Pioneering research leads to drug break-through

Pioneering research leads to drug break-through

Drug discovery research at the University of Auckland to treat a severe neurodevelopmental disorder has successfully passed Phase 2 clinical trials and will now be taken to the next stage.

Drug discovery research at the University of Auckland to treat a severe neurodevelopmental disorder has successfully passed Phase 2 clinical trials and will now be taken to the next stage.

NNZ-2566 is a synthetic analogue of the neuropeptide, GPE, which was originally shown to be neuroprotective by Jian Guan and Peter Gluckman in the Liggins Institute. NZ-2566 was discovered by Distinguished Professor Margaret Brimble, Chair of Organic and Medicinal Chemistry at the University of Auckland and principal investigator at the Maurice Wilkins Centre for Molecular Biodiscovery and developed as a stable form of GPE suitable for drug development. Dr Paul Harris, a Senior Research Fellow at the University’s School of Chemical Sciences, was also a key member of the research team. Dr Jian Guan of the Liggins Institute demonstrated its neuroprotective activities.

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.

“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 says.

Rett syndrome is a relatively rare disorder that almost exclusively affects girls, about 1 in 12,000. After birth, they appear to develop normally until symptoms such as a progressive loss of acquired motor and language skills occur. As the disease progresses, the child can lose use of the hands and the ability to speak.

National coordinator for Rett New Zealand, Gillian Deane, said the results from the trial were highly promising and would bring hope to the dozens of families.
“This is an exciting break-through and we are all pretty emotional about the results,” she says. “This will give families a real boost because at the moment there is no treatment available.”

NNZ-2566 is a synthetic analogue of a naturally-occurring molecule, or neuropeptide, produced by the brain in response to injury. For this reason, NNZ-2566 also has the potential to treat Traumatic Brain Injury (TBI). Neuren is currently running Phase 2 trials in TBI, as well as another neurodevelopmental disorder, Fragile X Syndrome. There is no treatment for either condition currently available.

Professor Brimble and her small team developed molecules that could regulate the levels of inflammatory mediators in the injured brain. After synthesising hundreds of compounds, NNZ-2566 was discovered.

Subsequent research in animal models showed that NNZ-2566 reduced the level of expression of genes associated with inflammation, necrosis and apoptosis, key elements of brain injury, but, more importantly, it reduced the resulting functional deficits. Professor Brimble has provided ongoing analytical and preparative support to Neuren.

Neuren executive chairman Richard Treagus said the trial results had exceeded expectations. “We look forward to discussing with the FDA the remaining requirements to develop NNZ-2566 for the treatment of Rett syndrome,” he said.
Professor Brimble said the discovery of NNZ-2566 and subsequent success in the clinical trials showed the competitive advantages of world-class academic science underpinning commercial science.

Sir Peter Gluckman said it was more than 20 years since the neuroprotective potential of this exciting class of peptides was discovered from excellent discovery science – an important reminder of the long lead time needed to reap the rewards of basic research but that without that investment in basic medical science, new therapies do not emerge.

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