Neuren on track with "watershed" clinical trials

Neuren Pharmaceuticals (ASX:NEU) is well on track with clinical trials of treatment candidate NNZ-2566 in four neurological conditions.

The company has provided an update to its clinical trial programs, revealing that recruitment is underway for separate trials in patients with Fragile X syndrome, Rett syndrome and moderate to severe traumatic brain injury.

Fragile X syndrome is a genetic syndrome that is the most widespread single-gene cause of autism in males. NNZ-2566 has both orphan drug designation and fast track designation in the syndrome from the US FDA.

A phase II double-blind, placebo-controlled trial is being led by the Rush University Medical Center in Chicago. Subjects are being enrolled across six sites, and the 60 patients required to complete the trial are expected to be recruited by the end of the year.

Rett syndrome is a neurodevelopmental disorder found almost exclusively in females. Neuren initiated a phase II, double-blind, placebo-controlled trial in Rett syndrome in April last year. The first group of girls has already been dosed.

In all, 36 patients have been enrolled in the trial out of the 48 required, and 22 have completed the entire trial. Neuren expects recruitment to be completed in 1H14 and top-line results to be available in the second half.

Neuren also revealed that 131 subjects with moderate to severe traumatic brain injury have been recruited in the INTREPID trial, which is being conducted by the company in collaboration with the US Army. This represents just over half of the 260-subject target.

The company expects recruitment to complete by the end of the year, with top-line results to follow in 1H15.

Finally, the company revealed that preparations are continuing for a phase II trial of the oral-dose form of NNZ-2566 in concussion. This trial, too, will be conducted in collaboration with the US Army and will involve 132 subjects at the Womack Army Medical Centre in North Carolina.

The trial is expected to commence in 1H14, complete recruitment in 1H15 and report results in 2H15.

In a research note, Bell Potter Securities said 2014 is likely to be “a watershed year” for Neuren.

“The first of the Phase II results from the Rett syndrome trial [are] expected to be released in the second half of the year. Positive results from this trial will be a crucial de-risking event for the company and likely to lead to a significant re-rating of the stock,” the firm said.

Neuren Pharmaceuticals (ASX:NEU) shares were trading 5% higher at $0.105 as of around 1 pm on Wednesday.