Actuate reports complete responses in refractory Ewing sarcoma trial

Published 17/07/2025, 13:52
Actuate reports complete responses in refractory Ewing sarcoma trial

CHICAGO/FORT WORTH - Actuate Therapeutics, Inc. (NASDAQ:ACTU), a clinical-stage biopharmaceutical company with a market capitalization of $122 million, announced Thursday the completion of the Phase 1 portion of its clinical study evaluating elraglusib in pediatric patients with refractory malignancies, with notable responses observed in Ewing sarcoma patients. InvestingPro data shows the stock trading at $6.23, with analyst price targets ranging from $20 to $35.

The trial enrolled ten patients with relapsed/refractory Ewing sarcoma treated with elraglusib in combination with topotecan and cyclophosphamide. Two patients achieved complete responses, while two others maintained stable disease. Additionally, one patient with desmoplastic small-round-cell tumor (DSRCT) showed a partial response.

Following these results, Actuate plans to advance to a Phase 2 study in children, adolescents, and adults with relapsed/refractory Ewing sarcoma, expected to begin in 2026, subject to funding availability.

"To observe complete responses in this setting is highly unexpected," said Dr. Andrew Mazar, Actuate’s Scientific Co-founder and Chief Operating Officer, according to the company’s press release.

Ewing sarcoma is an aggressive form of cancer primarily affecting children and young adults, with approximately 25% of patients presenting with metastatic disease at diagnosis. The condition currently lacks approved targeted therapies.

Elraglusib is a glycogen synthase kinase-3 beta (GSK-3β) inhibitor designed to target molecular pathways involved in tumor growth and resistance to conventional cancer treatments.

The open-label, multicenter Phase 1/2 trial (NCT 04239092) evaluated elraglusib alone or in combination with various chemotherapy regimens. The company is now collaborating with key opinion leaders to design the upcoming Phase 2 study to align with patient needs and regulatory expectations.

In other recent news, Actuate Therapeutics has announced promising results from its Phase 2 trial of the drug elraglusib for treating metastatic pancreatic ductal adenocarcinoma. The trial demonstrated a statistically significant improvement in overall survival for patients treated with elraglusib in combination with chemotherapy compared to chemotherapy alone. The company is preparing to discuss these findings with U.S. and EU regulators to advance toward a New Drug Application. Additionally, Actuate has identified seven biomarkers that may help predict which patients could benefit from elraglusib, with CXCL2 being a particularly reliable indicator of improved survival. These findings were presented at the American Society of Clinical Oncology annual meeting. In another development, Craig-Hallum has initiated coverage of Actuate Therapeutics with a Buy rating and a price target of $21, citing optimism about elraglusib’s potential to improve survival outcomes. The firm believes that the interim data from the Phase 2 study supports this positive outlook. Actuate’s financial position, with $8.6 million in cash, may be bolstered by these positive clinical developments.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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