HSF1 Pathway Inhibitor Clinical Candidate (CCT361814/NXP800) Developed from a Phenotypic Screen as a Potential Treatment for Refractory Ovarian Cancer and Other Malignancies.

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Embargo End Date

Authors

Pasqua, AE
Sharp, SY
Chessum, NEA
Hayes, A
Pellegrino, L
Tucker, MJ
Miah, A
Wilding, B
Evans, LE
Rye, CS
Mok, NY
Liu, M
Henley, AT
Gowan, S
De Billy, E
Te Poele, R
Powers, M
Eccles, SA
Clarke, PA
Raynaud, FI
Workman, P
Jones, K
Cheeseman, MD

Document Type

Journal Article

Date

2023-04-27

Date Accepted

2023-04-27

Abstract

CCT251236 1, a potent chemical probe, was previously developed from a cell-based phenotypic high-throughput screen (HTS) to discover inhibitors of transcription mediated by HSF1, a transcription factor that supports malignancy. Owing to its activity against models of refractory human ovarian cancer, 1 was progressed into lead optimization. The reduction of P-glycoprotein efflux became a focus of early compound optimization; central ring halogen substitution was demonstrated by matched molecular pair analysis to be an effective strategy to mitigate this liability. Further multiparameter optimization led to the design of the clinical candidate, CCT361814/NXP800 22, a potent and orally bioavailable fluorobisamide, which caused tumor regression in a human ovarian adenocarcinoma xenograft model with on-pathway biomarker modulation and a clean in vitro safety profile. Following its favorable dose prediction to human, 22 has now progressed to phase 1 clinical trial as a potential future treatment for refractory ovarian cancer and other malignancies.

Citation

Journal of Medicinal Chemistry, 2023, 66 (8), pp. 5907 - 5936

Source Title

Journal of Medicinal Chemistry

Publisher

AMER CHEMICAL SOC

ISSN

0022-2623

eISSN

1520-4804
1520-4804

Research Team

RNA Bio & Mol Therap
Clinical Pharma & Trials
Signal Trans & Mol Pharma
Medicinal Chemistry 3

Notes