Opening of Phase 1b Expansion Cohort of Clinical Trial of TK216

SAN DIEGO--(BUSINESS WIRE)--Dec. 11, 2019-- Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, today announced that it has opened for enrollment a Phase 1b expansion cohort of its ongoing clinical trial evaluating TK216, a first-in-class, targeted, investigational small-molecule inhibitor of the E26 transformation-specific (ETS) family of oncoproteins, in patients with relapsed or refractory Ewing sarcoma.

The decision to open an expansion cohort was based on favorable interim results from the dose-finding cohort of the trial, including a deep and sustained clinical response reported for one of the patients treated at the current, highest exposure dose regimen, who is without evidence of Ewing sarcoma after more than eight months on study and has tolerated treatments with TK216, alone and in combination with vincristine, well. TK216 has been generally well tolerated in this trial. Dose limiting toxicities consisted of transient and manageable myelosuppression, primarily neutropenia.

The expansion cohort will further evaluate the recommended Phase 2 dose regimen of TK216 (200 mg/m2/day for 14 days) in combination with vincristine and is anticipated to enroll approximately 18 patients with relapsed or refractory Ewing sarcoma.

“We are encouraged by the initial clinical activity demonstrated by TK216 in the dose-finding portion of this clinical trial in patients with relapsed/refractory Ewing sarcoma and look forward to further evaluating the recommended Phase 2 dose regimen of TK216 in a larger number of patients with this devastating disease,” said James Breitmeyer, M.D., Ph.D., Oncternal’s President and CEO. “This first-in-class targeted investigational agent may also be applicable in other malignancies driven by ETS alterations including acute myeloid leukemia (AML) and prostate cancer, which we continue to explore in preclinical studies.” Full Press Release and Additional Information

The 35th Annual Childrens Cancer Foundation Gala was held Saturday, November 2nd, 2019 where more than 500 individuals joined CCF and Mark Viviano, as emcee, and recognized Extraordinary Families – children and their families who have endured, fought, overcome, grieved and persevered in spite of this life-changing diagnosis. Two amazing young people – Darren Leonard and Grace Callwood – were featured with their families in a video played during the program. Many other children that evening were presented with medals, recognizing the battles they have overcome with having a diagnosis of cancer.

CCF also granted awards to nine area researchers, including the 4th Annual Fore 3 Fellowship Award to Dr. Jeffrey Toretsky, MD, and to several programs and facilities making significant differences in the lives of these children, totalling nearly $1 million.

Case Study of TK216 in Ewing Sarcoma: Achieved Sustained Response

Sep. 17, 2019-- Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, today announced 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. The presentation entitled, “TK216 for the Treatment of Ewing Sarcoma,” was given at the Fall Children’s Oncology Group (COG) Meeting.

Returning summer researcher, Sydney Parks, received a 2019 St. Baldrick's Summer Fellow Award aiding to fund a summer research project. Through the St. Baldrick’s Summer Fellowship, Sydney began cloning common EWS-FLI1 (EF) mutants into a tetracycline-inducible plasmid previously generated by Garrett Graham. In the future, a library of such plasmids can be expressed in human mesenchymal stem cells (hMSCs) to investigate hMSC transformation into ES-like cells following EF expression. Sydney is currently a senior at Rice University studying Biochemistry and Cell Biology.

This grant funds an undergraduate student to complete work in pediatric oncology research for the summer. The experience may encourage them to choose childhood cancer research as a specialty. Ewing Sarcoma (ES) is an aggressive pediatric cancer.