Elraglusib

CHICAGO and FORT WORTH, Texas, Jan. 07, 2025 (GLOBE NEWSWIRE) -- Actuate Therapeutics, Inc. (NASDAQ: ACTU) (“Actuate” or the “Company”), a clinical-stage biopharmaceutical company focused on developing therapies for the treatment of high-impact, difficult-to-treat cancers through the inhibition of glycogen synthase kinase-3 beta (GSK-3β), today announced that the European Medicines Agency (EMA) has granted Orphan Medicinal Product Designation (OMPD) to elraglusib, a novel GSK-3β inhibitor, for the treatment of pancreatic ductal adenocarcinoma (PDAC). “With limited treatment options available for patients with PDAC, the EMA Orphan Drug Designation and FDA Orphan Drug Designation granted to elraglusib for the same indication reflect elraglusib’s potential to address a critical need for people living with this aggressive disease with a notably poor prognosis. This regulatory milestone, along with the recently announced positive interim results from our Phase 2 trial of elraglusib in patients with mPDAC that showed a significant clinical benefit and anti-tumor activity, should expedite our efforts to advance elraglusib as a novel, potential treatment for mPDAC,” said Daniel Schmitt, President & Chief Executive Officer of Actuate. “We look forward to reporting topline data from our Phase 2 trial in 1H 2025 and working closely with the EMA and the FDA to accelerate elraglusib’s clinical development program in metastatic pancreatic cancer.” Elraglusib is a novel GSK-3β inhibitor currently in a randomized Phase 2 trial (Actuate-1801 Part 3B study, NCT03678883) comparing the combination of elraglusib with gemcitabine/nab-paclitaxel (GnP) to GnP alone as a first line therapy in patients with metastatic pancreatic cancer (mPDAC). A recently reported analysis of interim Phase 2 data demonstrated that treatment with the elraglusib combination led to statistically significant increases in 1-year survival rate (p value of 0.002) and median overall survival (hazard ratio of 0.63, p value of 0.016) versus treatment with GnP alone. Orphan Designation is granted to therapies intended for the treatment, prevention, or diagnosis of life-threatening or chronically debilitating diseases that affect no more than two in 10,000 people in the European Union (EU) and for which no satisfactory therapy is available. The treatment must also provide significant benefit to those affected by the condition. EMA orphan drug designation provides certain benefits, including the potential for 10 years of market exclusivity following regulatory approval in the EU, reduction in regulatory fees and a centralized EU approval process1. 1. European Medicines Agency, "Orphan Designation." https://www.ema.europa.eu/en/human-regulatory-overview/orphan-designation-overview 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 product, 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 including several DNA Damage Response (DDR) pathways. Elraglusib is designed to act as a mediator of anti-tumor immunity through the inhibition of nuclear factor kappa-light-chain-enhancer of activated B cells (NF-kB) and regulates multiple immune checkpoints and immune cell function. For additional information, please visit the Company’s website at http://www.actuatetherapeutics.com. Forward-Looking Statements This press release contains forward-looking statements about us, including our 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 clinical and preclinical drug development involves a lengthy and expensive process with uncertain timelines and outcomes, results of prior preclinical studies and early clinical trials are not necessarily predictive of future results, 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; preliminary and unpublished data may be subject to change 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; 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; and our ability to realize any benefits or value associated with the Orphan Medicinal Product Designation (OMPD) granted to elraglusib by the EMA for the treatment of pancreatic ductal adenocarcinoma (PDAC). 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 Quarterly Report on Form 10-Q for the quarter ended September 30, 2024 filed with the SEC on November 13, 2024 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. Investor Contact Mike MoyerManaging DirectorLifeSci Advisors, LLCmmoyer@lifesciadvisors.com

CHICAGO, IL and FORT WORTH, TX, USA I December 17, 2024 I Actuate Therapeutics, Inc. (NASDAQ: ACTU) (“Actuate” or the “Company”), a clinical-stage biopharmaceutical company focused on developing therapies for the treatment of high-impact, difficult-to-treat cancers through the inhibition of glycogen synthase kinase-3 beta (GSK-3β), today announced new positive data from its ongoing and fully enrolled randomized Phase 2 trial of elraglusib in combination with gemcitabine/nab-paclitaxel (GnP) in first line treatment of metastatic pancreatic ductal adenocarcinoma (mPDAC) (Actuate-1801 Part 3B). A pre-specified analysis was triggered by reaching >70% death events in the GnP control arm (data cut off November 15, 2024). The analysis of interim data demonstrated treatment with elraglusib in combination with GnP resulted in statistically significant increases in 1-year survival rate (p value of 0.002) and median overall survival (p value of 0.016, hazard ratio of 0.63) versus treatment with GnP alone. The combination treatment also resulted in increased Objective Response Rates (ORR) and Disease Control Rates (DCR) in the elraglusib/GnP combination arm versus the GnP control arm. “These interim results are highly encouraging and underscore the transformative potential of elraglusib in the treatment of metastatic pancreatic cancer,” said Daniel Schmitt, President & Chief Executive Officer of Actuate. “The improvement in median and overall long-term survival, disease control rate and overall response rate compared to GnP alone, combined with elraglusib’s favorable risk-benefit profile, offers the possibility of improved outcomes for patients with limited treatment alternatives. We are excited to continue evaluating the potential of elraglusib, a novel immune-oncology GSK-3 targeted approach for mPDAC, and look forward to engaging with the FDA in 1H 2025 to share topline data and discuss next steps, including a proposed phase 3 registration trial.” Inhibition of GSK-3β may inhibit tumor growth and improve survival through several complimentary mechanisms that include enhancement of chemotherapy activity, activation of innate anti-tumor immunity and regulation of gene expression, leading to alterations in tumor metabolism and Epithelial-to-Mesenchymal Transition (EMT). Study highlights include: “Pancreatic cancer is one of the most aggressive and lethal cancers with less than 5% survival rate in the US at 5 years. We are encouraged by consistent evidence of significant clinical benefit and anti-tumor activity in the elraglusib combination arm across multiple endpoints in this study,” said Dr. Andrew Mazar, Actuate’s Scientific Co-Founder and Chief Operating Officer. “Further, the favorable risk-benefit profile observed to date may provide a treatment option for many patients with metastatic pancreatic cancer that may not tolerate more aggressive and less tolerable chemotherapy approaches.” The ongoing Actuate-1801 Part 3B study (NCT04239092) is a randomized, controlled Phase 2 trial of elraglusib with GnP versus GnP alone in first line mPDAC. The trial enrolled 286 mPDAC patients with no prior systemic treatment for metastatic disease, who were randomized 2:1 to the elraglusib treatment arm (elraglusib + GnP) or the control arm (GnP alone). Elraglusib is administered at a dose of 9.3 mg/kg by IV infusion on Day 1 of each week of a 28-day cycle. The primary endpoint is 1-year survival rate, with mOS the primary parameter for summarization of survival data at the end of the study. Secondary endpoints are DCR, ORR, PFS and AE. 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 product, 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 including several DNA Damage Response (DDR) pathways. Elraglusib is designed to act as a mediator of anti-tumor immunity through the inhibition of nuclear factor kappa-light-chain-enhancer of activated B cells (NF-kB) and regulates multiple immune checkpoints and immune cell function. For additional information, please visit the Company’s website at http://www.actuatetherapeutics.com. SOURCE : Actuate Therapeutics