Drug intelligence / Profile preview

PTC299 + docetaxel

Development stage
Unknown
Lead developer
PTC Therapeutics
Modality
DNA Intercalators/Alkylators → Nucleic Acid-Directed Small Molecules → Small Molecules
Administration
Oral, Intravenous
01

Overview

PTC299 + docetaxel is a **combination investigational therapy** composed of PTC299, a novel, orally administered small molecule inhibitor of dihydroorotate dehydrogenase (DHODH), and docetaxel, a taxane chemotherapy approved for various cancers. PTC299 inhibits de novo pyrimidine nucleotide synthesis, suppressing the pathological production of vascular endothelial growth factor A (VEGFA), which is critical for tumor angiogenesis and growth[1][2][3][5]. Docetaxel destabilizes microtubules, interfering with cell division. The combination has been evaluated in clinical trials for advanced solid tumors, aiming to enhance antitumor activity by simultaneously blocking nucleotide biosynthesis, tumor angiogenesis, and cell proliferation[4].

02

Targets

MicrotubulePfDHODH (Plasmodium falciparum dihydroorotate dehydrogenase)

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