardiovascular drug--controlled by GSK ($GSK) but still promising royalties and a co-promotion deal for HGS--had the potential to earn billions, Watkins claimed. But after failing to live up to the same promises for Benlysta in the time allotted, the argument failed to significantly improve the bid on the table or inspire a white knight.

The anti-atherosclerosis drug is being tested in tens of thousands of patients. The big idea here is that by inhibiting the Lp-PLA2 enzyme, the treatment could significantly reduce the rate of strokes and heart attacks. And the developers clearly have a huge market in mind. 

This drug actually failed its midstage study, unable to demonstrate any ability to rehabilitate plaque-lined arteries. But investigators pointed to intriguing data indicating that it had a tonic effect on what is called the necrotic core. Patients with atherosclerosis were vulnerable to seeing the necrotic core rupture, causing possibly fatal blood clots. And darapladib kept the core stable, apparently, in the 330-patient study.

So despite the midstage failure, GSK--which used HGS's technology to find the drug--pushed ahead with a massive 23,000-patient study to put its theory to the test.

If it works properly, it could be huge in a world with a huge population of aging people suffering from hardening of the arteries. If not, it won't be worth a cent.

For more:
Big Pharma betting billions in high-stakes hunt for the next Lipitor
GSK closes the deal on Human Genome Sciences for $3B
Study highlights potential benefits of GSK heart drug


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