Repligen drug for autism misses mark

Repligen's experimental treatment for autism, human synthetic secretin,has failed to achieve its primary endpoint in a Phase II trial of children with moderate-to-severe symptoms of the condition. Shares in the firm fell by around 28% to $1.84 on the day the results were announced (April 3).

The primary goal of the trial was to see how patients scored on the Childhood Autism Rating Scale. Scores for secretin-treated children were not significantly different to those in the placebo group on this measure, according to the firm, although it said it was encouraged by significant improvements in a subset of patients on Clinical Global Impression scores. Repligen said there were no serious adverse side effects seen in either of the patient groups.

Despite the disappointing results, Repligen remains optimistic about the prospects for secretin in autism, and notes that the lessons learned in this trial will allow it to design future trials of the drug "with far greater precision than previously possible."