Vertex Faces Setback in Targeted Cancer Drug Program

Cambridge, MA-based Vertex Pharmaceuticals (Nasdaq: VRTX) yesterday announced a potentially serious setback in its program to develop novel cancer drugs aimed at a family of enzymes called the Aurora kinases. Vertex and its partner Merck have one of the most advanced drug candidates (MK-0457) in this category, which is part of an emerging “targeted” approach to cancer therapeutics that experts hope will deliver a wave of highly effective, and hugely profitable, new drugs. (Novartis’ Gleevec, for example, is a targeted cancer drug, aimed at a related enzyme, that rakes in about $2.6 billion annually.)

But Vertex’s hopes of being one of the first out the gate with a blockbuster Aurora-kinase inhibitor dimmed with yesterday’s news: Partner Merck has suspended enrollment in clinical of trials of MK-0457. The company is examining safety data because one patient showed signs of potentially dangerous heart-related side effects. This may sound like extreme caution, but this type of heart problem (called QTc prolongation) has led to many drugs being abandoned, and we are in an era of what some pharma types believe is hypersensitivity to drug-safety problems. If MK-0457 doesn’t get back on track, Vertex and Merck could be left far behind their competitors in this field. Then again, those competitors will now have to examine their data as well, to see if this problem heralds a trend.

This is the second time this month Vertex has gotten potentially bad news about a key program. Earlier, the company found itself unexpectedly sharing the limelight with a possible new competitor (Romark Labs’ Alinia) to its highly-anticipated hepatitis C drug telaprevir.

Hepatitis C is hard to treat, and many people skip treatment altogether because of side effects. Both Vertex and Romark appear to have drugs in hand that could get much better results in many more patients, possibly increasing the market size for hepatitis C drugs substantially. It’s a key race for Vertex, which has spent many years developing telaprevir. Romark, meanwhile, originally developed Alinia to treat parasite-related diarrhea. Its effects against hepatitis C are an unexpected bonus.

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