Metabolic in iron drug deal

By Melissa Trudinger
Thursday, 21 March, 2002

Melbourne biotechnology company Metabolic Pharmaceuticals (ASX:MBP) has entered into licence and research agreements with Sydney's Heart Research Institute (HRI) to develop a drug for the treatment of iron overload diseases.

The collaboration centres around the iron-chelating compounds developed by Prof Des Richardson at HRI. The compounds are derived from a vitamin B-related class of molecules called pyridoxal isonicotinoyl hydrazones.

The compounds are easy to manufacture, and appear to remove excess iron from inside the cells safely. They are also expected to be orally available.

Iron overload is a condition where levels of iron accumulating in cells reach toxic levels. This occurs in severely anaemic patients who receive repeated blood transfusions, and also in some hereditary conditions including haemachromatosis. About 0.5 per cent of the US white population is affected by haemachromatosis, and a similar proportion of sufferers have the condition in Australia.

The single existing treatment for iron overload (desferoxamine, 'Desferal', from Novartis) is expensive and must be injected or infused into the blood. It is only used for treatment of diseases such as beta-thalassaemia and sickle-cell anaemia. Hereditary haemachromatosis is usually treated by periodic phlebotomy (blood letting).

Metabolic Pharmaceuticals will pay HRI milestone fees and a fixed royalty and in return will obtain the exclusive worldwide commercialisation rights.

The market for iron-chelating drugs is potentially $US350 million per year, primarily for the treatment of anaemia-related iron overload.

Metabolic CEO Dr Chris Belyea said if it was suitable for treatment of haemochromatosis, the potential market could be as high as $US1 billion.

"This is an exciting project to expand our portfolio," Belyea said.

"The chief inventor is an international leader, the market is poorly served at present, and we believe these compounds may have a strong competitive advantage over the existing therapies.

"We plan to apply our growing expertise in drug development to take the drug efficiently from the laboratory bench to clinical trials."

Metabolic plans to do preliminary animal studies at HRI with the three lead compounds over the next six months in order to choose which one to continue developing. Full toxicology studies will then be performed to ensure that there are no adverse effects from the chosen compound.

Metabolic Pharmaceuticals currently has a drug for obesity in clinical trials and is also developing drugs for treatment of Type 2 diabetes.

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