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Record W4410426610 · doi:10.1021/acs.jmedchem.5c00529

Discovery of RP-1664: A First-in-Class Orally Bioavailable, Selective PLK4 Inhibitor

2025· article· en· W4410426610 on OpenAlexafffund
Frédéric Vallée, Matias Casás‐Selves, Monica Bubenik, Martin Duplessis, Boubacar Sow, Catalina Suarez, Bruno Sangiorgi, Li Li, Marc L. Hyer, Robert Papp, Marie-Ève Leclaire, Alexander L. Perryman, Bingcan Liu, Simon Surprenant, Philippe Mochirian, Victor P.T. Pau, Zdenka Maderova, P. Mäder, Shou Yun Yin, Elliot Goodfellow, Anne Roulston, Rino Stocco, Claude Godbout, Prasamit Baruah, Alexanne Bonneau-Fortin, Joseph D. Schonhoft, Parham Nejad, David W. Norman, Vouy Linh Truong, Sheldon N. Crane, Mohamed A. Attia, Daniel Y.L. Mao, Frank Sicheri, Cathleen Marshall, Michal Zimmermann, David Bendahan, Michel Gallant, W. Cameron Black

Bibliographic record

VenueJournal of Medicinal Chemistry · 2025
Typearticle
Languageen
FieldBiochemistry, Genetics and Molecular Biology
TopicUbiquitin and proteasome pathways
Canadian institutionsUniversity of TorontoLunenfeld-Tanenbaum Research Institute
FundersCanadian Institutes of Health Research
KeywordsChemistryBioavailabilityPharmacologyStereochemistry

Abstract

fetched live from OpenAlex

PLK4 is a cell cycle-regulated kinase important for the biogenesis of centrioles and is known to be synthetically lethal with TRIM37 gene amplification. Previous attempts to inhibit PLK4 have been hampered by selectivity or ADME liabilities. The known inhibitor Centrinone B, while potent and selective, is metabolically unstable and lacks oral bioavailability. Assisted by structure-based drug design (SBDD), dramatic improvements in potency, selectivity and ADME properties were made to this structure, resulting in the identification of RP-1664, a potent inhibitor of PLK4 with an excellent pharmacokinetic profile in preclinical species. Kinome profiling demonstrated exquisite selectivity over related kinases, including AURKA/B and PLK1. RP-1664 disrupts centriole biogenesis in cancer cells, modulates pharmacodynamic readouts of PLK4 activity in xenograft tumor tissues, and is efficacious in multiple TRIM37 -amplified xenograft models. This first-in-class clinical candidate is currently being evaluated in Phase 1 clinical trials (NCT06232408) for treatment of advanced solid tumors.

Fetched live from OpenAlex and de-inverted. Abstracts are not stored in this database: the inverted indexes are 8.6 GB of the frame’s 9.3 GB of text, and the host has 13 GB free.

How this classification was reachedexpand

Full frame machine prediction

Teacher imitation

Not calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.

metaresearch head score (Codex)0.000
metaresearch head score (Gemma)0.000
Version: metacan-v3-hybrid-931329e0061cValidation status: machine_predicted_unvalidated
Candidate categoriesnone
Consensus categoriesnone
DomainCandidate signal: none · Consensus signal: none
Study designCandidate signal: Bench or experimental · Consensus signal: Bench or experimental
GenreCandidate signal: Empirical · Consensus signal: Empirical
Teacher disagreement score0.002
Threshold uncertainty score0.008

Distilled classifier scores by category (both heads)

CategoryCodexGemma
Metaresearch0.0000.000
Meta-epidemiology (narrow)0.0010.000
Meta-epidemiology (broad)0.0000.000
Bibliometrics0.0000.000
Science and technology studies0.0000.000
Scholarly communication0.0010.000
Open science0.0010.000
Research integrity0.0000.001
Insufficient payload (model declined to judge)0.0020.001

Machine scores (provisional)

The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.

Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.

Opus teacher head0.007
GPT teacher head0.242
Teacher spread0.235 · how far apart the two teachers sit on this one work
Validation statusscore_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from it

Classification

machine, unvalidated

Machine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.

The models applied no category: nothing in the taxonomy fit this work.
Study designBench or experimental
Domainnot available
GenreEmpirical

How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".

Quick stats

Citations9
Published2025
Admission routes2
Has abstractyes

Explore more

Same venueJournal of Medicinal ChemistrySame topicUbiquitin and proteasome pathwaysFrench-language works237,207