BioCryst begins phase 1/2 trial for Fodosine in patients with leukemia

By E. Janene Geiss

Philadelphia, Dec. 22 - BioCryst Pharmaceuticals, Inc. announced Thursday that it has begun a phase 1/2 clinical trial of Fodosine (forodesine hydrochloride), the company's lead anti-cancer compound, to determine the safety of repeat doses of an intravenous formulation of the drug in patients with B-cell acute lymphoblastic leukemia.

Fodosine is a purine nucleoside phosphorylase (PNP) inhibitor, which functions by blocking the DNA synthesis machinery of the body's T-cells.

This small-molecule drug is being developed for treatment of T-cell malignancies and has received orphan drug status from the FDA for broader indications, including cutaneous T-cell lymphoma, chronic lymphocytic leukemia and acute lymphoblastic leukemia, according to a company news release.

The design of this B-cell acute lymphoblastic leukemia trial was based on data from ex vivo studies on cells from B-cell acute lymphoblastic leukemia patients at M.D. Anderson Cancer Center and by preliminary patient data from Cornell Medical Center, officials said.

The latter study concluded that Fodosine could represent an important breakthrough in the development of a more effective and less toxic treatment for acute lymphoblastic leukemia, officials said.

This multicenter, open-label, repeat-dose study is scheduled to enroll 30 patients and consists of two periods, an initial treatment period and an extended treatment period.

The initial period will last four weeks, during which patients will receive single daily infusions of 80 mg of Fodosine for five consecutive days each week with at least two non-treatment days per week, officials said.

This regimen will be extended by four additional weeks in the extended period if the patient continues to show benefit from the treatment. Additionally, patients who are responding to treatment with Fodosine at the end of the extended period may continue receiving the drug under compassionate-use guidelines.

"This study will allow us to continue the optimization of Fodosine in the treatment of B-cell acute lymphoblastic leukemia," Charles E. Bugg, chairman and chief executive officer of BioCryst, said in the release.

"Additionally, we will be monitoring these patients for evidence of efficacy, which was suggested from the encouraging clinical responses observed in the earlier Cornell trial," Bugg added.

BioCryst, based in Birmingham, Ala., designs, optimizes and develops novel drugs that block key enzymes involved in cancer, cardiovascular diseases, autoimmune diseases and viral infections.

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