Novel Glioblastoma Treatment Granted Orphan Drug Designation

WP1066 is an immuno-stimulating STAT3 inhibitor.

Moleculin Biotech announced that the Food and Drug Administration (FDA) has granted Orphan Drug Designation to WP1066 for the treatment of glioblastoma.

WP1066 is an immuno-stimulating STAT3 (signal transducers and activators of transcription) inhibitor being investigated as a potential treatment for brain tumors, pancreatic cancer, and acute myeloid leukemia. In preclinical models, the inhibition of phosphorylated STAT3 (p-STAT3) by WP1066 resulted in the direct attack of various tumor cells. In addition, WP1066 has been shown to increase STAT1 activity, which is believed to stimulate the immune system by stimulating T-cell activity.

“We believe that WP1066 represents a new class of drugs which we call ‘Immune/Transduction Modulators’ because it has demonstrated the ability in preclinical testing in animals to both stimulate a natural immune response to tumors and directly attack tumor cells by inhibiting multiple key oncogenic transcription factors, including STAT3, HIF1-α and c-Myc,” noted Walter Klemp, Chairman and CEO of Moleculin.

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