BioCryst Pharmaceuticals Continues Forodesine HCl Program in Chronic Lymphocytic Leukemia

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BioCryst Pharmaceuticals Continues Forodesine HCl Program in Chronic

Lymphocytic Leukemia

Saturday, 27 December 2008

BioCryst Pharmaceuticals today announced interim data from the ongoing

Forodesine HCl Phase 2 program in patients with chronic lymphocytic leukemia

(CLL) and data from a healthy subject pharmacokinetic and pharmacodynamic study.

The CLL study will continue with an amendment to study a new dosing regimen of

oral Forodesine, 200 mg twice-daily.

An interim analysis was conducted on data from an exploratory Phase 2

single-arm, open-label program in patients with CLL whose previous treatment had

failed. While this analysis showed that no partial or complete responses were

observed, five out of 13 patients administered 200 mg of Forodesine HCl

once-daily had substantial reductions in malignant lymphocytes, and at the time

of the analysis, seven patients were still on study. Forodesine HCl was

generally safe and well-tolerated at the 200 mg once-daily dose.

In a parallel, healthy subject, pharmacokinetic and pharmacodynamic study,

BioCryst compared the effect of seven days of 200 mg Forodesine HCl dosed

once-daily with seven days of 200 mg Forodesine HCl dosed twice-daily. The study

demonstrated substantially increased drug exposure and pharmacodynamic effect in

subjects administered Forodesine HCl 200 mg twice-daily. Drug exposure, as

measured by AUC, increased by 63 percent (P<0.001) for twice-daily dosing

compared to once-daily dosing. Serum uric acid levels were reduced at steady

state compared to baseline by 50.0 percent for twice-daily dosing compared to

23.5 percent for once-daily dosing (p<0.001), indicating increased PNP enzyme

inhibition with twice-daily dosing.

" The interim data demonstrates that Forodesine HCl has potential activity

in patients with CLL, " stated Dr. Sheridan, BioCryst's Chief Medical

Officer. " Based on these results and the normal subject pharmacokinetic and

pharmacodynamic study results, we have amended the ongoing Phase 2 study, and

will now administer Forodesine HCl twice-daily to examine the potential benefits

of increased drug exposure. We expect to provide an update on this trial by the

end of 2009. "

" In our experience to date, Forodesine HCl was very well tolerated by

patients in this Phase 2 study. We are pleased with these results and look

forward to further testing to determine the efficacy of Forodesine HCl when

administered twice-daily in patients with CLL, " stated Dr. Asher Chanan-Khan,

Associate Professor of Oncology at Roswell Park.