Neuren Pharmaceuticals (ASX:NEU) has received encouraging results from the Phase 2 clinical trial of its drug candidate trofinetide for the treatment of Rett syndrome.
The company’s share price increased circa 55% since the start of 2017, last trading at $0.087.
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, placebo controlled study that tested three doses of trofinetide compared with placebo in 82 girls with Rett syndrome aged 5 to 15.
The highest dose of trofinetide achieved statistically significant clinical benefits compared with placebo in three syndrome-specific efficacy measures.
Clinical improvements of 15% to 16% from baseline were observed, which is considered by leading Rett syndrome physicians to be clinically meaningful, particularly in a short duration trial.
The improvement increased through to the time that treatment ceased, which 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.
In addition, trofinetide was well tolerated and had a good safety profile in the younger subjects, with no dose-limiting effects observed.
Neuren is planning to discuss with the U.S. Food and Drug Administration (FDA) for a pivotal trial commencing in 2018.