Technology Briefing Vertex plays leapfrog

While genomics identifies ever more specific protein targets associated with disease states, it is important to recall the universality of some molecular mechanisms. Vertex Pharmaceuticals Inc. has put a compound originally intended as a multidrug resistance inhibitor for cancer into the clinic for an entirely different indication, diabetic neuropathy. The move has leapfrogged VRTX past competitors Guilford Pharmaceuticals Inc. and Amgen Inc. in the promising area of neuroimmunophilins.

VRTX (Cambridge, Mass.) last week presented preclinical data suggesting that VX-853 timcodar dimesylate reverses neural dysfunction, but not through the expected FKBP-12 pathway. According to the company, the animal model results show that timcodar and other VRTX compounds have little or no binding affinity for FKBP-12, yet produce the