Actuate Therapeutics, Inc. (NASDAQ: ACTU) has announced that its investigational drug elraglusib will be evaluated in the BEACON2 clinical trial for children with relapsed and refractory neuroblastoma. The selection for the trial underscores the potential of elraglusib to address a significant unmet medical need in pediatric oncology. BEACON2 is an international, multi-arm, multi-stage platform study sponsored by the Cancer Research UK Clinical Trials Unit (CRCTU) at the University of Birmingham.

Under the trial design, elraglusib will be assessed in combination with dinutuximab beta plus chemotherapy. The initial phase involves a dose confirmation cohort of up to 20 patients to evaluate safety and determine the maximum tolerated dose (MTD), recommended Phase 2 dose (RP2D), and pharmacokinetics (PK) profile of the regimen. Following successful completion of this stage, the regimen may advance to a randomized portion involving approximately 75 patients, which includes a planned interim analysis.

The scientific rationale for the trial is supported by preclinical and clinical evidence. Elraglusib is a GSK-3β inhibitor that has been shown to suppress neuroblastoma cell proliferation and promote apoptosis. Preclinical studies demonstrated that 60% of Th-MYCN mice treated with elraglusib plus chemotherapy remained tumor-free at one year, compared to 0% in the chemotherapy-only arm.

Clinical findings from a Phase 1/2 study showed that patients with advanced relapsed or refractory disease achieved clinical responses or disease control. Notably, a heavily pretreated neuroblastoma patient achieved a complete bone marrow response. Elraglusib has also received FDA Rare Pediatric Disease designation for neuroblastoma.

BEACON2 builds on the original BEACON trial, which enrolled 225 patients across Europe. The new trial is active across 14 UK sites and has received European regulatory approval via CTIS, with plans to expand to up to 60 sites across 16 countries.