MTX240 is an early drug from Biodexa Pharmaceuticals for gastrointestinal stromal tumour that kills tumour cells by a route that does not depend on the KIT protein, which matters once standard tablets stop working.
MTX240, formerly OPB-171775, was licensed by Biodexa Pharmaceuticals from Otsuka in February 2026. Otsuka assembled a preclinical package and obtained approval for a phase 1 study from Japan's PMDA. Biodexa's first priorities are clinical trial manufacturing, an IND filing and a phase 1b/2a dose-escalation study in GIST, including tumours resistant to tyrosine kinase inhibitors.
A molecular glue that brings together PDE3A and SLFN12, co-expressed in GIST cells, stabilising SLFN12 so that it drives RNase-mediated cell death independently of KIT signalling, as described by Biodexa Pharmaceuticals.
1.MTX240 binds its target with one end and an E3 ubiquitin ligase with the other.
Background: ADC sequencing, Antigen escape (antigen loss, lineage switch), BCG-unresponsive, Castration-resistant prostate cancer (CRPC), Circulating tumour DNA (ctDNA). Also on OnCo: Resistance: how tumours escape each drug class · Lines of therapy.
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Query for this drug: (TITLE:"MTX240" OR ABSTRACT:"MTX240" OR TITLE:"OPB-171775" OR ABSTRACT:"OPB-171775") AND (cancer OR tumor OR tumour OR oncology OR carcinoma OR lymphoma OR leukemia OR leukaemia OR myeloma OR sarcoma OR melanoma OR glioma). Results are unfiltered search hits about MTX240, not a curated reading list.
Shares Biodexa Pharmaceuticals and the tag pipeline.
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