TK216 has received Orphan Drug Designation from the FDA for the treatment of Ewing sarcoma. It is a first-in-class small molecule inhibiting ets-family transcription factor oncoproteins, which are the main disease drivers in Ewing tumors. A first-in-human phase I trial of TK216 in relapsed or refractory Ewing sarcoma has been initiated.
Ninety percent 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 past 30 years at nearly three cases per one million people.
The 5-year survival rate for patients with localized disease is approximately 75 percent, but if the tumor has spread or metastasized at the time of diagnosis, the 5-year survival rate drops to 30 percent.