Moleculin Biotech’s (NASDAQ:MBRX) WP1066 received FDA rare pediatric disease indication for the treatment of diffuse intrinsic pontine glioma (DIPG), medulloblastoma and atypical teratoid rhabdoid tumor.
WP1066 is an immune/transcription modulator designed to inhibit p-STAT3 and other oncogenic transcription factors, while stimulating a natural immune response.
In October 2020, the company reported that its Phase 1 DIPG trial was proceeding to the second dose level, and that one of the first three patients responded to the treatment with both clinical improvement and radiologic reduction of tumor size.
“The early activity we are seeing in WP1066 is both unexpected and encouraging,” Walter Klemp, Moleculin’s chairman and CEO, said in a statement.
“The approval of these three rare pediatric disease designations is a reminder of just how important our efforts are to potentially help children with brain tumors.”
