Metabolic nets BIF grant for pre-clinical studies

Melbourne company Metabolic Pharmaceuticals has received a BIF grant of $234,700 to pursue pre-clinical studies on an orally available iron-chelating compound.

The iron chelator, codenamed MBP0201, is able to bind to iron in the body and help excrete it. Iron overload is a condition that occurs as a result of repeated transfusions in patients with severe anaemia including beta-thalassaemia, as well as in the inherited condition haemochromatosis.

"Our aim is to make an orally active iron chelator," explained Metabolic managing director Chris Belyea.

He said that initial milestones in the project, which was picked up by Metabolic from research at the Sydney Heart Institute in March this year, were achieved. Pre-clinical studies in rodents demonstrated that MBP0201 was orally active and enhanced iron excretion in the animals by the expected amount.

"Our next steps will be gearing up production and conducting toxicity studies. We'll also be doing more efficacy studies in a mouse with iron overload," said Belyea. He said that human clinical trials were not likely to begin before the end of 2003.

Metabolic is also looking for a suitable manufacturing facility for production of the drug, which ultimately would have to be produced in very largely quantities for human use. Belyea explained that it was important that the manufacturing process be inexpensive, as patients would be required to take grams of the drug daily.

He said that Metabolic was talking to an Australian company about manufacturing for the upcoming pre-clinical and toxicity studies, but noted that they would also be looking overseas for GMP manufacturing.

The market for an iron-chelator drug is estimated to be around $US200 million, primarily for the treatment of transfusion induced iron overload. The only drug available on the market is desferoxamine (Desferal, from Novartis), which must be given by injection or infusion.

Belyea said an orally available drug would have a potential market of $US350 million, and would potentially receive orphan drug status from the FDA for treatment of thalassaemia. The market for haemochromatosis is estimated to be worth even more, as the current treatment requires regular phlebotomy.