Indications to Treat Glioblastoma & Melanoma Brain Metastases
WP1066 represents a new class of drugs, which WPD calls “Immune/Transcription Modulators”. WP1066 and related analogs have not only demonstrated the ability to directly induce apoptosis (tumor cell death) but also the ability to stimulate an immune response to tumors allowing T-cells to attack tumor cells. These unique drug properties are a result of WP1066’s ability to inhibit the activated form of STAT3, (p-STAT3). Furthermore, our studies demonstrated that these inhibitory effects are extended to downstream targets and include inhibition of antiapoptotic proteins and VEGF, factors responsible for the formation of new blood vessels (angiogenesis); and most importantly the ability to additionally inhibit two key oncogenic transcription factors, HIF-1α, and c-Myc, that were considered either undruggable or very challenging drug targets.
Currently, in a Phase I trial at MD Anderson for GBM and melanoma metastasized to the brain (WP1066 crosses the blood-brain barrier or BBB); presently in the 3rd cohort of dose escalation evaluating safety and activity; planned surgical expansion to be able to assess tumor tissue directly after administration of WP1066 at the maximum tolerated dose (MTD) for direct confirmation of target inhibition.
Grants / Funding
WP1066 development has received over $8 million and grants and has had $15 million invested in furthering the technology and is expected to receive an additional $3 million in the next 24 months for Phase I and II clinical trials for WP1066 and related analogs.
WP1066 has received an Orphan Drug designation in the US for the treatment of glioblastoma, which provides seven years of marketing exclusivity.
Partners, Affiliations & License
MD Anderson, leading cancer research center in the world
Moleculin Biotech, Inc. – co-development partner
Children’s Healthcare of Atlanta, Pediatric Neuro-Oncology Program, Emory University School of Medicine – Clinical studies of WP1066 in pediatric medulloblastoma, the most common malignant brain tumor in children
Mayo Clinic - Clinical Studies in diffuse intrinsic pontine gliomas (DIPG), a highly aggressive and difficult to treat brain tumor that affects almost exclusively children
WPD owns the exclusive license to 30 countries in Europe and Asia and also Russia.
The expected market size is multiple billions.