Neuren Pharmaceuticals (ASX:NEU) should trade substantially higher today after revealing top-line results from its Phase 2 clinical trial in Rett syndrome, which successfully demonstrated clinical benefit from treatment with NNZ-2566.
Neuren intends to submit applications to the US Food and Drug Administration for both Orphan Drug and Breakthrough Therapy designation.
A meeting with the FDA in the first quarter of 2015 is planned to discuss the trial results and the requirements for the further development of NNZ-2566 in Rett syndrome.
There are currently no approved medicines for the treatment of Rett syndrome, which is a severe neurological disorder caused by gene mutations on the X chromosome.
It is a post-natal neurological disorder that occurs almost exclusively in females following apparently normal development for the first six months of life.
Neuren's was the first multi-site, sponsor-led clinical trial in Rett syndrome and was the first trial in an adolescent and adult population.
The results of this trial suggest a promising proof of concept on the pathway to develop a disease-altering treatment for girls and women with Rett syndrome.
The trial was supported by the International Rett Syndrome Foundation.
Primary endpoint was to evaluate the safety and tolerability of each of the two dose levels of NNZ-2556 as compared to placebo.
Evidence of clinical benefit in the group-level analysis required improvement in at least 2 core outcome measures from 2 different efficacy domains, with no clinically significant worsening in all other core endpoints.
This requirement was exceeded at day 26 in the higher dose group, with 3 measures from 3 different efficacy domains achieving the target.
The pre-specified criterion of no clinically significant worsening in the remaining 3 core endpoints was also met.
The lower dose level group exhibited improvement in a number of core outcome measures, however the magnitude of improvement did not meet the targets pre-specified in the analysis plan.
Both dose levels showed trends of increasing effect with the duration of treatment.
Neuren intends to publish and present full analyses of results from the trial in the first half of 2015.
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