- BEACON2 is a landmark European multi-arm, multi-stage Phase 1/2 platform trial conducted and sponsored by the Cancer Research UK Clinical Trials Unit (CRCTU), University of Birmingham - Elraglusib will be assessed in combination with dinutuximab beta plus chemotherapy in patients with relapsed and refractory neuroblastoma- Complete response in neuroblastoma patient achieved in Phase 1 study of elraglusib in refractory pediatric cancers
- Elraglusib has received FDA Rare Pediatric Disease designation for neuroblastoma
CHICAGO and FORT WORTH, Texas, June 09, 2026 (GLOBE NEWSWIRE) -- Actuate Therapeutics, Inc. (NASDAQ: ACTU), a clinical-stage biopharmaceutical company focused on developing novel therapies for difficult-to-treat cancers, today announced that elraglusib will be evaluated in the BEACON2 clinical trial, an international, multi-arm, multi-stage platform study designed to identify and advance promising treatment approaches for children with relapsed and refractory neuroblastoma.
Under the BEACON2 trial design, the combination of elraglusib with dinutuximab beta plus chemotherapy will initially be assessed in a dose confirmation cohort of up to 20 patients to evaluate safety and to determine the maximum tolerated dose (MTD), recommended Phase 2 dose (RP2D), and pharmacokinetics (PK) profile of the regimen. Following successful completion of the dose confirmation stage, the regimen may advance into a randomized portion of the platform trial, where approximately 75 patients will be enrolled with a planned interim analysis.
“Selection for inclusion in BEACON2 represents a defining milestone for the elraglusib program and underscores growing recognition of its novel mechanism and potential applicability in treating pediatric cancers,” said Daniel Schmitt, President & Chief Executive Officer of Actuate. “Neuroblastoma remains one of the most devastating diseases for many children whose cancer relapses or becomes resistant to treatment, and we are honored to be part of a collaborative effort dedicated to improving outcomes of these patients. BEACON2 is sponsored by the Cancer Research UK Clinical Trials Unit, University of Birmingham, enabling us to generate meaningful clinical data in a capital-efficient manner. We are particularly encouraged by the convergence of compelling preclinical evidence, early clinical evidence in pediatric cancer, including a complete response achieved in a neuroblastoma patient, and regulatory recognition through FDA Rare Pediatric Disease Designation. Together, these factors provide a strong foundation for further exploring the potential of elraglusib in this area of significant unmet medical need.“
Scientific Rationale
The scientific rationale for evaluating elraglusib in neuroblastoma is firmly supported by a growing body of preclinical and clinical evidence. GSK-3β inhibition, elraglusib’s primary mechanism, has been shown to suppress neuroblastoma cell proliferation, promote apoptosis, and disrupt pathways involved in MYCN-driven disease biology, a key driver of high-risk neuroblastoma. Preclinical studies demonstrated that elraglusib enhanced the activity of both chemotherapy and anti-GD2-based immunotherapy, producing significant improvements in tumor control and survival across multiple neuroblastoma models. Notably, 60% of Th-MYCN mice treated with elraglusib plus temozolomide/irinotecan (TEMIRI)/14G2a, remained tumor-free at one year of age compared to 0% in the TEMIRI/14G2a only arm (P