Neuren reports top-line results from Phase 2 trial

Company News

Neuren Pharmaceuticals (ASX:NEU) has reported top-line results for its Phase 2 clinical trial in girls with Rett syndrome aged 5 to 15.

Rett syndrome is a serious and life threatening condition caused by a gene mutation, for which there are currently no approved treatments.

Neuren’s trial was a double-blind, randomized, placebo controlled study that tested three doses of trofinetide compared with placebo in 82 subjects.

The highest dose of trofinetide achieved statistically significant clinical benefit compared with placebo for each of three syndrome-specific efficacy measures, the Rett Syndrome Behaviour Questionnaire (p=0.042), the Clinical Global Impression of Improvement (p=0.029) and the Rett Syndrome Domain Specific Concerns (p=0.025).

"Clinical improvements of 15% to 16% from baseline were observed, which was considered by leading Rett syndrome physicians to be clinically meaningful, particularly in a short duration trial," said the company.

"The improvement increased through to the time that treatment ceased. This suggests that further benefit may be achieved with longer treatment duration. These results provide strong evidence of biological activity of the high dose across multiple symptom areas, indicating the potential for disease modification rather than simply addressing isolated symptoms."

The company said it now intends to discuss with the US FDA plans for a pivotal trial commencing in 2018 using the Rett Syndrome Behaviour Questionnaire (RSBQ) as a primary efficacy measure, supported by the Clinical Global Impression of Improvement (CGI-I) as a key secondary efficacy measure.

In parallel, as previously reported, it will move to complete the necessary chronic toxicity studies and manufacturing scale-up.