Elraglusib Gains FDA Rare Pediatric Disease Designation in Ewing Sarcoma
Elraglusib is undergoing investigation in an open-label, multicenter, phase 1/2 trial for the treatment of patients with Ewing sarcoma.
- Elraglusib has been granted rare pediatric disease designation from the FDA for the treatment of patients with Ewing sarcoma.
- Elraglusib (9-ING-41) is a glycogen synthase kinase-3 beta (GSK-3β) inhibitor.
- The ongoing phase 1/2 Actuate-1902 trial (NCT04239092) is evaluating elraglusib in this patient population.
The FDA has granted the novel GSK-3β inhibitor elraglusib rare pediatric disease designation for the potential treatment of patients with Ewing sarcoma.1
“Receiving rare pediatric disease designation from the FDA underscores the urgent need for new treatment options for patients with Ewing sarcoma and recognizes elraglusib’s transformative potential,” Daniel Schmitt, president and chief executive officer of Actuate Therapeutics, stated in a news release.
Elraglusib is undergoing investigation in the open-label, multicenter, phase 1/2 Actuate-1902 trial.2 Investigators are evaluating the agent’s safety and efficacy in pediatric patients with relapsed/refractory malignancies, including Ewing sarcoma and Ewing sarcoma–related pediatric small round cell sarcomas.
A total of 8 patients aged 22 years and under with relapsed/refractory Ewing sarcoma treated with elraglusib in combination with topotecan and cyclophosphamide were included in the study. Enrollment is open to patients with either measurable or evaluable disease who have no known curative therapy available, and adequate organ and bone marrow function. For those over the age of 16 years, patients are required to have a Karnofsky performance status of at least 50%. Patients under 16 included in the trial must have a Lansky performance status of at least 50.
Photo of sarcoma, magnification 200x, photo under microscope: © Chutima - stock.adobe.com
Patients must also have resolved all toxicities from previous therapies to an acceptable level to be included in the study and are required to have undergone at least 1 prior treatment regimen.
Elraglusib is being evaluated at 9.3 mg/kg twice per week as monotherapy; in combination with irinotecan; in combination with irinotecan and temozolomide (Temodar); and in combination with cyclophosphamide and topotecan.
The primary end point in the study is safety, including the number of patients with treatment-related adverse events as assessed by CTCAE v5.
Topline data from phase 1 of the Actuate-1902 trial are anticipated to be released in the second half of 2025.1
“Early clinical data from [the] ongoing phase 1/2 trial show promising antitumor activity with objective tumor responses, including 2 ongoing durable complete responses [reported] in the first 6 patients treated with relapsed/refractory Ewing sarcoma, reinforcing our confidence in elraglusib’s potential impact in this challenging disease setting. We are committed to advancing elraglusib’s clinical development with the ultimate goal of providing new therapeutic options where current approaches are unsatisfactory,” added Schmitt in the press release.
Elraglusib is a regulator of tumor signaling and antitumor immune response that stops GSK-3β from working in cancer cells. Preclinically, elraglusib has shown activity, and clinical evidence has shown the agent to demonstrate antitumor activity across multiple types of cancers.3
In September 2024, the FDA granted orphan drug designation to the agent for the treatment of patients with soft tissue sarcomas.4 A phase 2 trial (NCT04906876) is currently evaluating elraglusib in combination with gemcitabine and docetaxel in patients with soft tissue sarcomas.
REFERENCES
Actuate announces FDA rare pediatric disease designation granted to elraglusib for treatment of Ewing sarcoma. News release. November 12, 2024. Accessed November 12, 2024. https://tinyurl.com/4s9zdye6
9-ING-41 in pediatric patients with refractory malignancies. ClinicalTrials.gov. Updated November 8, 2024. Accessed November 12, 2024. https://clinicaltrials.gov/study/NCT04239092
Our science. Actuate Therapeutics. Accessed November 12, 2024. https://actuatetherapeutics.com/our-science
Actuate receives FDA orphan drug designation for elraglusib for treatment of soft tissue sarcomas. News release. Actuate Therapeutics, Inc. September 11, 2024. Accessed November 12, 2024. https://tinyurl.com/ns69enfa
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