Discovered by Professor Brimble, Chair of Organic and Medicinal Chemistry at the University of Auckland and principal investigator at the Maurice Wilkins Centre for Molecular Biodiscovery (and a panelist at the recent MiNDFOOD International Women’s Day workshop), NNZ-2566 was originally found to be neuroprotective by Jian Guan and Peter Gluckman in the Liggins Institute.
A neurodevelopmental disorder that affects girls almost exclusively, Ret Syndrome is characterised by normal early growth and development followed by a slowing of development, loss of purposeful use of the hands, slowed brain and head growth, problems with walking, seizures, and intellectual disability.
Biopharmaceutical company Neuren Pharmaceuticals (ASX: NEU), which carried out the Phase 2 trial and is behind efforts to develop a treatment for Rett syndrome, announced it will now meet with the US Food and Drug Administration (FDA) to discuss the remaining development requirements.
If NNZ-2566 passes the remaining hurdles, it will be the first drug discovered in New Zealand to be registered with the FDA and the first in this country to be discovered by a woman scientist.
A synthetic analogue of a naturally-occurring molecule, or neuropeptide, produced by the brain in response to injury, NNZ-2566 also has the potential to treat Traumatic Brain Injury. Currently, there is no treatment available for either.
“While this is enormously exciting for me personally, the real satisfaction is that this gives families affected by Rett Syndrome real hope that a treatment is on the horizon,” Professor Brimble, who was also the first New Zealander to receive the L’Oreal-UNESCO Women in Science award and the second woman to receive the Rutherford medal, said.
National coordinator for Rett New Zealand, Gillian Deane, is equally optimistic about the breakthrough.
“This is an exciting break-through and we are all pretty emotional about the results,” she said. “This will give families a real boost because at the moment there is no treatment available.”
Professor Brimble said the discovery of NNZ-2566 and success in the clinical trials showed the competitive advantages of world-class academic science underpinning commercial science.