On September 20, 2019 Neuren Pharmaceuticals (ASX: NEU) Executive Chairman Richard Treagus reported on the outlook for Q4 2019 (Press release, Neuren, SEP 20, 2019, View Source;[email protected] [SID1234539673]):
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"Neuren is approaching an exciting fourth quarter of 2019, with some important milestones for the business anticipated.
Firstly, our North American partner ACADIA plans to commence the "LAVENDER" Phase 3 trial of trofinetide in Rett syndrome. The trial aims to achieve the outcome seen in Neuren’s successful Phase 2 pediatric trial, enhanced by incorporating twice the treatment duration, an optimized dosing regimen and a higher statistical powering associated with a much larger sample size. The partnership with ACADIA is working very well and importantly is delivering in respect of the execution and funding of the many preparatory activities necessary for the Phase 3 program. More broadly, ACADIA continues to demonstrate its capabilities in the development and commercialization of innovative therapies to address unmet needs in central nervous system disorders and its market capitalization now stands in excess of US$6 billion.
Secondly, we expect the US Food and Drug Administration (FDA) to complete its review of the three applications for Orphan Drug designation for NNZ-2591 in each of Phelan-McDermid syndrome, Angelman syndrome and Pitt Hopkins syndrome that Neuren submitted at the end of July. Orphan Drug designation is an important commercial milestone which if granted will add significant value and momentum in respect of our plans to move into clinical trials in 2020.
Our process to evaluate potential corporate transactions, advised by Torreya a global investment bank specializing in life sciences, will continue in the fourth quarter given the importance of these near term milestones."