Neuren Pharmaceuticals (ASX:NEU) has secured a funding of A$11.5 million via the placement of shares at $0.062 each, to enable the initiation of key activities for commencing a Phase 3 trial in Rett syndrome.
Neuren is focused on developing new therapies for brain injury, neurodevelopmental and neurodegenerative disorders.
The company presently has a clinical stage molecule, trofinetide in Phase 2 clinical trials.
Trofinetide is being developed both in intravenous and oral formulations for a range of acute and chronic conditions.
The most advanced program is for Rett syndrome, supported by rettsyndrome.org.
The Rett syndrome program has been granted fast track designation by the U.S. FDA and have an orphan drug designation in both the U.S. and the European Union.
Neuren’s placement raising A$11.5 million is anchored by A$10 million from the UK fund Lanstead Capital, with supporting investments from Rettsyndrome.org and Neuren’s directors and management.
Neuren plans to meet with the US FDA Division of Neurology in the third quarter of 2017 to discuss the remaining development for trofinetide to treat Rett syndrome, including the Phase 3 trial design.
In parallel with the FDA interactions, Neuren can now initiate the following key manufacturing and nonclinical activities that are required to be completed before a Phase 3 trial can commence:
- conclude optimisation of the drug substance manufacturing process for commercial supply;
- conclude stability testing and analytical validation of the new to-be-marketed liquid drug formulation; and
- conduct non-clinical toxicity study in a second species, which is required for a Phase 3 trial with longer dosing.
The cash flow for these activities will occur in the second half of 2017 and the first half of 2018.