New Clinical Data Available on TK216 for Ewing Sarcoma

Recently, biopharmaceutical company Oncternal Therapeutics (“Oncternal”) announced that it has additional clinical data available from its Phase 1 clinical trial. Through the trial, researchers are analyzing an investigational therapy, TK216, for the treatment of patients with relapsed/refractory Ewing sarcoma. Oncternal will present this data during the Connective Tissue Oncology Society (CTOS) 2020 Annual Meeting, which is being held virtually. Data will be presented on November 12 starting at 11:30am, under Abstract 3464969. You may also find the presentation under “Events and Presentations.


While TK216 has not yet been approved for use, it is currently being assessed in a Phase 1 clinical trial for Ewing sarcoma. During the trial, researchers are evaluating TK216 as a single use therapy, as well as a therapy in conjunction with vincristine. This treatment inhibits E26 transformation-specific oncoproteins; these include fusion proteins, which are often found in Ewing sarcoma. By inhibiting these proteins, TK216 prevents tumor cell growth. Currently, TK216 received Orphan Drug, Fast Track, and Rare Pediatric Disease designations.

Throughout the Phase 1 trial, researchers have been analyzing the safety, efficacy, tolerability, and pharmacokinetics of TK216. Many participants have received up to 8 prior treatments. Researchers found TK216 to be well-tolerated and relatively safe. Side effects included:

  • Nausea
  • Fatigue
  • Appetite loss
  • Fever
  • Alopecia
  • Low production of blood cells

On average, patients experience no disease progression for an average of 1.8 months. Additionally, TK216 seemed to reduce tumor growth and prompt more of a response.

Ewing Sarcoma

Ewing sarcoma is part of the Ewing family of tumors, cancers which grow in the bone and surrounding soft tissues. Other members of the Ewing family of tumors include extraosseous Ewing tumors and peripheral primitive neuroectodermal tumors. EWSR1 gene mutations may play a role in Ewing sarcoma development. Much like other members of this tumor family, this rare cancer typically grows in the bones, particularly around the arms, spine, legs, ribs, and pelvis. However, tumors can be located in different areas throughout the body. Being Caucasian, white, or a teenager are risk factors to developing a Ewing tumor. On average, patients are 15 years old when diagnosed with Ewing sarcoma. The 5-year survival rate for patients with recurrent Ewing sarcoma is only 10-15%.

Symptoms include:

  • Pain near the tumor site
  • Lumps or swelling
  • Unintended weight loss
  • Fatigue
  • Fever

Learn more about the Ewing family of tumors.

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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