Repurposing a Potent MDM2 Inhibitor for Leukemia Treatment

Application

VERU-111, also known as sabizabulin, is a potent microtubule inhibitor and, notably, an MDM2 inhibitor. This repurposed compound demonstrates remarkable efficacy in the treatment of Acute Myeloid Leukemia (AML).

Key Benefits

• Unique dual-action functionality as a microtubule and MDM2 inhibitor.
• Successful inhibition of MDM2, an oncogene overexpressed in AML, indicating potential for improved AML treatment outcomes.
• Demonstrated antiproliferative activity with a mean IC50 value of 3.5 nM across 14 MDM2-expressing AML cell lines.
• Also active against melanoma and prostate cancer cell lines with a mean IC50 of 5.2 nM.

Market Summary

Acute Myeloid Leukemia (AML) represents a challenging form of leukemia with low survival rates. The American Cancer Society’s estimates for AML in the United States are approximately 20,800 new cases per year, highlighting the demand for innovative and effective treatments. VERU-111 addresses this need by repurposing a patented compound, offering a unique combination of microtubule and MDM2 inhibition for enhanced AML therapy.

Technical Summary

VERU-111, originally developed as a microtubule inhibitor, has shown significant antiproliferative activity. Emory inventors discovered its inhibitory action against MDM2, a crucial oncogene in AML. This is achieved through protein degradation in the cell. In AML cell culture and xenograft models, VERU-111 displayed therapeutic activity, supporting its potential for AML treatment.

Development Stage

• The technology is at an advanced stage, with demonstrated efficacy in breast cancer and prostate cancer treatment.
• Further development and clinical trials are ongoing for AML application.