- 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<0.0001). These effects were associated with increased infiltration of CD4+ and CD8+ T cells and NK cells within the tumor microenvironment, suggesting a meaningful immunomodulatory component.
Clinical findings further support the development in neuroblastoma. In the Phase 1/2 (NCT04239092) study of elraglusib in combination with cyclophosphamide plus topotecan chemotherapy, patients with advanced relapsed or refractory disease achieved clinical responses or disease control. Importantly, a heavily pretreated neuroblastoma patient with a high-risk molecular profile achieved a complete bone marrow response within 9 cycles.
About BEACON2
BEACON2 (EudraCT 2024-516115-24) is an international, open-label, multi-arm, multi-stage randomized Phase 1/2 trial evaluating multiple treatment regimens aimed at identifying treatment approaches that will lead to improved outcomes. BEACON2 is being conducted by the Cancer Research UK Clinical Trials Unit (CRCTU), University of Birmingham, one of the most respected academic clinical trials organizations in the world and one of only eight units directly funded by Cancer Research UK, the UK's largest cancer research charity. The trial is currently active across 14 UK sites and has received European regulatory approval via CTIS, with an international network spanning up to 60 sites across 16 countries, including the European Union, UK, Switzerland, Israel, Australia, and New Zealand.
BEACON2 builds on the original BEACON trial, which established a pan-European platform for evaluating and comparing treatment regimens in relapsed and refractory neuroblastoma. Conducted between 2012 and 2021, BEACON enrolled 225 patients across Europe, making it the largest randomized clinical trial ever conducted in this patient population. The study successfully identified two combination regimens worthy of further investigation, including irinotecan and temozolomide and dinutuximab beta administered with a chemotherapy backbone, which demonstrated one-year progression-free survival rates of 65% and 49%, respectively.
About Neuroblastoma
Neuroblastoma is a rare pediatric cancer that develops from immature nerve cells and remains associated with persistently poor outcomes following relapse. Despite advances in frontline therapy, treatment options remain limited for patients whose disease recurs or becomes resistant to treatment, highlighting the need for new therapeutic approaches.
About Actuate Therapeutics, Inc.
Actuate is a clinical-stage biopharmaceutical company focused on developing therapies for the treatment of high-impact, difficult-to-treat cancers. Actuate’s lead investigational drug, elraglusib (a novel GSK-3β inhibitor), targets molecular pathways in cancer that are involved in promoting tumor growth and resistance to conventional cancer drugs such as chemotherapy through the inhibition of nuclear factor kappa-light-chain-enhancer of activated B cells (NF-kB) and DNA Damage Response (DDR). Elraglusib may also mediate anti-tumor immunity through the regulation of multiple immune checkpoints and immune cell function.
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Forward Looking Statements
This press release contains forward-looking statements about us, including our and other parties’ clinical trials and development plans, and our industry. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “might,” “ongoing,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would,” or the negative of these terms or other comparable terminology are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. All statements, other than statements related to present facts or current conditions or of historical facts, contained in this press release are forward-looking statements. Accordingly, these statements involve estimates, assumptions, substantial risks and uncertainties which could cause actual results to differ materially from those expressed in them, including but not limited to that preliminary and unpublished data may be subject to change and further interpretation following the availability of more data or following a more comprehensive review of the data and should not be relied upon as a final analysis; clinical and preclinical drug development involves a lengthy and expensive process with uncertain timelines and outcomes, results of prior preclinical studies, early clinical trials and sub-group studies are not necessarily predictive of future results and may not correlate with improved responses, and elraglusib may not achieve positive clinical results or favorable preclinical results or receive regulatory approval on a timely basis, if at all; that we may not successfully enroll additional patients or establish or advance plans for further development, including through conversations with the FDA or EMA and the standards such bodies may impose for such development; that elraglusib could be associated with side effects, adverse events or other properties or safety risks, which could delay or preclude regulatory approval, cause us to suspend or discontinue clinical trials or result in other negative consequences; our reliance on third parties to conduct our non-clinical studies and our clinical trials; our reliance on third-party licensors and ability to preserve and protect our intellectual property rights; that we face significant competition from other biotechnology and pharmaceutical companies; our ability to fund development activities, including because our financial condition raises substantial doubt as to our ability to continue as a going concern and we require additional capital to finance our operations beyond July 2026, and a failure to obtain this necessary capital in the near term on acceptable terms, or at all, could force us to delay, limit, reduce or terminate our development programs, commercialization efforts or other operations. In addition, any forward-looking statements are qualified in their entirety by reference to the factors discussed under the heading “Item 1A. Risk Factors” in our Annual Report on Form 10-K for the year ended December 31, 2025, filed with the SEC on March 26, 2026, and our Quarterly Reports on Form 10-Q, and other filings with the SEC. Because the risk factors referred to above could cause actual results or outcomes to differ materially from those expressed in any forward-looking statements made by us or on our behalf, you should not place undue reliance on any forward-looking statements. Further, any forward-looking statement speaks only as of the date on which it is made. New factors emerge from time to time, and it is not possible for us to predict which factors will arise. In addition, we cannot assess the impact of each factor on our business or the extent to which any factor, or combination of factors, may cause actual results to differ materially from those contained in any forward-looking statements. Unless legally required, we do not undertake any obligation to release publicly any revisions to such forward-looking statements to reflect events or circumstances after the date of this press release or to reflect the occurrence of unanticipated events.
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