Food & Drug Administration Grants Orphan Drug Status for WP1066 in Glioblastoma


The promise of WP1066 for treatment of glioblastoma is recognized by FDA with orphan drug designation.

Medically reviewed by Dr. C.H. Weaver M.D. updated 2/2019

WP1066 represents a new class of drugs called ‘Immune/Transduction Modulators’ because it has demonstrated the ability to both stimulate a natural immune response to tumors and directly attack tumor cells by inhibiting multiple key oncogenic transcription factors, including STAT3, HIF1-α and c-Myc.

The United States Food and Drug Administration has granted Orphan Drug Status for WP1066 in Glioblastoma in Feb 2019. The FDA grants orphan drug designation to drugs that are intended for the treatment of rare diseases that affect fewer than 200,000 people in the U.S. Orphan drug status is intended to facilitate drug development for rare diseases.

Development of WP1066 started with a Phase 1 clinical trial following its discovery by Prof. Waldemar Priebe at The University of Texas MD Anderson Cancer Center. The compound has been shown in animal models to both inhibit an important cell signaling protein STAT3 that is involved in cell growth and proliferation and considered critical to tumor development, while also stimulating a patient’s own immune response. Orphan drug designation signifies WP0166's potential to be beneficial and will help it move through the drug development process faster.

About Glioblastoma

Glioblastoma is an extremely aggressive form of brain cancer. Standard therapy includes surgery to remove as much of the cancer as possible, chemotherapy and/or radiation therapy. Despite treatment, however, overall survival for patients with glioblastoma remains suboptimal, indicating a significant need for improved therapeutic approaches.

Built from the chemical backbone of the active ingredient in propolis, a natural product of honey bees, WP1066 is the first anticancer agent with drug-like properties that consistently inhibits the activated form of STAT3 within cancer cells, a target that has been long-sought because of its broad range of tumor promoting effects. Importantly, activated STAT3 supports the survival and proliferation of tumor cells, evasion of the immune response and metastasis to distant organs, as well as angiogenesis (growth of blood vessels) essential for tumor growth. Activated STAT3 is not only connected with directly supporting tumor activity, but also suppressing the immune system, making this target even more important to cancer therapy.

With the support of extensive preclinical studies demonstrating high antitumor activity and the critically important ability to cross the blood-brain barrier, WP1066 in this Phase 1 clinical trial will focus on treating aggressive brain tumors which all share a grim prognosis. The intent is to eventually treat up to 15 relapsed brain cancer patients over the next six to eight months. Phase 1 clinical trials typically focus on exploring safe and well tolerated doses, as well as evaluating initial signals of effectiveness. Each treatment is completed over three weeks.

How WP1066 Works in Tumor Cells

WP1066 is a small molecule compound that can not only directly kill tumor cells, but also has the ability to overcome the tumor’s ability to evade the natural immune response, which would otherwise be working to eliminate the cancerous activity. This compound is a first in class drug candidate capable of down-regulating the activated form of STAT3, a target that has been long-sought because of its role in supporting the survival and growth of tumor cells.

The compound has been shown to prevent tumor progression and increase survival in a wide range of animal models by directly attacking tumors and blocking the cell signaling by STAT3 that supports tumor development and simultaneously suppressing regulatory T cells (Tregs), which then allows stimulation of an enhanced natural anti-tumor immune response. The compound’s dual functions have been shown to increase survival in a wide range of animal models, which have been documented in more than 50 peer-reviewed articles.