Atossa Therapeutics, Insilico Medicine publish study of (Z)-endoxifen in GBM

Atossa Therapeutics (ATOS) and Insilico Medicine, a company dealing in AI-powered drug discovery, announced the publication of a joint study evaluating the potential of (Z)-endoxifen for glioblastoma multiforme, or GBM. The peer-reviewed article, now published in Nature’s Scientific Reports, represents one of the most comprehensive AI-enabled analyses to date exploring whether endoxifen, an active metabolite of tamoxifen with known activity in endocrine-resistant breast cancer, may offer new therapeutic opportunities for one of the deadliest malignant brain tumors in adults. The study aimed to identify new oncology indications with high therapeutic potential for endoxifen, as monotherapy or in combination, by applying Insilico’s AI-powered PandaOmics platform across a wide range of cancer types based on its mechanisms of action. Through this systematic evaluation, GBM emerged as a top candidate for further investigation. By applying multi-omics and AI-enabled methods-including transcriptomic integration, computational modeling, single-cell sequencing, survival analysis, and experimental validation-the study offers mechanistic insights that support further exploration of endoxifen’s potential role in this setting. AI-driven analyses identified more than 1,400 genes shared between GBM tumors and endoxifen-treated cells, revealing strong reversal of biological programs that drive tumor growth and treatment resistance. The multi-omic analyses and experimental findings highlight endoxifen as a promising therapeutic candidate for GBM.