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

▎药明康德内容团队编辑   Actuate Therapeutics日前公布其在研GSK-3β小分子抑制剂elraglusib联合吉西他滨/白蛋白紫杉醇（GnP），用于转移性胰腺导管腺癌（mPDAC）一线治疗2期试验Actuate-1801 Part 3B的积极结果。 Actuate-1801 Part 3B研究是一项随机对照的2期临床试验，旨在评估elraglusib联合GnP与GnP单独相比，作为mPDAC一线治疗中的疗效和安全性。该研究共纳入286例未接受过系统性治疗的mPDAC患者，按2：1的比例随机分配至elraglusib联合治疗组（elraglusib + GnP）或对照组（GnP单独治疗）。 中期数据分析显示，试验达成主要终点。Elraglusib联合治疗组患者的1年生存率为43.6%，显著高于活性对照组的22.5%（p=0.002）。其中elraglusib联合治疗组患者的18个月与24个月生存率分别为20.9%和16.7%，而活性对照组则皆为0%。Elraglusib联合治疗组患者的死亡风险降低了37%，中位总生存期（mOS）显著延长至9.3个月，而活性对照组患者的mOS则为7.2个月（HR=0.63，p=0.016）。 ▲Actuate Therapeutics管线图（图片来源：Actuate Therapeutics公司官网） 截至2024年11月15日，elraglusib联合治疗组与活性对照组患者的客观缓解率（ORR）分别为27.7%与20.5%；疾病控制率（DCR）分别为42.6%与33.3%；无进展生存期（PFS）分别是5.6个月与4.9个月。 值得一提的是，elraglusib联合治疗组中有2例原本不可手术的患者，经过治疗后靶病灶明显缩小，成功接受了手术切除，其中1例在手术后达到了100%肿瘤负荷清除。此外，该组还观察到1例完全缓解（CR）和1例部分缓解（PR），两者均实现了靶病灶100%缩小，而对照组未出现类似结果。 安全性方面，试验达到了主要的安全性终点。Elraglusib联合治疗组的不良事件（TEAEs）和严重不良事件（SAEs）与活性对照组相似，显示出良好的风险-获益特征。 Elraglusib（9-ING-41）是一种选择性的强效GSK-3β抑制剂，已在临床试验中显示出积极的抗癌活性。目前，elraglusib正在开发用于治疗mPDAC。在临床前模型中，elraglusib表现出免疫调节作用，并在临床样本中显示出对血清细胞因子谱的调节能力。 相关阅读 减少化疗，疗效相当，还更安全！《柳叶刀》子刊：转移性胰腺癌新治疗策略 《柳叶刀》子刊：胰腺癌获突破性进展！溶瘤病毒疗法安全可行 医科院肿瘤医院：每16人就有1人会死于消化系统癌症 参考资料： [1] Actuate Therapeutics Announces Positive Interim Phase 2 Data of Elraglusib in First Line Treatment of Metastatic Pancreatic Cancer. Retrieved December 17, 2024 from https://www.globenewswire.com/news-release/2024/12/17/2998209/0/en/Actuate-Therapeutics-Announces-Positive-Interim-Phase-2-Data-of-Elraglusib-in-First-Line-Treatment-of-Metastatic-Pancreatic-Cancer.html [2] 2024-ASCO-Identification of immune biomarkers in treatment-naive patients with metastatic pancreatic cancer treated with the GSK-3 inhibitor elraglusib (9-ING-41) in combination with gemcitabine/nab-paclitaxel in the 1801 phase 2 study. Retrieved December 17, 2024 from https://actuatetherapeutics.com/wp-content/uploads/2024/09/Weiskittel-Publication-Pancreatic-cancer-ASCO-2024.pdf 免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。 版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。 分享，点赞，在看，传递医学新知