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.


  • 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


  • MD Anderson, leading cancer research center in the world
  • MoleculinBiotech, 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. 

R & D