Roche appears to have navigated safe passage around a couple of the clinical landmines awaiting any mid-stage HDL study, noting that data from their Phase II trial of dalcetrapib demonstrated that the drug did not raise blood pressure or impair the flow rate of blood vessels. Reporting in Paris, investigators said that the drug spiked levels of high-density cholesterol, the "good" HDL linked to improved cardiovascular outcomes, by 31%. The successful conclusion of the Phase II study sets the stage for closely watched interim results from a late-stage study due next year.
HDL treatments have been under a dark cloud ever since the catastrophic failure of Pfizer's torcetrapib 5 years ago. So the primary focus for investigators and analysts in this Phase II trial was the blood pressure data. Now Roche hopes to put that concern to rest, though it will take data from the much larger Phase III to fully resolve concerns.
"We can say it's safe," principal investigator Thomas Luescher told reporters on Sunday. In this new study, investigators failed to see any significant differences in the frequency of adverse events compared to placebo. But with 476 patients enrolled, the study wasn't big enough to detect small differences which could scuttle the program.
There's quite a varying set of numbers on potential sales for this drug. Some analysts, including Vontobel's Andrew Weiss, believe a clinical hit in this arena would produce a blockbuster capable of earning $5 billion a year. Others have projected a much smaller $203 million in annual revenue by 2015, according to Thomson Reuters Pharma, provided Roche can go on to beat the odds and gain an approval.
Merck is also pushing an HDL drug program and the small French biotech Cerenis is studying its own treatment now in Phase II, which it says could be used to prevent second or third heart attacks.