- Neuren Pharmaceuticals is approaching an “exciting fourth quarter of 2019”
- The biopharmaceutical company develops new therapies for rare, neuro-developmental disorders
- A key milestone includes ACADIA planning to commence Phase III of the trofinetide trial for Rett syndrome
- FDA is completing its review of the three applications for Orphan Drug designation for Neuren’s “NNZ-2591” drug candidate
Neuren Pharmaceuticals’ Executive Chairman Richard Treagus commented on exciting times ahead as Q4 2019 approaches.
“Neuren is approaching an exciting fourth quarter of 2019, with some important milestones for the business anticipated,” Richard said.
The biopharmaceutical company develops new therapies for neuro-developmental disorders with high, unmet need.
An important milestone refers to Neuren’s U.S. partner, ACADIA, planning to commence the “LAVENDER” Phase III trial of trofinetide in Rett syndrome.
Neuren completed the Phase II trial of trofinetide for Rett syndrome in January 2017 with successful results reported.
The planned LAVENDER trial will aim to achieve Phase II’s outcome by incorporating twice the treatment duration, an optimised dosing regimen and a higher statistical powering with a significantly larger sample size.
Neuren claims the partnership with ACADIA is working well and proving financially beneficial in preparation for the Phase III program.
ACADIA’s capabilities in the development and commercialisation of central nervous system-related therapies is renowned. Its market cap now stands in excess of US$6 billion.
Further significant news involves the U.S. Food and Drug Administration (FDA) completing its review of the three applications for Orphan Drug designation for Neuren’s “NNZ-2591” drug candidate.
“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,” Richard stated.
An Orphan Drug designation is a status given to medicines developed for rare conditions. This is required as medicines for rare conditions are much less profitable, therefore needing government assistance.
The applications, which Neuren submitted at the end of July, comprise the Phelan-McDermid syndrome, Angelman syndrome and Pitt Hopkins syndrome – all of which are rare genetic conditions.
Neuren will continue to evaluate potential corporate transactions, advised by Torreya, a global investment bank.