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Neuren Pharmaceuticals Ltd advancing clinical trial for Rett syndrome

Published: 11:00 25 Aug 2016 AEST

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Neuren Pharmaceuticals Ltd (ASX:NEU) is making steady progress in the Phase 2 clinical trial of its drug candidate trofinetide for the treatment of Rett syndrome.

Rett syndrome is a rare genetic postnatal neurological disorder that almost exclusively affects females.  It affects about 1 in 10,000 live female births.

The company is presently conducting a randomized, double-blind, placebo-controlled clinical trial for girls aged 5 to 15 years with Rett syndrome.

The enrolment of girls into the trial has progressed ahead of schedule, with 49 subjects randomized to date and 5 in screening.

The results of the current paediatric trials, due in the first quarter of 2017, will be an important component of the trofinetide development package, which has received interest from large pharma companies.

Neuren has recognized the potential commercial value this could represent and therefore has determined that partnering in the major markets is best considered once the current trial results are available.

Phase 3 of the Rett syndrome clinical trial is planned in 2018.

Neuren’s largest shareholder, Lang Walker, who currently holds 19% of the company’s shares, has expressed his willingness to invest more to support the trials.

Neuren will seek shareholder approval required under the New Zealand Takeovers Code to give the company the flexibility to take Lang Walker’s holding above 20%.

The company held $8 million in cash as at 30 June 2016.

 

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