Simply Tragic: Pfizer's Torcetrapib Cholesterol Drug Halted

Read the press release announcing the halt of all torcetrapib clinical development. According to Pfizer spokesman Paul Fitzhenry, as reported by the AP, an interim DMC safety review discovered 60% more deaths (82 vs. 51) among the combination atorvastatin-torcetrapib arm as compared with the atorvastatin arm in the ca. 15,000-subject ILLUMINATE trial. Based on this “imbalance” the DMC recommended the study be stopped. Pfizer took this recommendation and further decided to stop all torcetrapib clinical studies.

Torcetrapib, a cholesterol-ester transfer protein [CETP] inhibitor, was one of the most eagerly anticipated drugs in development. It was also the costliest to develop; Pfizer reportedly was planning to spend over $800 million on its Phase 3 development alone. It was a drug many analysts, including this one, deemed crucial to Pfizer’s growth prospects after Lipitor loses US market exclusivity in early 2010.

A timeline of torcetrapib milestones is found in this 2005 Pfizer press release, which announced expansion of the Irish plant Pfizer was using to manufacture the torcetrapib/atorvastatin tablets. The drug had recently suffered a non-fatal setback when it was discovered that it raised blood pressure more than thought following Phase 2 studies.

I am neither a Pfizer investor, nor do I count Pfizer among consulting clients, yet I am terribly disappointed in this outcome. Besides being a stark demonstration of our industry’s inability to channel late-stage investments with more predictability than could be had by relying on a coin toss, the torcetrapib failure is a hard-lost opportunity to lessen the world’s ever-mounting burden of atherosclerotic cardiovascular disease [CVD]. Whatever good comes from the torcetrapib “case” let’s not lose sight of this loss; it’s tragic in its proportions.

As for the potential good emanating from the news, surely the loss of such a large investment in time and money will serve as a reminder of the industry’s urgent needs to improve R&D decision-making and to take lessons from the financial-investment industry and practice sound product-portfolio risk management. Eight hundred million dollar Phase 3 bets that don’t pay off will ruin this industry more assuredly than government-mandated price controls, counterfeit products, and product “re-importation” combined could.

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