NZ firm's breakthrough trialled in US
A type of carbohydrate sugar known as ManNAc has been manufactured by New Zealand Pharmaceuticals since 2003.
The sugar is one that is important to the human body, however it is one that can be difficult for the body to produce naturally.
NZ Pharmaceuticals hold certain Intellectual Property rights to the molecule, which is an intermediate product used in the manufacture of many different drugs and medications.
The pharmaceutical company has been working with crown research institute Industrial Research Limited, to conduct research and development and manufacture the product which it exports to a number of drug companies.
NZ Pharmaceuticals business development manager Selwyn Yorke said recent overseas research showed people with the rare disease Hereditary Inclusion Body Myopathy lack an enzyme to produce Sialic Acid, of which ManNAc is a natural precursor.
Hereditary Inclusion Body Myopathy is a muscle-wasting disorder which usually starts around the age of 20-30 years.
Patients are usually confined to a wheelchair by the time they are in their 30s.
A partnership between NZ Pharmaceuticals and the US National Institutes of Health will see ManNAc produced into a therapeutic treatment that is hoped to reduce or halt the progression of the disease.
ManNAc is one of the first molecules to enter development in the Therapeutics for Rare and Neglected Diseases program run by the National Human Genome Research Institute.
Pre-clinical tests have already been promising with test results in mice showing major improvements in the production of sialic acid.
ManNAc given to mice for several days lead to increased sialylation in kidney tissue and limb muscles.
An application to begin a phase one clinical trial will be filed later this month with the American Food and Drug Administration.
Yorke said the trials would hopefully begin in the US in several months.
"From there, if it is successful we hope it will go to a phase two trial."
Yorke said it would be a long road until the drug was potentially considered safe and effective for use, but the opportunity was "invaluable" for them.
"It would be worth millions of dollars, and we would hope help many thousands of people."