Accent Therapeutics宣布在新型 KIF18A抑制剂 ATX-295的 1/2期试验中完成首例患者给药，并获得 FDA对其主要资产 ATX-295和 DHX9抑制剂 ATX-559的快速通道资格-动脉网

Accent Therapeutics Announces First Patient Dosed in Phase 1/2 Trial of Novel KIF18A Inhibitor ATX-295 and Receives FDA Fast Track Designation for Lead Assets ATX-295 and DHX9 Inhibitor ATX-559

CISION 等信源发布 2025-04-15 18:00

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First-in-human Phase 1/2 study initiated for ATX-295, a potential best-in-class oral inhibitor of KIF18A, focused on patients with advanced solid tumors including ovarian cancer

针对ATX-295（一种潜在的同类最优KIF18A口服抑制剂）的首次人体1/2期研究已经启动，重点面向包括卵巢癌在内的晚期实体瘤患者。

FDA grants Fast Track designation to Accent's two lead clinical-stage programs – ATX-295 and ATX-559 – for the treatment of adult patients with advanced/metastatic platinum-resistant or refractory ovarian cancer, and unresectable/metastatic dMMR/MSI-H colorectal cancer post checkpoint inhibitor treatment, respectively.

FDA授予Accent公司的两个主要临床阶段项目——ATX-295和ATX-559——快速通道资格，分别用于治疗晚期/转移性铂类耐药或难治性卵巢癌成年患者，以及检查点抑制剂治疗后的不可切除/转移性dMMR/MSI-H结直肠癌患者。

LEXINGTON, Mass.

马萨诸塞州列克星敦

，

April 15, 2025

2025年4月15日

/PRNewswire/ -- Accent Therapeutics, a clinical-stage biopharmaceutical company focused on novel, targeted, small molecule cancer therapeutics, today announced that the first patient has been dosed in a first-in-human Phase 1/2 clinical trial evaluating the safety and tolerability of ATX-295, a potential best-in-class oral KIF18A inhibitor.

/PRNewswire/ -- 专注于新型靶向小分子癌症治疗药物的临床阶段生物制药公司 Accent Therapeutics 今天宣布，第一名患者已在一项首次人体 Phase 1/2 期临床试验中接受给药，该试验旨在评估潜在同类最佳口服 KIF18A 抑制剂 ATX-295 的安全性和耐受性。

In addition, the company has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for ATX-295 for the treatment of adult patients with advanced/metastatic platinum-resistant or refractory ovarian cancer, and for ATX-559, a first-in-class potent and selective inhibitor of DHX9, for the treatment of adult patients with unresectable/metastatic dMMR/MSI-H colorectal cancer post checkpoint inhibitor treatment..

此外，该公司已获得美国食品和药物管理局 (FDA) 的快速通道资格，分别为 ATX-295 治疗晚期/转移性铂类耐药或难治性卵巢癌成年患者，以及 ATX-559（一种首创的强效且选择性的 DHX9 抑制剂）用于治疗在接受检查点抑制剂治疗后不可切除/转移性的 dMMR/MSI-H 结直肠癌成年患者。

'Cancers with high chromosomal instability, such as in certain ovarian, breast, and lung cancers, collectively affect a large patient population but have limited treatment options. With ATX-295 entering the clinic, we are excited to translate multiple years of KIF18A research into the development of a potentially best-in-class program,' said .

“具有高染色体不稳定性癌症，如某些卵巢癌、乳腺癌和肺癌，总体上影响了大量患者，但治疗选择有限。随着ATX-295进入临床，我们很高兴将多年对KIF18A的研究转化为可能成为同类最佳的开发项目，”他表示。

Jason Sager

杰森·塞格尔

, M.D., Chief Medical Officer of Accent Therapeutics. 'With the launch of our Phase 1/2 clinical trial of ATX-295, we now have two investigational drugs in the clinic, bringing us closer to achieving our mission of transforming cancer care. Additionally, receiving FDA Fast Track designation for both of our lead assets underscores the power of our approach and the potential for these investigational drugs to urgently address high unmet medical needs.'.

，医学博士，Accent Therapeutics首席医疗官。“随着我们ATX-295的1/2期临床试验的启动，我们现在已有两种在研药物进入临床阶段，这使我们更接近于实现改变癌症治疗的使命。此外，我们两个主要在研药物获得FDA快速通道资格，也凸显了我们方法的优势以及这些在研药物在紧急满足高度未满足医疗需求方面的潜力。”

ATX-295 is a selective inhibitor of KIF18A, a mitotic kinesin motor protein critical for cell division in select tumors with chromosomal instability, but not in healthy cells. Accent has demonstrated that its novel, potent, and selective small molecule KIF18A inhibitor displays selective dose-dependent tumor growth inhibition in preclinical models, including in high grade serous ovarian cancer and triple negative breast cancer, supporting its advancement to the clinic..

ATX-295是一种选择性KIF18A抑制剂，KIF18A是一种在染色体不稳定的特定肿瘤中对细胞分裂至关重要的有丝分裂驱动蛋白，但在健康细胞中并不重要。Accent已证明其新型、强效且选择性的小分子KIF18A抑制剂在临床前模型中显示出选择性的剂量依赖性肿瘤生长抑制作用，包括高级别浆液性卵巢癌和三阴性乳腺癌，为其进入临床试验提供了支持。

The ATX-295 Phase 1/2 open-label, dose-escalation and expansion study (

ATX-295 第1/2阶段开放标签、剂量递增和扩展研究 (

NCT06799065

) is designed to evaluate the molecule's safety profile at multiple dose levels, assessing the tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of orally administered ATX-295. The trial is enrolling patients with locally advanced or metastatic solid tumors, including high-grade serious ovarian cancer..

旨在评估该分子在多个剂量水平下的安全性，评估口服ATX-295的耐受性、药代动力学、药效学和初步疗效。该试验正在招募局部晚期或转移性实体瘤患者，包括高级别严重卵巢癌。

The initiation of the Phase 1/2 ATX-295 study follows closely after Accent's initial clinical asset, ATX-559, a first-in-class oral inhibitor of DHX9, entered clinical evaluation in late 2024. Both clinical assets have been granted Fast Track status by the FDA. Fast Track designation is designed to facilitate the development and expedite the review of novel drug candidates that address serious conditions marked by unmet medical need, with the aim of accelerating patient access to novel treatment options..

1/2期ATX-295研究的启动紧随Accent的首个临床资产ATX-559之后，ATX-559是一种首创的DHX9口服抑制剂，于2024年底进入临床评估。这两种临床资产均已获得FDA的快速通道资格。快速通道指定旨在促进针对未满足医疗需求的重大疾病的新药候选物的开发，并加快其审评进程，以加速患者获得创新治疗选择的速度。

Accent will present new preclinical data supporting the continued clinical assessment of ATX-295 and ATX-559 at the 2025 AACR Annual Meeting taking place

Accent 将在 2025 年 AACR 年会上展示支持继续对 ATX-295 和 ATX-559 进行临床评估的新的临床前数据。

April 25-30

4月25日至30日

在

Chicago

芝加哥

, Illinois. Accent will also present a trial-in-progress update on the ATX-559 Phase 1/2 clinical trial at the 2025 ASCO Annual Meeting taking place

，伊利诺伊州。Accent还将在2025年ASCO年会上提供ATX-559一期/二期临床试验的进展更新，会议将于

May 30

5月30日

–

June 3

6月3日

在

Chicago, Illinois

伊利诺伊州芝加哥市

。

About ATX-559

关于ATX-559

ATX-559 is a first-in-class potent and selective inhibitor of DHX9, a novel and previously undrugged RNA and DNA/RNA helicase, shown to play a critical role in tumors with high levels of replication stress (including breast, ovarian, colorectal, endometrial, gastric, and others), representing large patient populations with significant unmet medical need.

ATX-559 是一种首创的高效且选择性的 DHX9 抑制剂，DHX9 是一种新型的、此前未被药物靶向的 RNA 和 DNA/RNA 解旋酶，已被证明在具有高水平复制应激的肿瘤（包括乳腺癌、卵巢癌、结直肠癌、子宫内膜癌、胃癌等）中发挥关键作用，代表了具有显著未满足医疗需求的广大患者群体。

DHX9 has been reported to play important roles in replication, transcription, translation, RNA splicing, RNA processing, and maintenance of genomic stability, making it a compelling novel oncology target. In addition to exploiting key tumor vulnerabilities in DNA repair deficient backgrounds (e.g., BRCA) and hyper-mutated states (e.g., MSI-H/dMMR), Accent is exploring the sensitivity of other tumor types to DHX9 inhibition, and the potential to combine DHX9 inhibitors with other cancer treatments to maximize its full potential for helping patients.

DHX9 已被报道在复制、转录、翻译、RNA 剪接、RNA 加工和基因组稳定性维持中发挥重要作用，使其成为一个引人注目的新型肿瘤学靶点。除了利用 DNA 修复缺陷背景（如 BRCA）和高突变状态（如 MSI-H/dMMR）中的关键肿瘤脆弱性外，Accent 还在探索其他肿瘤类型对 DHX9 抑制的敏感性，以及将 DHX9 抑制剂与其他癌症治疗方法结合使用的潜力，以充分实现其帮助患者的潜力。

Accent retains full worldwide rights to ATX-559, currently being evaluated in a Phase 1/2 clinical trial .

Accent 保留了 ATX-559 的全球完整权利，目前该药物正在 1/2 期临床试验中进行评估。

(NCT06625515)

, and the DHX9 program.

，以及 DHX9 计划。

About ATX-295

关于ATX-295

Accent's second lead program, ATX-295, is a potential best-in-class inhibitor for KIF18A which may address a large patient population across several cancer indications, including ovarian and triple negative breast cancer (TNBC). KIF18A is a mitotic kinesin motor protein critical for cell division in select tumors with chromosomal instability, but not in healthy cells.

Accent的第二个主要项目ATX-295是一种潜在的同类最佳KIF18A抑制剂，可应用于包括卵巢癌和三阴性乳腺癌（TNBC）在内的多种癌症适应症，覆盖广泛的患者群体。KIF18A是一种有丝分裂驱动蛋白，在染色体不稳定的特定肿瘤中对细胞分裂至关重要，但在健康细胞中并不起作用。

KIF18A inhibitor treatment results in rapid cell death for cancers with an abnormal number of chromosomes (aneuploid) .

KIF18A抑制剂治疗会导致染色体数目异常（非整倍体）的癌细胞迅速死亡。

in vitro

体外

and

和

in vivo

体内

, while cells with normal numbers of chromosomes (euploid) are unaffected. Accent retains full worldwide rights to the KIF18A program, currently being evaluated in a Phase 1/2 clinical trial enrolling solid tumor patients (

，而染色体数目正常的细胞（整倍体）则不受影响。Accent 拥有 KIF18A 项目的全球完整权利，该项目目前正在一项 1/2 期临床试验中进行评估，招募实体瘤患者 (

NCT06799065

）。

About Accent Therapeutics

关于Accent Therapeutics

Accent Therapeutics is pioneering a new class of small molecule precision cancer therapies targeting critical intracellular dependencies that span multiple types of cancer. Building upon industry-leading expertise in RNA-modifying proteins (RMPs) and the systematic mapping of both the RMP space and adjacent high-value areas for drug discovery, the company employs a flexible model that allows for a diversity of approaches to developing potentially transformative biomarker-driven cancer medicines.

Accent Therapeutics公司正在开创一类新的小分子精准癌症治疗方法，靶向多种癌症的关键细胞内依赖性。基于在RNA修饰蛋白（RMPs）领域的行业领先专业知识，并系统地绘制了RMP领域及药物发现相关的高价值邻近区域，该公司采用灵活的模式，允许通过多样化的途径开发潜在变革性的生物标志物驱动型癌症药物。

Accent's therapies are designed for both novel and known, but suboptimally addressed, high-impact oncology targets with the potential to benefit large patient populations with significant unmet need. For more information on Accent's mission to translate extraordinary science into life-changing therapeutics for patients living with cancer, visit .

Accent的疗法设计针对新颖及已知但未得到最佳解决的高影响力肿瘤靶点，有潜力使具有显著未满足需求的广大患者群体受益。如需了解更多关于Accent将非凡科学转化为改变生命疗法、服务癌症患者使命的信息，请访问。

www.accenttx.com

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