Kevin Gorman, CEO of Neurocrine Biosciences, presents at a Jefferies investor conference in 2013. bloomberg/via getty images
Neurocrine has been on a roll for the past five weeks — right up to Tuesday evening.
First the FDA approved Ingrezza (valbenazine) for tardive dyskinesia in April, their first ever regulatory OK. Then just a few days ago its partner AbbVie came through with promising late-stage data for their endometriosis drug elagolix, outlicensed to them in 2010.
Investigators for the company immediately pinned the blame on what they called ineffective dosing of the drug, vowing to plow ahead with a better design. And they pulled some subset data out of the batch to illustrate their case — that this is a setback, not a lost cause.
The subset data were none too clear in their statement on the trial failure. All the company said was that for “the subset of subjects with pharmaceutical exposure in the appropriate range, there was a substantial reduction in tics (e.g., -11.3 to -13.7 points on the YGTSS (Yale Global Tic Severity Scale)). For subjects with sub-therapeutic exposure, tic reduction was comparable to placebo (e.g., -4.7 to -8.3 points on the YGTSS).”
Subset data are often not substantial enough to provide anything more than an indication. And they can be woefully misleading.
Neurocrine shares dropped 9% on the failure.
“This study showed that we underestimated the INGREZZA dose needed for the pediatric population but also provided us with a clear-cut view into the level of dosing required for future studies,” said Christopher F. O’Brien, chief medical officer of Neurocrine. “We have developed a complete exposure-response model from this study that we believe accurately defines the appropriate dose-range to be tested in the next clinical study that will be started later this year. We were very pleased with the conduct of the T-Force GREEN study in that we were able to identify the appropriate pediatric subjects for the clinical trial, and the investigators demonstrated consistent and appropriate application of the Yale Global Tic Severity Scale.”
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