A new drug from Auckland-based Neuren Pharmaceuticals for treating chronic neurological disorders including Parkinson's and Alzheimer's aims to tap into a market worth billions of dollars, the company says.
It could take up to seven years for NNZ-2591 to reach commercialisation but with 4.5 million Alzheimer's cases in the United States each year driving a drugs market worth US$2.5 billion ($4 billion) the payback could be huge.
Manufacturing is being ramped up in preparation for formal toxicology tests which, if successful, could lead to human safety trials towards the end of next year.
Chief executive David Clarke said the new drug treated the disorder rather than just the symptoms.
Pre-clinical tests on rats with Parkinson's symptoms showed beneficial effects on ambulatory function - the ability to walk - continued for many weeks after treatment was stopped.
The rats recorded a 94 per cent ambulatory function at week zero before the onset of Parkinson's, which dropped to 16 per cent in week two after development of the symptoms.
After treatment, which was confined to week three, ambulatory function increased considerably and was recorded at 50 per cent after 11 weeks.
Clarke said early indications showed the potential to significantly slow down or stop the further progression of Parkinson's.
"There's clear evidence that it actually gives symptomatic relief but there's also clear evidence that it actually has long-term benefits after the drug's withdrawn," he said.
"In terms of something [already in the market] that actually halts the disease or significantly slows it, there's nothing to our knowledge."
Between one and two people in every 1000 had Parkinson's, rising to one in 100 in people older than 60.
NNZ-2591 had shown no liability for interaction with other drugs and no adverse effects when orally administered to rats at doses more than 15 times above the effective level.
In an experimental model of stroke, the drug was shown to reduce brain damage when given orally, which the ASX-listed company said was an important feature when dealing with long-term treatment. Clarke said the drug - which was Neuren's third lead candidate drug - marked an exciting step for the company.
"NNZ-2591 has all the right characteristics for a promising candidate and has gone through a rigorous selection process over the last 12 months."
It will follow the development programme used for Neuren's brain injury drugs Glypromate and NNZ-2566, which were undergoing human trials.
"We certainly know a lot more about drug development systems and what we can and can't do very quickly and cheaply," Clarke said.
"So 2591 will reap all the benefits of that hard work."
Most of the work to date on NNZ-2591 had been funded by a "vital" grant from the Foundation for Research, Science and Technology.
"If we didn't have that support we probably wouldn't have a third drug because we would have to concentrate everything on the first two."
Drug holds billion-dollar hopes
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