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WP1066 + radiation is an investigational combination regimen consisting of the small molecule immune/transcription modulator WP1066 and standard-of-care radiation therapy. WP1066, originally developed at MD Anderson Cancer Center and licensed to Moleculin Biotech, is a blood-brain barrier-penetrant inhibitor of phosphorylated STAT3 (p-STAT3), c-Myc, and HIF-1α. It is designed to stimulate the immune response against tumors by inhibiting regulatory T cells (Tregs) and directly inducing tumor cell death. Preclinical studies have demonstrated that combining WP1066 with radiation therapy leads to synergistic anti-tumor activity, increased survival, and the induction of immunological memory in glioma models. An investigator-initiated Phase 2 clinical trial (NU 21C06, NCT05879250) is currently evaluating the combination of WP1066 and radiation therapy in patients with newly diagnosed IDH wild-type, MGMT-unmethylated glioblastoma.
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