AZD4320

Preclinical pharmacology

AZD4320 is a dual inhibitor of Bcl2 and Bcl-xL with 5nM  and 4nM potency respectively in biochemical assays (FRET) and able to induce intrinsic apoptosis in cells with 6h caspase EC50 values in the nM range (RS4;11 EC50= 10nM; Ri-1 EC50= 15nM, OCI-M1 EC50= 60nM).

AZD4320 monotherapy caused apoptosis preferentially in hematological cell lines with 13/26 AML and 16/27 DLBCL cell lines showing caspase EC50 values <100nM.  In solid tumor cell lines, single agent activity was seen with 9/16 small cell lung cancer cell lines.

In mice xenograft studies, AZD4320 caused tumor regressions following iv dose in subcutaneous and disseminated models of B-ALL and DLBCL.  Induction of cleaved caspase 3 was detected as soon as 6h after iv bolus injection.  AZD4320 also significantly enhanced the antitumor activity of rituximab and ibrutinib in in vivo models of DLBCL.

Suitable for and exclusions

Currently available for preclinical oncology studies only.

Mechanism of Action

BH3 mimetic inhibitor of the anti-apoptotic protein BCL-2 and BCL-xL

Original Therapeutic Area

Oncology

CNS Penetrant

Unknown

Route of administration

IV

Modality

Small Molecule

Additional Information

1. Balachander SB., et al. (2020).

AZD4320, A Dual Inhibitor of Bcl-2 and Bcl-xL, Induces Tumor Regression in Hematologic Cancer Models without Dose-limiting Thrombocytopenia. Clin Cancer Res. 2020 Dec 15;26(24):6535-6549. doi: 10.1158/1078-0432.CCR-20-0863. Epub 2020 Sep 28. PMID: 32988967.

2. Takimoto-Shimomura T., et al. (2018).

Dual targeting of bromodomain-containing 4 by AZD5153 and BCL2 by AZD4320 against B-cell lymphomas concomitantly overexpressing c-MYC and BCL2. Invest New Drugs. 2019 Apr;37(2):210-222. doi: 10.1007/s10637-018-0623-8. Epub 2018 Jun 21. PMID: 29931583.

3. Patterson CM., et al. (2021).

Design and optimisation of dendrimer-conjugated Bcl-2/xL inhibitor, AZD0466, with improved therapeutic index for cancer therapy. Commun Biol. 2021 Jan 25;4(1):112. doi: 10.1038/s42003-020-01631-8. PMID: 33495510; PMCID: PMC7835349.