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)--Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with RAS/MAPK pathway-driven cancers, today announced its 2025 priorities and upcoming catalysts for its novel clinical pipeline.

)--Verastem Oncology（Nasdaq:VSTM）是一家致力于为RAS/MAPK途径驱动的癌症患者开发新药的生物制药公司，今天宣布了其2025年的优先事项和即将推出的新型临床管道催化剂。

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We ended 2024 having made tremendous progress across our pipeline programs, including FDA acceptance of our NDA with Priority Review for avutometinib plus defactinib in recurrent KRAS mutant low-grade serous ovarian cancer. As we head into 2025, we are building on the foundational milestones achieved in 2024 and are poised for a transformative year of growth as we evolve into a commercial-stage company while advancing several clinical programs,” said Dan Paterson, president and chief executive officer at Verastem Oncology.

截至2024年，我们在管道计划中取得了巨大进展，包括FDA接受我们的NDA，并优先审查avutometinib加defactinib治疗复发性KRAS突变型低度浆液性卵巢癌。随着我们进入2025年，我们将在2024年取得的基础里程碑的基础上再接再厉，并准备迎来一个变革性的增长年份，因为我们将发展成为一家商业阶段的公司，同时推进多项临床项目，”Verastem Oncology总裁兼首席执行官丹·帕特森（DanPaterson）表示。

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With the addition of VS-7375, a potential best-in-class oral KRAS G12D (ON/OFF) inhibitor, we are well-positioned to further establish our leadership in targeting RAS/MAPK pathway-driven cancers, including metastatic pancreatic cancer, non-small cell lung cancer, and KRAS G12D mutant solid tumors.”

随着VS-7375（一种潜在的同类最佳口服KRAS G12D（开/关）抑制剂）的加入，我们将进一步确立我们在靶向RAS/MAPK途径驱动的癌症方面的领导地位，包括转移性胰腺癌，非小细胞肺癌和KRAS G12D突变实体瘤。”

In 2025, Verastem will focus on three strategic priorities to drive sustainable long-term growth:

2025年，Verastem将重点关注三个战略优先事项，以推动可持续的长期增长：

Successfully launch avutometinib plus defactinib in recurrent KRAS mutant low-grade serous ovarian cancer (LGSOC) in the U.S. and continue to advance the regulatory pathway in Japan and Europe

在美国成功推出avutometinib加defactinib治疗复发性KRAS突变型低度浆液性卵巢癌（LGSOC），并继续推进日本和欧洲的监管途径

Maximize the synergistic potential of the avutometinib plus defactinib combination in other advanced solid tumors for market expansion opportunities

最大限度地发挥avutometinib联合defactinib在其他晚期实体瘤中的协同潜力，以扩大市场

Advance its novel, early-stage pipeline, including its potential best-in-class oral KRAS G12D (ON/OFF) inhibitor, to create multiple opportunities to demonstrate transformative outcomes for people living with RAS/MAPK pathway-driven cancers

推进其新颖的早期管道，包括其潜在的同类最佳口服KRAS G12D（开/关）抑制剂，为RAS/MAPK途径驱动的癌症患者创造多种机会，证明其具有变革性的结果

Successfully Launch Avutometinib Plus Defactinib in the U.S. and Continue to Advance the Regulatory Pathway in Japan and Europe

在美国成功推出Avutometinib Plus Defactinib，并继续推进日本和欧洲的监管途径

On December 30, 2024, the U.S. Food and Drug Administration (FDA) accepted the Company’s New Drug Application (NDA) under the accelerated approval pathway and granted Priority Review for avutometinib, an oral RAF/MEK clamp, in combination with defactinib, an oral, selective FAK inhibitor, in adult patients with recurrent KRAS mutant LGSOC and designated June 30, 2025, as the Prescription Drug User Fee Act (PDUFA) action date.

2024年12月30日，美国食品和药物管理局（FDA）根据加速批准途径接受了该公司的新药申请（NDA），并批准优先审查avutometinib（一种口服RAF/MEK钳）与defactinib（一种口服选择性FAK抑制剂）联合用于复发性KRAS突变LGSOC的成年患者，并指定2025年6月30日为处方药使用者费用法案（PDUFA）行动日期。

The NDA was based on the positive, mature safety and efficacy data from the RAMP 201 trial as .

NDA基于RAMP 201试验as的积极，成熟的安全性和有效性数据。

presented

介绍

at the International Gynecologic Cancer Society (IGCS) 2024 Annual Meeting. The NDA also includes supportive data from the FRAME Phase 1 trial, the first study conducted with the combination therapy in recurrent LGSOC. These data underscore how avutometinib plus defactinib could address a significant unmet medical need among patients with recurrent LGSOC, if approved..

在国际妇科癌症协会（IGCS）2024年年会上。NDA还包括来自FRAME 1期试验的支持性数据，这是首次在复发性LGSOC中进行联合治疗的研究。这些数据强调了如果获得批准，avutometinib加defactinib如何解决复发性LGSOC患者严重未满足的医疗需求。。

To further strengthen its positioning for a potential mid-2025 launch, Verastem previously

为了进一步加强其在2025年年中可能推出的定位，Verastem之前

announced

已宣布

agreements with Oberland Capital and IQVIA. The agreements with Oberland Capital include a debt refinancing and an equity investment, which strengthens the Company’s cash position and will help fund commercialization past FDA approval and other pipeline programs. The strategic collaboration with IQVIA leverages IQVIA’s world-class infrastructure and commercialization solutions to complement the Company’s launch strategy in recurrent LGSOC..

与Oberland Capital和IQVIA的协议。与Oberland Capital的协议包括债务再融资和股权投资，这加强了公司的现金状况，并将有助于通过FDA批准和其他管道计划为商业化提供资金。与IQVIA的战略合作利用了IQVIA世界级的基础设施和商业化解决方案，以补充公司在复发性LGSOC中的发布战略。。

Key Milestones Expected for 2025:

2025年的关键里程碑：

Primary analysis from both the FRAME and RAMP 201 clinical trials will be published in H1 2025; submit RAMP 201 primary analysis publication for NCCN consideration in H1 2025.

FRAME和RAMP 201临床试验的初步分析将于2025年上半年发布；在2025年上半年提交RAMP 201主要分析出版物供NCCN考虑。

Present additional analyses from the RAMP 201 trial at a medical meeting in Q1 2025.

在2025年第1季度的医学会议上介绍RAMP 201试验的其他分析。

Plan for FDA decision on approval of the combination of avutometinib plus defactinib in recurrent KRAS mutant LGSOC, expected by June 30, 2025.

FDA计划于2025年6月30日前批准avutometinib联合defactinib治疗复发性KRAS突变LGSOC。

Complete enrollment for the international Phase 3 confirmatory RAMP 301 clinical trial for patients with recurrent LGSOC regardless of KRAS mutation status by the end of 2025.

到2025年底，完成复发性LGSOC患者的国际3期确认性RAMP 301临床试验的注册，无论KRAS突变状态如何。

Report initial data from the RAMP 201J Phase 2 clinical trial being conducted in Japan with the Japanese Gynecologic Oncology Group (JGOG) evaluating the safety and efficacy of avutometinib in combination with defactinib for recurrent LGSOC in H2 2025.

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Continue to advance the regulatory pathway in Japan and Europe.

继续推进日本和欧洲的监管途径。

Maximize the Synergistic Potential of Avutometinib Plus Defactinib for Advanced Solid Tumor Market Expansion Opportunities

RAMP 205: Avutometinib Plus Defactinib in Combination with Chemotherapy in First-Line Metastatic Pancreatic Cancer

RAMP 205:Avutometinib加Defactinib联合化疗治疗一线转移性胰腺癌

At the American Society of Clinical Oncology (ASCO) Annual Meeting in June 2024, Verastem

在2024年6月的美国临床肿瘤学会（ASCO）年会上，Verastem

presented

介绍

initial interim data from the ongoing RAMP 205 Phase 1/2 clinical trial evaluating multiple dose cohorts of avutometinib plus defactinib in combination with gemcitabine and Nab-paclitaxel as first-line systemic treatment for patients with metastatic pancreatic cancer. Patients receiving the combination in the dose level 1 cohort achieved a confirmed overall response rate (ORR) of 83% (5/6).

来自正在进行的RAMP 205 1/2期临床试验的初步中期数据，该试验评估了avutometinib加defactinib联合吉西他滨和Nab-紫杉醇作为转移性胰腺癌患者的一线全身治疗的多剂量组群。在剂量水平1队列中接受联合治疗的患者的确诊总有效率（ORR）为83%（5/6）。

One dose-limiting toxicity (DLT) was observed in the dose level 1 cohort, and the dose level was subsequently cleared..

在剂量水平1队列中观察到一种剂量限制性毒性（DLT），随后清除了剂量水平。。

Key Milestones Expected for 2025:

2025年的关键里程碑：

Report updated data from the ongoing RAMP 205 trial in Q1 2025 and present data at a medical meeting in mid-year 2025.

报告2025年第1季度正在进行的RAMP 205试验的更新数据，并在2025年年中的一次医学会议上提供数据。

Choose a Recommended Phase 2 Dose (RP2D) for trial expansion in H1 2025.

为2025年上半年的试验扩展选择推荐的2期剂量（RP2D）。

RAMP 203: Avutometinib Plus Defactinib in Combination with a KRAS G12C Inhibitor in Non-Small Cell Lung Cancer (NSCLC)

RAMP 203：Avutometinib加Defactinib联合KRAS G12C抑制剂治疗非小细胞肺癌（NSCLC）

In December 2024, the Company

2024年12月，公司

announced

已宣布

preliminary clinical data for the triplet combination cohort of avutometinib and LUMAKRAS™ (sotorasib) plus defactinib in the RAMP 203 Phase 1/2 study in KRAS G12C mutant advanced NSCLC. No DLTs have been observed in the triplet combination.

在KRAS G12C突变晚期NSCLC的RAMP 203 1/2期研究中，avutometinib和LUMAKRAS™（sotorasib）加defactinib的三联体组合队列的初步临床数据。在三重态组合中未观察到DLT。

Key Milestones Expected for 2025:

2025年的关键里程碑：

Complete enrollment in the KRAS G12C inhibitor, prior-treated Stage 1 Part B cohort in Q1 2025. Continue to follow patients in both doublet cohorts (KRAS G12C inhibitor naïve and prior-treated) for safety and efficacy to determine if observed efficacy supports expanded enrollment.

2025年第1季度，完成KRAS G12C抑制剂，先前治疗的第1阶段B部分队列的注册。继续跟踪两个双重队列（KRAS G12C抑制剂初始和先前治疗）的患者的安全性和有效性，以确定观察到的疗效是否支持扩大登记。

Complete enrollment and evaluate the safety and efficacy of the triplet combination in H1 2025.

在2025年上半年完成登记并评估三联疗法的安全性和有效性。

Present an interim update at a medical meeting in H2 2025.

在2025年下半年的医疗会议上提交临时更新。

Advance Novel, Early-stage Pipeline to Create Multiple Opportunities to Demonstrate Potentially Transformative Outcomes in RAS/MAPK Pathway-driven Cancers

推进新颖的早期管道，创造多种机会，证明RAS/MAPK途径驱动的癌症具有潜在的变革性结果

VS-7375, an Oral KRAS G12D (ON/OFF) Inhibitor, in Advanced Solid Tumors

VS-7375是一种口服KRAS G12D（开/关）抑制剂，用于晚期实体瘤

In July 2024, GenFleet Therapeutics began dosing several patients in a Phase 1/2 trial in China that is evaluating VS-7375 in patients with KRAS G12D-mutated advanced solid tumors. Verastem

2024年7月，GenFleet Therapeutics开始在中国进行1/2期试验，评估KRAS G12D突变晚期实体瘤患者的VS-7375。藜芦

announced

已宣布

on January 14, 2025, that it has exercised its option early to license VS-7375 from GenFleet. In addition, the Company announced preliminary clinical data from the Phase 1 dose-escalation study conducted by GenFleet in China. In the study, VS-7375, demonstrated oral bioavailability, no dose-limiting toxicities across six dose levels, and several partial responses, including patients with pancreatic and lung cancers.

。此外，该公司宣布了GenFleet在中国进行的第一阶段剂量递增研究的初步临床数据。在这项研究中，VS-7375表现出口服生物利用度，在六个剂量水平上没有剂量限制性毒性，以及一些部分反应，包括胰腺癌和肺癌患者。

Enrollment in the Phase 1 dose-escalation cohort is ongoing..

正在进行第一阶段剂量递增队列的登记。。

Key Milestones Expected for 2025:

2025年的关键里程碑：

File an investigational new drug (IND) application in the U.S. for VS-7375 in Q1 2025.

2025年第一季度在美国为VS-7375提交研究性新药（IND）申请。

Initiate a Phase 1/2a trial in the U.S. by mid-2025.

到2025年年中，在美国启动1/2a期试验。

Share preclinical and clinical data from the Phase 1 study of VS-7375 in China in H1 2025.

分享2025年上半年中国VS-7375第一阶段研究的临床前和临床数据。

Discovery/lead optimization continues for the second and third programs in the GenFleet collaboration.

GenFleet协作中的第二和第三个项目继续进行发现/线索优化。

About the Avutometinib and Defactinib ​​Combination

关于Avutometinib和Defactinib的组合

Avutometinib is an oral RAF/MEK clamp that potentially inhibits MEK1/2 kinase activities and induces inactive complexes of MEK with ARAF, BRAF, and CRAF, potentially creating a more complete and durable anti-tumor response through maximal RAS/MAPK pathway inhibition. In contrast to currently available MEK-only inhibitors, avutometinib blocks both MEK kinase activity and the ability of RAF to phosphorylate MEK.

。与目前可用的仅MEK抑制剂相反，avutometinib阻断MEK激酶活性和RAF磷酸化MEK的能力。

This unique mechanism allows avutometinib to block MEK signaling without the compensatory activation of MEK that appears to limit the efficacy of the MEK-only inhibitors..

这种独特的机制允许avutometinib阻断MEK信号传导，而不需要MEK的补偿性激活，这似乎限制了仅MEK抑制剂的功效。。

Defactinib is an oral, selective inhibitor of focal adhesion kinase (FAK) and proline-rich tyrosine kinase-2 (Pyk2), the two members of the focal adhesion kinase family of non-receptor protein tyrosine kinases. FAK and Pyk2 integrate signals from integrin and growth factor receptors to regulate cell proliferation, survival, migration, and invasion.

Defactinib是粘着斑激酶（FAK）和富含脯氨酸的酪氨酸激酶-2（Pyk2）的口服选择性抑制剂，这是非受体蛋白酪氨酸激酶粘着斑激酶家族的两个成员。FAK和Pyk2整合整合素和生长因子受体的信号，以调节细胞增殖，存活，迁移和侵袭。

FAK activation has been shown to mediate resistance to multiple anti-cancer agents, including RAF and MEK inhibitors..

FAK激活已被证明可介导对多种抗癌药物的耐药性，包括RAF和MEK抑制剂。。

Verastem Oncology is currently conducting clinical trials with avutometinib with and without defactinib in RAS/MAPK-driven tumors as part of its

Verastem肿瘤学目前正在进行avutometinib联合或不联合defactinib治疗RAS/MAPK驱动的肿瘤的临床试验，作为其一部分

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rogram or RAMP. Verastem is currently enrolling patients and activating sites for RAMP 301 (GOG-3097/ENGOT-ov81/NCRI) (NCT06072781), an international Phase 3 confirmatory trial evaluating the combination of avutometinib and defactinib versus standard chemotherapy or hormonal therapy for the treatment of recurrent low-grade serous ovarian cancer (LGSOC)..

程序或斜坡。Verastem目前正在招募RAMP 301（GOG-3097/ENGOT-ov81/NCRI）（NCT06072781）的患者和激活位点，这是一项国际3期验证性试验，评估avutometinib和defactinib联合治疗复发性低度浆液性卵巢癌（LGSOC）的标准化疗或激素治疗。。

Verastem was granted Priority Review and a Prescription Drug User Fee Act (PDUFA) date of June 30, 2025, for its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA), for the investigational combination of avutometinib and defactinib in adults with recurrent KRAS mutant LGSOC who received at least one prior systemic therapy.

Verastem于2025年6月30日向美国食品和药物管理局（FDA）提交了新药申请（NDA），并于2025年6月30日获得了优先审查和处方药使用费法案（PDUFA），用于研究avutometinib和defactinib在复发性KRAS突变LGSOC患者中的联合应用，这些患者至少接受过一次全身治疗。

Verastem initiated a rolling NDA in May 2024 to the FDA and completed its NDA submission in October 2024. The FDA granted Breakthrough Therapy Designation for the treatment of patients with recurrent LGSOC after one or more prior lines of therapy, including platinum-based chemotherapy, in May 2021. Avutometinib alone or in combination with defactinib was also granted Orphan Drug Designation by the FDA for the treatment of LGSOC..

Verastem于2024年5月向FDA提交了滚动保密协议，并于2024年10月完成了保密协议的提交。2021年5月，FDA批准了突破性治疗方案，用于治疗一种或多种先前治疗方案（包括铂类化疗）后复发性LGSOC患者。单独使用Avutometinib或与defactinib联合使用也被FDA授予孤儿药名称，用于治疗LGSOC。。

Verastem Oncology has established a clinical collaboration with Amgen to evaluate LUMAKRAS™ (sotorasib) in combination with avutometinib and defactinib in both treatment-naïve patients and in patients whose KRAS G12C mutant non-small cell lung cancer progressed on a G12C inhibitor as part of the RAMP 203 trial (NCT05074810).

Verastem Oncology与Amgen建立了临床合作，以评估LUMAKRAS™（sotorasib）联合avutometinib和defactinib治疗初治患者以及KRAS G12C突变非小细胞肺癌在G12C抑制剂上进展的患者作为RAMP 203试验的一部分（NCT05074810）。

Verastem has received Fast Track Designation from the FDA for the triplet combination in April 2024. RAMP 205 (NCT05669482), a Phase 1b/2 clinical trial evaluating avutometinib and defactinib with gemcitabine/nab-paclitaxel in patients with front-line metastatic pancreatic cancer, is supported by the PanCAN Therapeutic Accelerator Award.

Verastem于2024年4月获得了FDA关于三联组合的快速通道指定。RAMP 205（NCT05669482）是一项1b/2期临床试验，评估阿维替尼和defactinib联合吉西他滨/纳布紫杉醇治疗一线转移性胰腺癌患者，得到了PanCAN治疗加速器奖的支持。

FDA granted Orphan Drug Designation to the avutometinib and defactinib combination for the treatment of pancreatic cancer..

美国食品和药物管理局（FDA）将孤儿药命名为avutometinib和defactinib联合治疗胰腺癌。。

About VS-7375, an Oral KRAS G12D (ON/OFF) Inhibitor

关于VS-7375，一种口服KRAS G12D（开/关）抑制剂

VS-7375 is a potential best-in-class, potent, and selective oral KRAS G12D dual ON/OFF inhibitor. VS-7375 is the lead program from the Verastem Oncology discovery and development collaboration with GenFleet Therapeutics. GenFleet’s IND for VS-7375 (known as GFH375 in China) was approved in China in June 2024, and the first patient was dosed in a Phase 1/2 study in July 2024.

VS-7375是一种潜在的同类最佳，有效且选择性的口服KRAS G12D双重开/关抑制剂。VS-7375是Verastem肿瘤学发现与开发与GenFleet Therapeutics合作的领先项目。GenFleet针对VS-7375（在中国称为GFH375）的IND于2024年6月在中国获得批准，第一名患者于2024年7月在1/2期研究中服用。

Verastem plans to file a U.S. investigational new drug (IND) application for VS-7375 during the first quarter of 2025 and expects to initiate a Phase 1/2a study in mid-2025..

Verastem计划在2025年第一季度提交VS-7375的美国研究性新药（IND）申请，并预计在2025年年中启动1/2a期研究。。

About Verastem Oncology

关于真性肿瘤学

Verastem Oncology (Nasdaq: VSTM) is a late-stage development biopharmaceutical company committed to the development and commercialization of new medicines to improve the lives of patients diagnosed with RAS/MAPK pathway-driven cancers. Our pipeline is focused on novel small molecule drugs that inhibit critical signaling pathways in cancer that promote cancer cell survival and tumor growth, including RAF/MEK inhibition, FAK inhibition and KRAS G12D inhibition.

Verastem Oncology（纳斯达克：VSTM）是一家晚期开发生物制药公司，致力于新药的开发和商业化，以改善被诊断患有RAS/MAPK途径驱动的癌症的患者的生活。我们的管道专注于抑制癌症中促进癌细胞存活和肿瘤生长的关键信号传导途径的新型小分子药物，包括RAF/MEK抑制，FAK抑制和KRAS G12D抑制。

For more information, please visit .

有关更多信息，请访问。

www.verastem.com

www.verastem.com

and follow us on

继续关注我们

LinkedIn

LinkedIn

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Forward-Looking Statements

前瞻性声明

This press release includes forward-looking statements. These forward-looking statements generally can be identified by the use of words such as “anticipate,” “expect,” “plan,” “could,” “may,” “believe,” “estimate,” “forecast,” “goal,” “project,” and other words of similar meaning. Such forward-looking statements address various matters about, among other things, Verastem Oncology’s programs and product candidates, strategy, future plans and prospects, including statements related to the potential for and timing of commercialization of product candidates, the anticipated timing for the IND application for VS-7375/GFH375, the expected outcome and benefits of the Company’s collaboration with GenFleet Therapeutics (Shanghai), Inc., the timing of commencing and completing trials and compiling data, the expected timing of the presentation of data by the Company and the potential clinical value of various of the Company’s clinical trials.

本新闻稿包括前瞻性声明。这些前瞻性陈述通常可以通过使用诸如“预期”、“预期”、“计划”、“可能”、“可能”、“相信”、“估计”、“预测”、“目标”、“项目”和其他类似含义的词语来识别。此类前瞻性声明涉及Verastem Oncology的计划和候选产品、战略、未来计划和前景等方面的各种问题，包括与候选产品商业化的潜力和时间相关的声明，VS-7375/GFH375 IND应用的预期时间，公司与GenFleet Therapeutics（Shanghai），Inc.合作的预期结果和收益，开始和完成试验和汇编数据的时间，公司提交数据的预期时间以及公司各种临床试验的潜在临床价值。

Each forward-looking statement contained in this press release is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statement. Applicable risks and uncertainties include, among others: the uncertainties inherent in research and development, such as negative or unexpected results of clinical trials; that we may not see a return on investment on the payments we have and may continue to make pursuant to the collaboration and option agreement with GenFleet, or that GenFleet may fail to fully perform under the agreement; that the development and commercialization of our product candidates may take longer or cost more than planned, including as a result of conducting additional studies or our decisions regarding execution of such commercialization; that data may not be a.

本新闻稿中的每一项前瞻性声明都存在风险和不确定性，这些风险和不确定性可能导致实际结果与该声明中明示或暗示的结果存在重大差异。适用的风险和不确定性包括：研究和开发中固有的不确定性，例如临床试验的负面或意外结果；根据与GenFleet签订的合作与期权协议，我们可能不会看到我们已经支付的款项的投资回报，也可能会继续支付，或者GenFleet可能无法完全履行协议；我们候选产品的开发和商业化可能比计划的时间更长或成本更高，包括由于进行额外的研究或我们关于执行此类商业化的决定；该数据可能不是a。

www.sec.gov

www.sec.gov

, for a discussion of these and other risks and uncertainties. The forward-looking statements in this press release speak only as of the date of this press release, and we undertake no obligation to update or revise any of these statements. Our business is subject to substantial risks and uncertainties, including those referenced above.

，以讨论这些以及其他风险和不确定性。本新闻稿中的前瞻性声明仅在本新闻稿发布之日起生效，我们没有义务更新或修订任何此类声明。我们的业务面临重大风险和不确定性，包括上述风险和不确定性。

Investors, potential investors, and others should give careful consideration to these risks and uncertainties..

投资者、潜在投资者和其他人应仔细考虑这些风险和不确定性。。