On September 17, 2019 Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, reported that Paul A. Meyers, M.D., Chief, Pediatric Sarcoma Service and Vice Chair for Clinical Affairs of Memorial Sloan Kettering Cancer Center, presented a case study of a patient with Ewing sarcoma who achieved a sustained response following treatment with Oncternal’s investigational product candidate, TK216, in an ongoing Phase 1, first-in-human clinical trial (Press release, Oncternal Therapeutics, SEP 17, 2019, View Source [SID1234539588]). The presentation entitled, "TK216 for the Treatment of Ewing Sarcoma," was given at the Fall Children’s Oncology Group (COG) Meeting.

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Dr. Meyers reported that the patient, who had a history of Ewing sarcoma with pulmonary metastases, had recurrent disease despite multiple courses of chemotherapy, radiation, bevacizumab, pazopanib and surgery. Following two cycles of TK216 therapy given as a single agent, the patient achieved a confirmed objective response, which included resolution of several pulmonary lesions. This response has been sustained and has continued at six months of treatment, with the patient receiving TK216 plus vincristine in subsequent treatment courses. The final remaining residual tumor nodule which was less than one centimeter in diameter was later surgically removed, leading to a surgical complete remission. Treatment with TK216 has been well-tolerated by this patient.

"I am encouraged that this patient has had a sustained, impressive response on the TK216 study," said Dr. Meyers. "There is a high unmet medical need for new options to treat Ewing sarcoma, which is a rare cancer that affects mostly pediatric patients and young adults and has been very challenging to treat effectively."

"We are pleased by the results reported by Dr. Meyers and look forward to examining what we believe could be a recommended dosing regimen of TK216 in a larger number of patients with Ewing sarcoma," said James Breitmeyer, M.D., Ph.D., Oncternal’s President and CEO.

About TK216

TK216 is an investigational, potentially first-in-class small molecule that is designed to inhibit the biological activity of E26 transformation-specific (ETS) transcription factor oncoproteins including fusion proteins. Tumorigenic gene fusions involving ETS factors are frequently found in tumors such as Ewing sarcoma and prostate cancer, and ETS factors are often overexpressed in other tumors such as prostate cancer, and acute myeloid leukemia (AML). TK216 was developed based on discoveries of Jeffrey Toretsky, M.D. and his team at Georgetown University, who found inhibitors of EWS-FLI1 using a novel chemical screening assay. In preclinical models, TK216 binds to EWS-FLI1 and blocks the interaction between ETS family members and RNA helicase A leading to tumor cell apoptosis.

About the Study

TK216 is being evaluated in a Phase 1 clinical study as a single agent and in combination with vincristine in patients with relapsed or refractory Ewing sarcoma, a rare pediatric cancer that has historically been very challenging to treat effectively, particularly for recurrent and metastatic disease. A dose-finding arm of this study is nearing completion, after which Oncternal intends to begin enrolling patients in an expansion cohort of the study to evaluate the clinical response of treatment with TK216 in combination with vincristine, an approved chemotherapy agent. This multi-center study is actively enrolling patients at six clinical trial centers across the U.S. Additional information about the TK216 study may be accessed at ClinicalTrials.gov (NCT02657005).