On October 6, 2020 Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, reported that the U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation for TK216, an investigational potentially first-in-class targeted small-molecule inhibitor of the E26 transformation-specific (ETS) family of oncoproteins, for treatment of Ewing sarcoma (Press release, Oncternal Therapeutics, OCT 6, 2020, View Source [SID1234568158]).
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Under the FDA’s rare pediatric disease designation and voucher program, the FDA may grant a priority review voucher to a sponsor who receives a product approval for a "rare pediatric disease," which is defined as a serious or life-threatening disease in which the serious or life-threatening manifestations primarily affect individuals aged from birth to 18 years and which either affects fewer than 200,000 people in the U.S., or affects more than 200,000 people in the U.S. but with no reasonable expectation that the cost of developing and making the drug available in the U.S. will be recovered from U.S. sales. Subject to FDA approval of TK216 for the treatment of Ewing sarcoma, Oncternal may be eligible to receive a priority review voucher if the marketing application submitted for the product satisfies certain additional conditions, including approval no later than September 30, 2022 (unless this statutory sunset provision is modified by Congress). If issued, this voucher may be redeemed to receive priority review for a subsequent marketing application or may be sold or transferred to another sponsor.
"The FDA’s rare pediatric disease designation of TK216 for treatment of Ewing sarcoma, for which Oncternal had previously received FDA’s Orphan Drug and Fast Track designations, underscores the agency’s recognition that Ewing sarcoma is a devastating cancer, with a high unmet medical need," said James Breitmeyer, M.D., Ph.D., President and CEO, Oncternal. "An expansion cohort in the clinical trial of TK216 for patients with relapsed/refractory Ewing sarcoma is currently enrolling, and we expect to present additional interim clinical data from our ongoing Phase 1 clinical trial at a scientific conference in the fourth quarter of 2020."
About Ewing sarcoma
Ewing sarcoma is the second most common bone tumor among children and adolescents. The median age at diagnosis of patients with Ewing sarcoma is 15, and the incidence is about 3 cases per 1 million per year in children under the age of 20 and about 1.3 cases per 1 million overall in the U.S. Nearly all Ewing sarcoma cases are driven by translocations of ETS family oncogenes, including 85-90% of cases driven by the EWS-FLI1 fusion, and approximately 10% by EWS-ERG. Patients diagnosed with metastatic disease have five-year survival rates between 18% and 30%. The prognosis for patients with recurrent Ewing sarcoma is particularly poor, and five-year survival after recurrence is approximately 10 to 15%.
About TK216
TK216 is an investigational, potentially first-in-class, targeted small-molecule inhibitor of the E26 transformation-specific (ETS) family of oncoproteins including fusion proteins. Tumorigenic fusion proteins involving the EWS protein and an ETS protein can be found in most cases of Ewing sarcoma. ETS-related translocations or overexpression are also found in many other tumors such as prostate cancer and acute myeloid leukemia (AML). TK216 was developed based on discoveries in the laboratory of Jeffrey Toretsky, M.D., at Georgetown Lombardi Comprehensive Cancer Center, who discovered inhibitors of EWS-FLI1 using a novel chemical screening assay. In preclinical models, TK216 was observed to bind to EWS-FLI1, blocking the interaction between this fusion protein and other transcriptome proteins such as RNA helicase A, leading to tumor cell apoptosis and inhibiting tumor growth in animal models. The U.S. Food and Drug Administration (FDA) has granted Orphan designation, Fast Track designation, and Rare Pediatric Disease designation to TK216 for the treatment of Ewing sarcoma. TK216 is an investigational medication that has not been approved by the FDA for any indication.