Neuren Pharmaceuticals Ltd (ASX:NEU) has presented its clinical-stage drug candidate trofinetide for treating Rett syndrome at a research symposium, held near Chicago, U.S.A.
The company is making progress in its New Drug Application with the NDA for the drug compound.
Neuren’s Phase 2 clinical trial in subjects aged 5 to 15 with Rett syndrome is on track to be completed by the end of 2016.
Rett syndrome is a rare genetic postnatal neurological disorder that almost exclusively affects females.
It affects about 1 in 10,000 live female births in all geographies, and across all races and ethnicities.
Early results from trofinetide trials provide evidence of its effectiveness as a potentially viable treatment for the core signs and symptoms of Rett syndrome.
Additionally, Neuren has agreed with the FDA, the construct of the efficacy measure derived from the Motor Behaviour Assessment that will be the primary endpoint in a Phase 3 trial.
Meanwhile, the optimization of the manufacturing process at commercial scale by third party contract manufacturers is progressing to enable drug product from the commercial process to be used in the Phase 3 trial.
Neuren had more than $10 million in cash at the end of the March 2016 quarter.
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