BCX4208, a new gout drug, is showing promise. BioCryst Pharmaceuticals Inc, US, developing the drug, reported significant reductions in uric acid blood levels. It also said there were no major safety issues on completion of the first phase of the study it was conducting.
The drug was discovered in a collaboration between Industrial Research Limited (
IRL) and Professor Vern Schramm, Ruth Merns Chair of Biochemistry at the Albert Einstein College of Medicine, New York.
A severe and painful form of arthritis, gout is caused by deposits of uric acid in joints, particularly in the big toe, and affects tens of millions of people globally. In the first part of the randomised, double-blind study, three different doses of BCX4208, a novel, next-generation inhibitor of the enzyme purine nucleoside phosphorylase (PNP), were administered against a placebo to gout patients once a day for 21 days.
All three doses of BCX4208 demonstrated a statistically significant reduction in uric acid blood levels at day 22 compared with the placebo. The drug candidate was generally safe and well-tolerated at the doses evaluated.
"High levels of uric acid in the blood are a necessary precursor to gout," says Dr Richard Furneaux, the leader of IRL's Carbohydrate Chemistry team in which the compound was first synthesised.
"While these results are from early stages in the drug development path, and there is much more testing ahead, these results are exciting for us and particularly relevant to New Zealanders. Unfortunately New Zealand has as much gout in the population as any nation, if not more, with six percent of Kiwi men having the condition. Of particular concern is the genetic predisposition of some individuals to suffer gout, with a 14 percent incidence in Maori men."
Trial results showed reductions in peripheral blood lymphocytes in patients treated with BCX4208, but none of the treatments were stopped due to this reduction. Overall, the frequency of adverse events in each of the BCX4208 treatment groups was comparable with that observed in the placebo group.
Part two of the study, designed to sequentially evaluate the safety and efficacy of up to three higher doses of BCX4208, is now underway.
In addition, on June 1, BioCryst announced it had initiated a Phase 2 study of BCX4208 alone and in combination with allopurinol—a drug approved for the treatment of excess uric acid in blood—in patients with gout.
"We are pleased to initiate this Phase 2 study of BCX4208, a highly potent and selective PNP inhibitor, as it will help to determine whether the inhibition of xanthine oxidase and PNP together has additive or synergistic effects in reducing uric acid levels in patients with gout," says Dr William Sheridan, Chief Medical Officer at BioCryst.
"We expect to complete this study during 2010 and look forward to providing top-line data in the fourth quarter."