BOSTON--(BUSINESS WIRE)--Verastem Oncology (Nasdaq: VSTM) (the “Company”), a biopharmaceutical company committed to advancing new medicines for patients with cancer, announced today that it has initiated its international confirmatory Phase 3 RAMP 301 trial (GOG-3097; ENGOT-ov81/NCRI), evaluating the combination of avutometinib and defactinib versus standard chemotherapy or hormonal therapy for the treatment of recurrent low-grade serous ovarian cancer (LGSOC)..

波士顿--（商业新闻短讯）--致力于为癌症患者开发新药的生物制药公司Verastem Oncology（Nasdaq:VSTM）（“公司”）今天宣布，它已启动其国际验证性3期RAMP 301试验（GOG-3097；ENGOT-ov81/NCRI），评估avutometinib和defactinib联合治疗复发性低度浆液性卵巢癌（LGSOC）与标准化疗或激素治疗的比较。。

“Patients and treating physicians have advocated for more research and development in support of LGSOC. At Verastem Oncology, we are moving with urgency to offer a Phase 3 study specifically directed at this disease in an effort to address this need,” said Dan Paterson, President and Chief Executive Officer, Verastem Oncology.

Verastem Oncology总裁兼首席执行官丹·帕特森（DanPaterson）表示：“患者和主治医师都主张进行更多的研究和开发，以支持LGSOC。在Verastem Oncology，我们迫切需要提供一项专门针对这种疾病的3期研究，以满足这一需求。”。

“Based on our Breakthrough Therapy Designation, the initiation and expected progress of this trial, along with the FRAME study data and mature RAMP 201 data, we plan to file for Accelerated Approval in the first half of next year, moving us a significant step closer to addressing the treatment needs of patients living with LGSOC.”.

“根据我们的突破性治疗指定，该试验的启动和预期进展，以及框架研究数据和成熟的RAMP 201数据，我们计划在明年上半年提交加速批准，使我们在解决LGSOC患者的治疗需求方面迈出了重要的一步。”。

RAMP 301 is the confirmatory study required by the U.S. Food and Drug Administration (FDA) for the combination of avutometinib and defactinib to potentially receive full approval for the treatment of recurrent LGSOC. The Company intends to submit an Accelerated Approval New Drug Application (NDA) for the combination of avutometinib and defactinib based on mature data from the Company’s Phase 2 registration-directed RAMP 201 trial, together with the results of the investigator-initiated FRAME trial.

RAMP 301是美国食品和药物管理局（FDA）要求的验证性研究，用于联合avutometinib和defactinib治疗复发性LGSOC。该公司打算根据该公司第二阶段注册指导的RAMP 201试验的成熟数据，以及研究者发起的框架试验的结果，提交一份关于avutometinib和defactinib联合使用的加速批准新药申请（NDA）。

The company recently reported results of Part A of the RAMP 201 trial, including confirmed objective response rates (ORR) by blinded independent central review of 45% with a response rate and safety profile consistent with previous studies.​.

该公司最近报告了RAMP 201试验A部分的结果，包括通过盲法独立中央审查确认的客观缓解率（ORR）为45%，缓解率和安全性与之前的研究一致。​.

“LGSOC has a unique molecular, histologic, and clinical profile that differs dramatically from the most common type of ovarian cancer. Response rates to standard of care treatments are disappointing, and there are still no FDA approved treatments specifically for LGSOC,” said Rachel Grisham, M.D., Section Head, Ovarian Cancer and Director, Gynecologic Medical Oncology at Memorial Sloan Kettering Cancer Center in Westchester, NY and RAMP 301 global lead investigator.

“LGSOC具有独特的分子，组织学和临床特征，与最常见的卵巢癌类型有很大不同。对标准治疗的反应率令人失望，仍然没有FDA批准的专门针对LGSOC的治疗方法，”卵巢癌科主任兼主任Rachel Grisham医学博士说，纽约州西切斯特纪念斯隆·凯特琳癌症中心的妇科肿瘤学和RAMP 301全球首席研究员。

“The combination of avutometinib and defactinib continues to show promise in recurrent LGSOC, and I am looking forward to leading this confirmatory trial with the goal of establishing a new standard of care for people with this rare form of ovarian cancer.”.

“avutometinib和defactinib的组合在复发性LGSOC中继续显示出前景，我期待着领导这项验证性试验，目的是为这种罕见形式的卵巢癌患者建立新的护理标准。”。

According to Professor Susana Banerjee, MBBS, MA, PhD, FRCP, Consultant Medical Oncologist and Research Lead for the Gynaecology Unit at The Royal Marsden NHS Foundation Trust, Team Leader in Women's Cancers at The Institute of Cancer Research, London, and lead European investigator of the RAMP 301 trial, “Based on my experience treating women with LGSOC, it’s clear that we need better therapeutic options.

据皇家马斯登NHS基金会信托基金会妇科科顾问医学肿瘤学家和研究负责人、伦敦癌症研究所女性癌症团队负责人、RAMP 301试验欧洲首席研究员Susana Banerjee教授介绍，“根据我治疗LGSOC女性的经验，很明显我们需要更好的治疗选择。

I am pleased this Phase 3 trial, following the initial positive results from the Phase 2 RAMP 201 trial, is enrolling patients to potentially address the significant limitations we have seen with other available therapies.”.

我很高兴这项第三阶段试验在第二阶段RAMP 201试验的初步阳性结果之后，正在招募患者，以潜在地解决我们在其他可用疗法中看到的重大局限性。”。

RAMP 301 (GOG-3097; ENGOT-ov81/NCRI) is an international collaboration between The GOG Foundation, Inc. (GOG) and the European Network of Gynaecological Oncological Trial groups (ENGOT) sponsored by Verastem Oncology. The trial is expected to enroll 270 patients who will be randomized to either the combination of avutometinib and defactinib or investigator’s choice chemotherapy (pegylated liposomal doxorubicin, paclitaxel, topotecan) or hormone therapy (letrozole, anastrozole).

RAMP 301（GOG-3097；ENGOT-ov81/NCRI）是GOG基金会（GOG）和Verastem Oncology赞助的欧洲妇科肿瘤试验组网络（ENGOT）之间的国际合作。该试验预计将招募270名患者，他们将被随机分配到avutometinib和defactinib的组合或研究者选择的化疗（聚乙二醇化脂质体多柔比星，紫杉醇，拓扑替康）或激素治疗（来曲唑，阿那曲唑）。

The primary endpoint is progression free survival (PFS) by Blinded Independent Central Review. Secondary endpoints include ORR, duration of response​, disease control rate, safety and tolerability, patient reported outcomes, and overall survival. RAMP 301 is a global trial with enrollment open in the U.S.

主要终点是通过盲法独立中央评估的无进展生存期（PFS）。次要终点包括ORR，反应持续时间​,疾病控制率，安全性和耐受性，患者报告的结果和总体生存率。RAMP 301是一项全球试验，在美国开放注册。

and planned enrollment in Canada, the United Kingdom, Europe, Australia, and Korea..

并计划在加拿大、英国、欧洲、澳大利亚和韩国注册。。

Dr. Grisham and Dr. Banerjee are paid consultants for Verastem Oncology.

Grisham博士和Banerjee博士是Verastem肿瘤学的有偿顾问。

About the Avutometinib and Defactinib Combination

关于Avutometinib和Defactinib的组合

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

Avutometinib是一种RAF/MEK钳，可诱导MEK与ARAF，BRAF和CRAF的无活性复合物，从而通过最大程度的RAS途径抑制产生更完整和持久的抗肿瘤反应。与目前可用的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 other inhibitors. The U.S. Food and Drug Administration granted Breakthrough Therapy designation for the combination of Verastem Oncology’s investigational RAF/MEK clamp avutometinib, with defactinib, its FAK inhibitor, for the treatment of all patients with recurrent LGSOC regardless of KRAS status after one or more prior lines of therapy, including platinum-based chemotherapy..

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，这似乎限制了其他抑制剂的功效。美国食品和药物管理局批准将Verastem Oncology的研究性RAF/MEK钳夹avutometinib与其FAK抑制剂defactinib联合用于治疗所有复发性LGSOC患者，无论其在一种或多种先前治疗方案（包括铂类化疗）后的KRAS状态如何。。

Verastem Oncology is currently conducting clinical trials with its RAF/MEK clamp avutometinib in RAS pathway-driven tumors as part of its (Raf And Mek Program). RAMP 301 is a Phase 3 confirmatory trial evaluating the combination of avutometinib and defactinib versus standard chemotherapy or hormonal therapy for the treatment of recurrent LGSOC.

作为its（RAF和MEK计划）的一部分，Verastem Oncology目前正在RAS途径驱动的肿瘤中使用其RAF/MEK钳avutometinib进行临床试验。RAMP 301是一项3期验证性试验，评估avutometinib和defactinib联合治疗与标准化疗或激素治疗治疗复发性LGSOC的疗效。

RAMP 201 is a Phase 2 registration-directed trial of avutometinib in combination with defactinib in patients with recurrent LGSOC and has completed enrollment in the dose optimization and expansion phases and is enrolling for low-dose evaluation. Verastem Oncology has established clinical collaborations with Amgen and Mirati to evaluate LUMAKRAS™ (sotorasib) and KRAZATI™ (adagrasib) in combination with avutometinib in KRAS G12C mutant NSCLC as part of the RAMP 203 and RAMP 204 trials, respectively.

RAMP 201是avutometinib联合defactinib治疗复发性LGSOC患者的2期登记指导试验，已完成剂量优化和扩大阶段的登记，并正在进行低剂量评估。Verastem肿瘤学与Amgen和Mirati建立了临床合作，以评估LUMAKRAS™（sotorasib）和KRAZATI™（adagrasib）与avutometinib联合用于KRAS G12C突变NSCLC，分别作为RAMP 203和RAMP 204试验的一部分。

Supported by the “Therapeutic Accelerator Award” Verastem Oncology received from PanCAN, the Company is conducting RAMP 205, a Phase 1b/2 clinical trial evaluating avutometinib and defactinib with gemcitabine/nab-paclitaxel in patients with front-line metastatic pancreatic cancer..

在PanCAN获得的“治疗加速器奖”Verastem Oncology的支持下，该公司正在进行RAMP 205，这是一项1b/2期临床试验，评估阿维托替尼和defactinib与吉西他滨/纳布-紫杉醇联合治疗一线转移性胰腺癌患者。。

About Low-Grade Serous Ovarian Cancer (LGSOC)

关于低度浆液性卵巢癌（LGSOC）

LGSOC is a highly recurrent, chemotherapy-resistant cancer, associated with slow tumor growth and high mortality rate. Approximately 6,000 women in the U.S. and 80,000 worldwide are living with this disease. Mutations in the KRAS gene are present in 35-57% cases of LGSOC. LGSOC is most often diagnosed in women between the ages of 45-55 years and has a median survival of approximately ten years.

LGSOC是一种高度复发的化疗耐药癌症，与肿瘤生长缓慢和死亡率高有关。美国大约6000名女性和全世界80000名女性患有这种疾病。35-57%的LGSOC病例中存在KRAS基因突变。LGSOC最常被诊断为年龄在45-55岁之间的女性，中位生存期约为10年。

The majority of patients experience severe pain and complications as the disease progresses. Chemotherapy is the standard of care for this disease, with limited treatment options currently available..

随着疾病的进展，大多数患者会经历严重的疼痛和并发症。化疗是这种疾病的标准治疗方法，目前可用的治疗选择有限。。

About Verastem Oncology

关于真性肿瘤学

Verastem Oncology (Nasdaq: VSTM) is a development-stage biopharmaceutical company committed to the development and commercialization of new medicines to improve the lives of patients diagnosed with cancer. 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 and focal adhesion kinase (FAK) inhibition.

Verastem Oncology（纳斯达克股票代码：VSTM）是一家处于发展阶段的生物制药公司，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抑制和粘着斑激酶（FAK）抑制。

For more information, please visit www.verastem.com..

欲了解更多信息，请访问www.verastem.com。。

About The GOG Foundation, Inc. (www.gog.org)

关于GOG基金会（www.GOG.org）

The GOG Foundation, Inc. is a not-for-profit organization with the purpose of promoting excellence in the quality and integrity of clinical and translational scientific research in the field of gynecologic malignancies. The GOG Foundation is committed to maintaining the highest standards in clinical trials development, execution, analysis, and distribution of results.

GOG基金会是一家非营利性组织，旨在促进妇科恶性肿瘤领域临床和转化科学研究的质量和完整性。GOG基金会致力于在临床试验开发，执行，分析和结果分发方面保持最高标准。

The GOG Foundation is the only clinical trialist group in the United States that focuses its research on patients with pelvic malignancies, such as cancer of the ovary (including surface peritoneal malignancies), uterus (including endometrium, soft tissue sarcoma, and gestational trophoblastic neoplasia), cervix, and vulva.

GOG基金会是美国唯一一个将研究重点放在盆腔恶性肿瘤患者上的临床试验组，例如卵巢癌（包括表面腹膜恶性肿瘤），子宫（包括子宫内膜，软组织肉瘤和妊娠滋养细胞肿瘤），子宫颈和外阴。

The GOG Foundation is multi-disciplinary in its approach to clinical trials, and includes gynecologic oncologists, medical oncologists, pathologists, radiation oncologists, oncology nurses, biostatisticians (including those with expertise in bioinformatics), basic scientists, quality of life experts, data managers, and administrative personnel..

GOG基金会的临床试验方法是多学科的，包括妇科肿瘤学家、医学肿瘤学家、病理学家、放射肿瘤学家、肿瘤护士、生物统计学家（包括具有生物信息学专业知识的人）、基础科学家、生活质量专家、数据管理人员和行政人员。。

About the GOG Partners Program

关于GOG合作伙伴计划

Supported by industry, GOG Partners program is structured to work directly with pharmaceutical organizations and operate clinical trials outside the National Cancer Institute (NCI) framework. The GOG Partners program promotes the mission of the GOG Foundation dedicated to transforming the standard of care in Gynecologic Oncology.

在业界的支持下，GOG合作伙伴计划的结构是直接与制药组织合作，并在国家癌症研究所（NCI）框架之外进行临床试验。GOG合作伙伴计划促进了GOG基金会致力于改变妇科肿瘤学护理标准的使命。

By providing an alternative venue for patient accrual and site infrastructure support, GOG Partners has helped provide additional trials and opportunities for patients outside the national gynecologic clinical trials network..

通过为患者应计和现场基础设施支持提供替代场所，GOG合作伙伴帮助为国家妇科临床试验网络之外的患者提供了额外的试验和机会。。

About ENGOT (www.engot.esgo.org)

关于Engot（www.engot.esgo.org）

The European Network for Gynaecological Oncological Trial (ENGOT) groups is a research network of the European Society of Gynaecological Oncology and was founded in Berlin in October 2007. Currently, ENGOT consists of 21 trial groups from 31 European countries that perform cooperative clinical trials.

欧洲妇科肿瘤试验网络（ENGOT）小组是欧洲妇科肿瘤学会的一个研究网络，于2007年10月在柏林成立。目前，ENGOT由来自31个欧洲国家的21个试验组组成，这些试验组进行合作临床试验。

ENGOT’s ultimate goal is to bring the best treatment to gynecological cancer patients through the best science and enabling every patient in every European country to access a clinical trial..

ENGOT的最终目标是通过最好的科学为妇科癌症患者带来最好的治疗，并使每个欧洲国家的每位患者都能获得临床试验。。

Forward-Looking Statements Notice

前瞻性声明通知

This press release includes forward-looking statements about Verastem Oncology’s strategy, future plans and prospects, including statements related to the potential clinical value of various of its clinical trials and the timing of commencing and completing trials and regulatory submissions. The words 'anticipate,' 'believe,' 'estimate,' 'expect,' 'intend,' 'may,' 'plan,' 'predict,' 'project,' 'target,' 'potential,' 'will,' 'would,' 'could,' 'should,' 'continue,' “can,” “promising” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.

本新闻稿包括关于Verastem Oncology的战略，未来计划和前景的前瞻性声明，包括与各种临床试验的潜在临床价值以及开始和完成试验和监管提交的时间有关的声明。“预期”，“相信”，“估计”，“期望”，“打算”，“可能”，“计划”，“预测”，“项目”，“目标”，“潜力”，“意志”，“会”，“可能”，“应该”，“继续”，“可以”，“有希望”和类似的表达旨在识别前瞻性陈述，尽管并非所有前瞻性陈述都包含这些识别词。

Each forward-looking statement is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement..

每个前瞻性声明都有可能导致实际结果与该声明中明示或暗示的结果产生重大差异的风险和不确定性。。

Applicable risks and uncertainties include the risks and uncertainties, among other things, regarding: the success in the development and potential commercialization of our product candidates, including avutometinib in combination with other compounds, including defactinib, LUMAKRAS® and others; the uncertainties inherent in research and development, such as negative or unexpected results of clinical trials, the occurrence or timing of applications for our product candidates that may be filed with regulatory authorities in any jurisdictions; whether and when regulatory authorities in any jurisdictions may approve any such applications that may be filed for our product candidates, and, if approved, whether our product candidates will be commercially successful in such jurisdictions; our ability to obtain, maintain and enforce patent and other intellectual property protection for our product candidates; the scope, timing, and outcome of any legal proceedings; decisions by regulatory authorities regarding labeling and other matters that could affect the availability or commercial potential of our product candidates; whether preclinical testing of our product candidates and preliminary or interim data from clinical trials will be predictive of the results or success of ongoing or later clinical trials; that the timing, scope and rate of reimbursement for our product candidates is uncertain; that third- party payors (including government agencies) may not reimburse; that there may be competitive developments affecting our product candidates; that data may not be available when expected; that enrollment of clinical trials may take longer than expected; that our product candidates will cause adverse safety events and/or unexpected concerns may arise from additional data or ana.

适用的风险和不确定性包括以下方面的风险和不确定性：我们的候选产品（包括avutometinib与其他化合物（包括defactinib，LUMAKRAS®等）的开发成功和潜在商业化；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，可能向任何司法管辖区的监管机构提交的候选产品申请的发生或时间；任何司法管辖区的监管机构是否以及何时可以批准可能为我们的候选产品提交的任何此类申请，如果获得批准，我们的候选产品是否会在这些司法管辖区取得商业成功；我们为候选产品获得、维护和实施专利和其他知识产权保护的能力；任何法律诉讼的范围、时间和结果；监管机构关于标签和其他可能影响我们候选产品可用性或商业潜力的事项的决定；我们的候选产品的临床前测试以及临床试验的初步或中期数据是否可以预测正在进行或以后的临床试验的结果或成功；我们候选产品的报销时间、范围和费率不确定；第三方付款人（包括政府机构）可能无法报销；可能有竞争发展影响我们的候选产品；该数据可能在预期时不可用；临床试验的注册可能需要比预期更长的时间；我们的候选产品可能会导致不良安全事件和/或意外问题，这可能来自其他数据或ana。

Other risks and uncertainties include those identified under the heading “Risk Factors” in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022 as filed with the Securities and Exchange Commission (SEC) on March 14, 2023 and in any subsequent filings with the SEC. The forward-looking statements contained in this press release reflect Verastem Oncology’s views as of the date hereof, and the Company does not assume and specifically disclaims any obligation to update any forward-looking statements whether as a result of new information, future events or otherwise, except as required by law..

其他风险和不确定性包括2023年3月14日提交给美国证券交易委员会（SEC）的截至2022年12月31日的公司年度报告表格10-K中“风险因素”标题下确定的风险和不确定性，以及随后提交给SEC的任何文件中确定的风险和不确定性。本新闻稿中包含的前瞻性声明反映了Verastem Oncology截至本新闻稿发布之日的观点，除法律要求外，公司不承担并明确否认因新信息、未来事件或其他原因而更新任何前瞻性声明的义务。。