Novel Glioblastoma Treatment Granted Orphan Drug Designation

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.

For more information visit Moleculin.com.