- Neuren Pharmaceuticals’ partner ACADIA, has started the Phase 3 trial for treating Rett syndrome in the U.S.
- ACADIA is fully funding the trial and all other costs, keeping Neuren in a strong financial position
- Rett syndrome is a brain disorder affecting females from infancy with symptoms such as language problems and motor impairment
- Phase 3 will follow from the 2017 Phase 2 trial which saw positive results
- Neuren’s shares are unchanged today, trading for $2.28
Neuren Pharmaceuticals has commenced its phase 3 trial of trofinetide in Rett syndrome in the U.S.
The Phase 3 trial and all associated development costs are fully funded by ACADIA. Neuren has free and complete access to all data for commercial use outside the States.
“The commencement of Phase 3 is a very important milestone for all stakeholders as we work towards achieving an approved treatment for patients and families living with the debilitating effects of Retts syndrome,” Neuren Executive Chairman Richard Treagus said.
Rett syndrome is a brain disorder caused by a gene mutation. It typically becomes apparent in females once they’ve reached six to 18 months of age. Symptoms include problems with language, coordination, and motor impairment.
In January 2017, Neuren conducted a Phase 2 placebo-controlled clinical trial in girls aged five to 15 years with Rett syndrome, and significant clinical statistics were found.
Phase 3 will follow on from the outcome of Phase 2 by incorporating twice the treatment duration, an optimised dosing regimen and a higher statistical powering with much larger sample size.
Neuren claims to be in a strong position as its lead drug is now in Phase 3, its second drug NNZ-2591 was granted Orphan Drug Designation on October 11 and will be moving into clinical trials for Phelan-McDermid syndrome, Pitt Hopkins syndrome and Angelman syndrome, in early 2020.
Neuren’s shares are unchanged today, trading for $2.28 each at 11:48 am AEDT.