Shares in Neuren Pharmaceuticals Limited
(ASX:NEU) spiked after releasing top-line results from its US Phase 2 clinical trial in Rett syndrome.
The biopharmaceutical company says the ground-breaking trial successfully demonstrated clinical benefit from treatment with NNZ-2566.
Neuren now intends to submit applications to the US Food and Drug Administration (FDA) for both Orphan Drug and Breakthrough Therapy designation.
The company expects to meet with the FDA early next year to discuss the results and requirements for further development of NNZ-2566 in Rett syndrome.
Experts say the trial’s outcome is very promising and opens the possibility of successful treatment of adults with Rett syndrome as well as early interventions modifying the course of the disease.
Shares in Neuren Pharmaceuticals spiked 53.85 per cent following the news to close at $0.12.
Neuren Pharmaceuticals reported a net loss of $4.67 million in the first half of the current 2014 calendar year.
