Roche and PTC’s Spinal Muscular Atrophy clinical drug trials were waylaid by an unexpected tissue specific effect. After the trial had already started (and 100s of millions of dollars already spent), an unexpected activity was found in the eyes (retinas) of monkeys on which the drug was tested.
Of course, this is not an uncommon phenomenon. Some effects of drugs on subjects manifest only after a long period of time being on that drug, so study animals are kept and observed while human trials are initiated. In addition, the species difference between preclinical study animals and humans in clinical trials has proven so sufficiently great that tissue-specific adverse events are almost expected these days. The only question is: Will they doom the clinical trial and lead to drug attrition?
Remarkably, while drug companies do extensive screening for red flags, no systematic approach exists to examine the tissue distribution of drug targets or off-targets, which may be a clue to unexpected tissue-specific side-effects like the eye problem in this story. Gencentrix’s profiler is the only available tool designed to identify exactly that sort of unexpected tissue specific activity, by drawing attention to potential activity in unexpected tissues. Take a walk through the capabilities of the product now here!