IRL chemistry opens new front in global war on gout

US biotech firm BioCryst Pharmaceuticals, Inc recently announced the initiation of a Phase 2 human clinical trial of the drug candidate BCX4208 in patients suffering from gout, a severe form of arthritis affecting tens of millions of people globally and which is particularly common in Maori and pacific island peoples.

BCX4208, co-developed by NZ crown research institute IRL, is a next generation purine nucleoside phosphorylase (PNP) inhibitor, which a recent study shows may have utility in diseases dependent on T-cells, B-cells or uric acid.

The latest trial is designed to determine the effect of different doses of orally administered BCX4208 on uric acid levels in the blood, the build-up of which can lead to the often painful condition known as gout.

BCX4208 differs from existing gout treatments in that it targets an enzyme known as PNP (purine nucleoside phosphorylase). This novel mechanism gives the drug candidate the potential to address unmet medical needs across a broad spectrum of inflammatory diseases in which this enzyme plays a role. It has the potential for once-a day dosing suitable for chronic administration and has been shown to be well tolerated in safety studies. Existing treatments have a range of side effects.

Dr Jacquie Harper, who leads the gout research programme at Malaghan Institute of Medical Research in Wellington, NZ, says: “Gout prevalence is increasing worldwide and is a real problem in New Zealand. A major risk factor for developing gout is excessive uric acid levels in the blood but the choice of uric acid lowering drugs is limited. A new, well-tolerated treatment for controlling uric acid levels would be good news for gout sufferers.”

Dr Richard Furneaux, who heads IRL’s Industrial Biotechnology research, says: “BCX4208 is the second drug candidate we have discovered in collaboration with Professor Vern L Schramm, the Ruth Merns Chair in Biochemistry at the Albert Einstein College of Medicine at Yeshiva University in New York City. Our first is in a pivotal Phase 2 b human cancer trial for Cutaneous T-Cell Lymphoma with results expected in 2010.”

The gout trial is recruiting 120 patients at 12 hospitals in the US and is expected to run till September 2010.

“IRL receives milestone payments and royalties on net sales,” says Dr Furneaux.

“Importantly, we are working towards greater capture of the 100s of millions of dollars of development spend in New Zealand as we expand.”

IRL has other drug candidates moving into development under licence to Pico Pharmaceuticals, Inc.

[View TV3 news item 13 Jan 2010, read stuff.co.nz new story 14 Jan 2010]