Queensland company NuNerve has announced promising early results in a Phase 1 patient cohort of eight people with MND and 20 healthy volunteers who were dosed with its lead compound NUN-004 for six months.
The results were published in Clinical Drug Investigation.
NuNerve founder and co-founder of The University of Queensland’s Queensland Brain Institute Emeritus Professor Perry Bartlett AO FAA said the results paved the way for NuNerve to begin a Phase 2 clinical study in people with MND.
“The Phase 1 trial demonstrated the drug compound, a protein comprised of parts of the EphA4 receptor that can treat damaged motor neurons, was tolerated well with no significant safety issues,” said Professor Bartlett.
“There were highly promising signs of disease stabilisation, and the results compared favourably, in some instances better, than established treatments for MND already approved in overseas markets.
“Importantly, there was positive anecdotal feedback from patients who received the drug, including improved gross and fine motor movement.”
Approximately 140,000 new cases of MND are diagnosed worldwide each year, while some 2,000 Australians are affected at any one time.
Professor Bartlett said NuNerve was seeking vital investment to bring the potential therapy through the next stage of development.
“MND is a fatal motor neurone disease that progressively causes the degeneration of the nerve cells within the brain and spinal cord,” he said.
“Globally, there are only three approved drugs which may slow down the progression of the disease and partially improve quality of life, but there is no treatment that reverses its progression.
“There’s a dire need for better treatments. But it could also have a broader application than as a transformative treatment for MND, with potential therapies for stroke, spinal cord injury and sepsis possible downstream.”
The drug candidate was the subject of more than a decade of research by Professor Bartlett with Emeritus Professor Andrew Boyd from UQ.
This clinical trial has been supported by the Queensland Government, Fight MND and the BioPharmaceuticals Australia Development Fund.
NuNerve is a company spun-out of UQ and has licensed intellectual property from UniQuest, UQ’s commercialisation company, as part of its focus on new technologies to treat or prevent the neurological disease that progressively attacks nerve cells in the brain and spinal cord.