"Ewing sarcoma is a rare pediatric cancer, and most patients who present with metastatic disease or fail first-line therapy face a poor prognosis. These young patients are in urgent need of better treatment options," said James Breitmeyer, M.D., Ph.D., Oncternal's President and CEO. "We are pleased to have support from the FDA through the Orphan Drug Designation program as we initiate our first clinical trial of TK216, treating relapsed or refractory Ewing sarcoma patients 12 years and older."
90% of Ewing sarcoma cases occur in patients between the ages of 5 and 25 years, and two thirds are found in those between 10 and 20 years of age. The median patient age is 15 years. Ewing sarcoma is a rare disease, with approximately 600-800 new cases diagnosed annually in the U.S., an incidence rate that has remained fairly constant over the last 30 years at nearly three cases per 1 million people. The 5-year survival rate for patients with localized disease is approximately 75%, but if the tumor has spread or metastasized at the time of diagnosis, the 5-year survival rate drops to 30%.
TK216 is a first-in-class small molecule that inhibits the biological activity of ets-family transcription factor oncoproteins in a variety of tumor types, stopping cancer cell growth and tumor formation. In Ewing sarcoma, it is designed to target a single and well-characterized genetic mutation that causes the disease. TK216 is being developed collaboratively by Georgetown University and Oncternal. The original work took place at Georgetown Lombardi Comprehensive Cancer Center in the lab of Jeffrey A. Toretsky, M.D., who was the inventor of the underlying technology and related intellectual property owned by Georgetown. It is licensed to Oncternal for further development as a potential therapeutic agent for cancer, including treatment of Ewing sarcoma. Oncternal and Georgetown are also planning clinical studies of TK216 in glioblastoma, prostate cancer and leukemia in the next year.
About Oncternal Therapeutics
Oncternal Therapeutics is a clinical-stage oncology company developing first-in-class therapies for both rare and common malignancies. The company is leveraging its scientific and development expertise, as well as academic collaborations, to rapidly advance its two pipeline products focused on targets that are uniquely expressed by cancer cells. Cirmtuzumab is an-anti ROR1 monoclonal antibody, and TK216 is a small molecule that inhibits the biological activity of ets-family transcription factor oncoproteins. For more information, visit http://www.oncternal.com.
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SOURCE Oncternal Therapeutics, Inc.