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Elraglusib Receives FDA Rare Pediatric Disease Designation for Ewing Sarcoma

• Elraglusib, a GSK-3β inhibitor, has been granted rare pediatric disease designation by the FDA for treating Ewing sarcoma, highlighting the need for new therapies. • The designation supports the ongoing phase 1/2 Actuate-1902 trial, which is evaluating elraglusib's safety and efficacy in pediatric patients with relapsed/refractory malignancies. • Early data from the Actuate-1902 trial shows promising antitumor activity, including durable complete responses in relapsed/refractory Ewing sarcoma patients, reinforcing its potential impact.

The FDA has granted rare pediatric disease designation to elraglusib, a novel glycogen synthase kinase-3 beta (GSK-3β) inhibitor, for the potential treatment of Ewing sarcoma. This designation underscores the urgent need for new therapeutic options for this challenging disease, as stated by Actuate Therapeutics' president and CEO, Daniel Schmitt.
Elraglusib is currently being investigated in the open-label, multicenter, phase 1/2 Actuate-1902 trial (NCT04239092). This trial is evaluating the safety and efficacy of elraglusib in pediatric patients with relapsed or refractory malignancies, including Ewing sarcoma and Ewing sarcoma–related pediatric small round cell sarcomas.
The Actuate-1902 trial includes patients aged 22 years and under with relapsed/refractory Ewing sarcoma treated with elraglusib in combination with topotecan and cyclophosphamide. The study requires patients to have no known curative therapy available and adequate organ and bone marrow function. Patients over 16 must have a Karnofsky performance status of at least 50%, while those under 16 must have a Lansky performance status of at least 50%.
Patients must have recovered from toxicities of prior therapies to be included in the study and must have undergone at least one prior treatment regimen. Elraglusib is being evaluated at 9.3 mg/kg twice per week as a monotherapy, in combination with irinotecan, in combination with irinotecan and temozolomide, and in combination with cyclophosphamide and topotecan.
The primary endpoint of the Actuate-1902 trial is safety, specifically the number of patients with treatment-related adverse events as assessed by CTCAE v5. Topline data from phase 1 of the trial are expected in the second half of 2025.
According to the press release, early clinical data from the ongoing phase 1/2 trial show promising antitumor activity with objective tumor responses, including two ongoing durable complete responses reported in the first six patients treated with relapsed/refractory Ewing sarcoma. This reinforces confidence in elraglusib’s potential impact in this challenging disease setting.
Elraglusib functions by inhibiting GSK-3β in cancer cells, thereby regulating tumor signaling and antitumor immune response. Preclinical studies have demonstrated elraglusib's activity, and clinical evidence suggests antitumor activity across multiple cancer types.
Notably, in September 2024, the FDA granted orphan drug designation to elraglusib for the treatment of soft tissue sarcomas. A phase 2 trial (NCT04906876) is currently evaluating elraglusib in combination with gemcitabine and docetaxel in patients with soft tissue sarcomas.
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Highlighted Clinical Trials

9-ING-41 in Pediatric Patients with Refractory Malignancies.

NCT04239092RecruitingPhase 1
Actuate Therapeutics Inc.
Posted 6/5/2020

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Reference News

[1]
Elraglusib Gains FDA Rare Pediatric Disease Designation in Ewing Sarcoma
targetedonc.com · Nov 12, 2024

Elraglusib, a GSK-3β inhibitor, received FDA's rare pediatric disease designation for treating Ewing sarcoma. The ongoin...

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