Fodosine, is a transition-state analog inhibitor of the target enzyme purine nucleoside phosphorylase (PNP). The drug is currently in a phase IIa trial for patients with T-cell leukemia and a combination IV and oral phase I pharmacokinetic trial in healthy volunteers.
Results of the phase IIa and the phase I pharmacokinetic trial will assist in the design of a planned combination IV and oral phase IIb pivotal clinical trial in patients with T-cell leukemia.
Additionally, Fodosine is currently being studied in a phase I trial with an oral formulation in cutaneous T-cell lymphoma (CTCL) and a phase II trial in chronic lymphocytic leukemia (CLL). BioCryst also plans to initiate a phase I/II trial in B-cell acute lymphoblastic leukemia during 2005.
Fodosine has been granted Orphan Drug status by the FDA for three indications: T-cell non-Hodgkin’s lymphoma, including CTCL; CLL and related leukemias including T-cell prolymphocytic leukemia, adult T-cell leukemia, and hairy cell leukemia; and for treatment of acute lymphoblastic leukemia (ALL).
The FDA has also granted fast track status to the development of Fodosine for the treatment of relapsed or refractory T-cell leukemia. A phase Ib study with BioCryst’s second-generation PNP inhibitor, BCX-4208, was recently initiated and is being conducted with the goal of initiating a phase I/II study in patients with psoriasis in late 2005 or early 2006.