Boston Business Journal, 7/9/13
Non-Profit Biotech Chief: Mix-up Won't Derail ALS Drug
The head of a local non-profit biotech dedicated to advancing drugs to cure amyotrophic lateral sclerosis (ALS, or Lou Gehrig's disease) says a recent mix-up in a trial by Cytokinetics Inc. won’t derail the drug’s development, but may make it last a little longer.
Cytokinetics (Nasdaq: CYTK) announced Monday it mistakenly gave 58 patients in a 500-patient trial a placebo. They were supposed to be given its drug candidate, tirasemtiv. The company blamed the mistake on a computer error, according to an article on Bloomberg News.
Steve Perrin, CEO and Chief Scientific Officer of the Cambridge, Mass.-usits based non-profit ALS-TDI, said Cytokinetics deserves credit for announcing the error right off the bat, and says it’s lucky that the error didn’t put any patients at risk. He said the company now must decide whether to enroll another 58 patients, or go forward with a slightly smaller trial size.
“It certainly is unfortunate to lose 10 percent of the patients,” he said, but replacing those patients will “extend the timeline of the study.” It will also cost Cytokinetics more money.
The company, which initially lost stock value after the announcement but has since risen almost 5 percent from it’s opening stock price on Monday, said it’s talking to the U.S. Food and Drug Administration about what to do next in the trial.
Perrin says ALS-TDI, meanwhile, is ready to start enrolling patients for a Phase 2a trial of a drug it's developing in conjunction with Novartis called Gilyena, which is already approved for treatment of patients with multiple sclerosis. The organization also has several pre-clinical candidates in development, and hopes to advance one to the human trials before the end of the year.