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Novel azabicyclic series of potent SHP2 allosteric inhibitors.

Title: Novel azabicyclic series of potent SHP2 allosteric inhibitors.
Authors: Sferrazza A; Department of Drug Discovery, IRBM S.p.A., 00071 Pomezia, Rome, Italy. Electronic address: a.sferrazza@irbm.com.; Rossetti I; Department of Drug Discovery, IRBM S.p.A., 00071 Pomezia, Rome, Italy.; Fabbrini D; Department of Drug Discovery, IRBM S.p.A., 00071 Pomezia, Rome, Italy.; Torrente E; Department of Drug Discovery, IRBM S.p.A., 00071 Pomezia, Rome, Italy.; Relitti N; Department of Drug Discovery, IRBM S.p.A., 00071 Pomezia, Rome, Italy.; Ferrigno F; Department of Drug Discovery, IRBM S.p.A., 00071 Pomezia, Rome, Italy.; Fezzardi P; Department of Drug Discovery, IRBM S.p.A., 00071 Pomezia, Rome, Italy.; Iaccarino C; Department of Drug Discovery, IRBM S.p.A., 00071 Pomezia, Rome, Italy.; Bisbocci M; Department of Translational Biology, IRBM S.p.A., 00071 Pomezia, Rome, Italy.; Cellucci A; Department of Drug Discovery, IRBM S.p.A., 00071 Pomezia, Rome, Italy.; Ventre D; Department of Drug Discovery, IRBM S.p.A., 00071 Pomezia, Rome, Italy.; Anzillotti L; Department of Drug Discovery, IRBM S.p.A., 00071 Pomezia, Rome, Italy.; Palombo S; Department of Drug Discovery, IRBM S.p.A., 00071 Pomezia, Rome, Italy.; Nibbio M; Department of Drug Discovery, IRBM S.p.A., 00071 Pomezia, Rome, Italy.; Alli C; Department of Translational Biology, IRBM S.p.A., 00071 Pomezia, Rome, Italy.; Montalbetti C; Department of Drug Discovery, IRBM S.p.A., 00071 Pomezia, Rome, Italy.; Toniatti C; CSO - IRBM S.p.A., 00071 Pomezia, Rome, Italy.; Petrocchi A; Department of Drug Discovery, IRBM S.p.A., 00071 Pomezia, Rome, Italy.
Source: Bioorganic & medicinal chemistry letters [Bioorg Med Chem Lett] 2025 Dec 15; Vol. 129, pp. 130383. Date of Electronic Publication: 2025 Aug 19.
Publication Type: Journal Article
Language: English
Journal Info: Publisher: Elsevier Science Ltd Country of Publication: England NLM ID: 9107377 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1464-3405 (Electronic) Linking ISSN: 0960894X NLM ISO Abbreviation: Bioorg Med Chem Lett Subsets: MEDLINE
Imprint Name(s): Publication: Oxford : Elsevier Science Ltd; Original Publication: Oxford ; New York : Pergamon Press, c1991-
MeSH Terms: Protein Tyrosine Phosphatase, Non-Receptor Type 11*/antagonists & inhibitors ; Protein Tyrosine Phosphatase, Non-Receptor Type 11*/metabolism ; Enzyme Inhibitors*/pharmacology ; Enzyme Inhibitors*/chemistry ; Enzyme Inhibitors*/chemical synthesis ; Antineoplastic Agents*/pharmacology ; Antineoplastic Agents*/chemistry ; Antineoplastic Agents*/chemical synthesis ; Aza Compounds*/chemistry ; Aza Compounds*/pharmacology ; Aza Compounds*/chemical synthesis; Allosteric Regulation/drug effects ; Cell Proliferation/drug effects ; Humans ; Structure-Activity Relationship ; Cell Line, Tumor ; Molecular Structure ; Dose-Response Relationship, Drug ; Drug Screening Assays, Antitumor
Abstract: The non-receptor protein tyrosine phosphatase SHP2, encoded by PTPN11, is a critical component of the RAS/RAF/MEK/ERK signaling pathway, functioning upstream of RAS to promote oncogenic signaling and tumor growth. As part of a drug discovery program aimed at obtaining novel allosteric SHP2 inhibitors, a series of original azabicyclic compounds was identified. Extensive preliminary SAR around the novel bicyclic basic moiety (left-hand side) and the heteroaryl portion (right-hand side) yielded a highly potent series of SHP2 inhibitors with demonstrated cellular potency (pERK inhibition, as downstream marker of MAPK pathway activity) as well as antiproliferative activity in a KYSE-520 cancer cell line. Further optimization of the physicochemical properties and reduction of in vitro off-target liabilities, including hERG inhibition, led to the identification of a unique series of SHP2 inhibitors with strong potential for development in efficacy studies using appropriate animal models.; (Copyright © 2025 Elsevier Ltd. All rights reserved.)
Competing Interests: Declaration of competing interest The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper.
Contributed Indexing: Keywords: Allosteric inhibition; Azabicyclo[4.1.0]heptane; Oncology; Phosphatase; SHP2; pERK
Substance Nomenclature: EC 3.1.3.48 (Protein Tyrosine Phosphatase, Non-Receptor Type 11); 0 (Enzyme Inhibitors); EC 3.1.3.48 (PTPN11 protein, human); 0 (Antineoplastic Agents); 0 (Aza Compounds)
Entry Date(s): Date Created: 20250821 Date Completed: 20250922 Latest Revision: 20250922
Update Code: 20260130
DOI: 10.1016/j.bmcl.2025.130383
PMID: 40840719
Database: MEDLINE

Journal Article