ONX-0801

ONX-0801

Clinical data
ATC code	none
Identifiers
IUPAC name N-[4-[2-propyn-1-yl[(6S)-4,6,7,8-tetrahydro-2-(hydroxymethyl)-4-oxo-3H-cyclopenta[g]quinazolin-6-yl]amino]benzoyl]-l-γ-glutamyl-D-glutamic acid
CAS Number	501332-69-0 1097638-00-0 (trisodium salt)
PubChem CID	11671880
UNII	Q718FS1C7X
Chemical and physical data
Formula	C33H31N5O10
Molar mass	657.636 g·mol−1
3D model (JSmol)	Interactive image
SMILES C#CCN(c1ccc(C(=O)N[C@@H](CCC(=O)N[C@H](CCC(=O)O)C(=O)O)C(=O)O)cc1)[C@H]4CCc3cc2nc(CO)[nH]c(=O)c2cc34

ONX-0801 (BGC 945) is an experimental drug that has been developed to target ovarian cancer. It is a folate receptor alpha mediated thymidylate synthase inhibitor.

ONX-0801 was originally developed by BTG and the Institute of Cancer Research in the UK, and subsequently licensed to Onyx Pharmaceuticals for clinical development. It is designed to selectively target tumour tissues of certain kinds of cancer. It is poorly absorbed into most cells, but is actively transported by folate receptor alpha (FRα), which is usually only expressed at low levels in the apical membrane of some specialised tissues, but is expressed at much higher levels in some subtypes of ovarian cancer. This causes the drug to accumulate selectively in tumour tissues, while healthy tissues are only exposed to a much lower concentration.

In 2017, it received press coverage following the successful completion of a Phase I clinical trial.