BioCryst Pharmaceuticals, Inc has achieved its protocol-specified objective of enrolling 100 late-stage patients (Stage IIB to IVA) in its pivotal study for forodesine in the treatment of cutaneous T-cell lymphoma (CTCL). Top-line data is expected in the second half of 2010. Additionally, BioCryst's exploratory phase-2 study for forodesine in subjects with chronic lymphocytic leukaemia (CLL) is continuing to progress and has enrolled over half of its targeted number of patients.

"Reaching this milestone for the CTCL pivotal study is an important event for the development of forodesine, and will enable a complete analysis of the trial results once protocol-specified patient follow-up has been concluded," said Dr William P Sheridan, chief medical officer at BioCryst. "Also, we are pleased with progress in the CLL phase-2 study and look forward to completing enrolment."

Currently, 143 patients are enrolled in the CTCL study. Eligible patients are those with CTCL of stages IB through IVA who have disease that is persistent, progressive or recurrent during or after treatment with at least three systemic therapies. The study is a multinational, non-randomized, open-label, single-arm trial that is evaluating 200 mg once-daily oral forodesine treatment. The study will examine the rate of objective responses in patients enrolled at sites in North America, Europe and Australia. The study's primary endpoint is objective response rate, defined as either complete response or partial cutaneous response that is sustained for at least 28 days. This trial is being conducted under a Special Protocol Assessment (SPA) agreement negotiated with the US Food and Drug Administration (FDA) and will serve as a basis for a new drug application (NDA) to the FDA.

BioCryst's phase-2 study for forodesine in subjects with CLL has enrolled 15 of the targeted 26 patients, with 12 patients currently still on treatment. The primary purpose of the study is to evaluate the effectiveness and safety of oral forodesine administered as monotherapy at a dose of 200 mg twice-daily in relapsed CLL patients. Previous clinical trial data indicated that forodesine demonstrated clinical activity in CLL patients at a dose of 200 mg once-daily, and was generally safe and well-tolerated. The current trial is testing the benefit and safety of increasing forodesine drug exposure with twice-daily dosing. Top-line study results are expected in the second half 2010.

Forodesine is an orally-available transition-state analog inhibitor of purine nucleoside phosphorylase (PNP), a purine salvage pathway enzyme that is essential for the proliferation of T-cells and B-cells.

BioCryst Pharma designs, optimizes and develops novel small-molecule pharmaceuticals that block key enzymes involved in infectious diseases, cancer and inflammatory diseases.