Insilico Medicine and Atossa Therapeutics announced the publication of a joint, peer-reviewed study in Nature Scientific Reports identifying (Z)-endoxifen as a promising candidate for treatment of Glioblastoma multiforme (GBM).
Using Insilico’s AI-powered “PandaOmics” platform, the researchers screened over 900 cancer indications based on molecular signatures from endoxifen-treated cells. GBM emerged among the highest-priority cancers for repurposing endoxifen, according to the analysis.
The study uncovered more than 1,400 genes shared between GBM tumors and endoxifen-responsive cells. According to the authors, endoxifen appeared to reverse biological programs linked to uncontrolled cell proliferation, inflammation, metabolic dysregulation and aggressive tumor behavior.
Laboratory experiments supported the computational predictions. In vitro, (Z)-endoxifen significantly suppressed GBM cell proliferation and triggered apoptosis—showing stronger cytotoxic activity than high-dose standard chemotherapy with temozolomide. In combination with other agents, endoxifen’s effect reportedly increased. In vivo studies demonstrated that endoxifen was well tolerated at the doses used.
The companies describe these findings as a strong rationale for further preclinical and possibly clinical development of (Z)-endoxifen in glioblastoma, and a demonstration of how AI-driven approaches can accelerate the identification of new uses for established drugs.
