WP 1066


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, Animal Life Science 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-1 and c-Myc, that were considered either undruggable or very challenging drug targets.


Funding for Phase 1 & 2

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.


Orphan Drug

WP1066 has received an Orphan Drug designation in the US for the treatment of glioblastoma, which provides seven years of marketing exclusivity


Additional Indications

MD Anderson
Leading cancer research center in the world

Moleculin Biotech, Inc.
Co-development partner

WPD Pharmaceuticals, Inc.
Co-development partner

Children’s Healthcare of Atlanta, Pediatric
Neuro-Oncology Program, Emory University School of
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.


Phase 1

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.


Subsequent Studies

Glioblastoma(GBM) and melanoma brain metastases. Due to the importance of STAT3 in AML, a subsequent initiation of clinical studies in AML patients is planned in 2019-20.


Additional Indications

Additional indications include pancreatic cancer, lung cancer, renal cancer, melanomas resistant to BRAF inhibitors, breast cancer, and ovarian cancer.