It's shaping up as a rough open Monday for
after the Massachusetts biotech company said its candidate for treatment of autism failed to meet endpoints in a phase III clinical trial.
The company's synthetic human secretin RG1068 couldn't improve social interaction in autism patients in the study, although there were some indications of efficacy in patients with higher cognitive function. Concerns about the science underlying the treatment, an injection given every three weeks,
were highlighted Friday on
subscription sister site,
, by columnist Adam Feuerstein.
The shares were recently changing hands on the Instinet premarket session for $2.65, down $1.51, or 36%, from their 4 p.m. EST Friday close.
The study used two standards for determining efficacy: the Autism Diagnostic Observation Schedule and the Clinical Global Impression of Change. Neither endpoint showed a significant treatment effect in the overall group and in fact, placebos worked better in the phase III trial than they did in phase II.
Repligen said a subset analysis of higher-functioning patients showed a statistically significant improvement using the first standard but not the second. "This finding may indicate that measuring a response to RG1068 is more difficult in patients with lower levels of cognitive function," the company said.