Orphan Drug Designation Granted for Brain Tumor Drug

Development of “Immune/Transduction Modulator” Gaining Momentum

Moleculin Biotech, Inc. has been granted orphan drug status for WP1066 for the treatment of glioblastoma, the most aggressive form of brain tumor.

Moleculin says that WP1066 represents a new class of drugs, which they refer to as “immune/transduction modulators,” because preclinical testing in animals demonstrated the drug’s ability to 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.

In addition to the glioblastoma trial that was carried out at the University of Texas’ MD Anderson Cancer Center, other investigators have shown interest in conducting clinical trials for pediatric brain tumor treatment and highly resistant tumors including those found in AML and pancreatic cancer. 

Researchers believe that the strong anti-tumor activity in a wide range of animal models is indicative of an important new approach to treating various forms of cancer.

Source: Moleculin, February 5, 2019