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.




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, drug discovery.

Moleculin Biotech, Inc.
Drug development

WPD Pharmaceuticals
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, drug development.

Mayo Clinic
Clinical Studies in diffuse intrinsic pontine gliomas (DIPG), a highly aggressive and difficult to treat brain tumor that affects almost exclusively children, drug development.


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.