Drug intelligence / Profile preview

forodesine

Development stage
Phase 2
Lead developer
Mundipharma International
Modality
Small Molecules
Administration
Oral
01

Overview

Forodesine is a highly potent, orally active small molecule inhibitor of purine nucleoside phosphorylase (PNP). It acts as a transition-state analog, binding to and inhibiting PNP, which leads to the accumulation of deoxyguanosine triphosphate (dGTP) in T and B lymphocytes. This disrupts DNA synthesis by inhibiting ribonucleoside diphosphate reductase, selectively inducing apoptosis in malignant or activated T-cells. Forodesine has demonstrated clinical activity against relapsed/refractory peripheral T-cell lymphoma (PTCL), cutaneous T-cell lymphoma (CTCL), and has been investigated for other hematologic malignancies such as acute lymphoblastic leukemia (ALL) and chronic lymphocytic leukemia (CLL). It was originally discovered by Vern Schramm's laboratory at Albert Einstein College of Medicine and Industrial Research Limited. BioCryst Pharmaceuticals developed the drug, with Mundipharma International responsible for commercialization in several regions[1][2][5][7][9].

Brand names
MundesineFodosine
Other names
forodesine hydrochloride
02

Targets

PNP (Purine nucleoside phosphorylase)

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