Data demonstrated statistically significant and clinically meaningful reduction in risk of disease progression or death with Cabometyx® (cabozantinib) versus placebo in advanced pancreatic and extra-pancreatic neuroendocrine tumors (NETs)1,2

数据显示，在晚期胰腺和胰腺外神经内分泌肿瘤（NETs）中，Cabometyx®（cabozantinib）与安慰剂相比，在统计学上显着且具有临床意义的疾病进展或死亡风险降低1,2

Data presented at ESMO 2024 and published in New England Journal of Medicine

数据于2024年在ESMO上发布，并发表在《新英格兰医学杂志》上

Ipsen has submitted an extension of indication Marketing Authorization to the European Medicines Agency

Ipsen已向欧洲药品管理局提交了适应症营销授权的延期

Limited approved treatment options for advanced NETs dependent on primary site of disease, with no approved therapies in lung NETs upon progression after prior systemic therapy3,4

对于依赖于疾病原发部位的晚期NETs，批准的治疗选择有限，在先前的全身治疗后进展的肺NETs中没有批准的治疗方法3,4

PARIS, FRANCE, 16 September 2024 – Ipsen (Euronext: IPN; ADR: IPSEY) announced today final data from the CABINET Phase III trial investigating Cabometyx® (cabozantinib) versus placebo in people living with advanced pancreatic neuroendocrine tumors (pNETs) or advanced extra-pancreatic neuroendocrine tumors (epNETs) whose disease had progressed after prior systemic therapy.

2024年9月16日，法国巴黎–Ipsen（Euronext:IPN；ADR:IPSEY）今天宣布了Cabometyx®（cabozantinib）与安慰剂治疗晚期胰腺神经内分泌肿瘤（pNETs）或晚期胰腺外神经内分泌肿瘤（epNETs）患者的内阁III期临床试验的最终数据，这些患者的疾病在先前的全身治疗后有所进展。

These data demonstrated a statistically significant reduction in the risk of disease progression or death for Cabometyx versus placebo of 77% (hazard ratio (HR) 0.23) and 62% (HR 0.38) for people living with advanced pNETs and epNETs, respectively.1,2 Presentation of these data is taking place today at the 2024 European Society for Medical Oncology Congress (ESMO 2024) during the Proffered Paper Session: NETs and Endocrine Tumors at 2:45 p.m.

这些数据表明，对于晚期PNET和ePNET患者，Cabometyx与安慰剂相比，疾病进展或死亡风险分别降低了77%（风险比（HR）0.23）和62%（HR 0.38）[1,2]。这些数据的介绍今天在2024年欧洲医学肿瘤学会大会（ESMO 2024）上进行，在下午2:45举行的论文会议：NETs和内分泌肿瘤。

CEST, and is published in the New England Journal of Medicine (NEJM)..

CEST，并发表在《新英格兰医学杂志》（NEJM）上。。

“People living with neuroendocrine tumors face many challenges, from securing a timely diagnosis to optimal treatment options which address the needs of the increasing number of people affected by this cancer worldwide,” said Teodora Kolarova, Executive Director, International Neuroendocrine Cancer Alliance.

国际神经内分泌癌症联盟执行主任特奥多拉·科拉罗娃（TeodoraKolarova）说：“患有神经内分泌肿瘤的人面临着许多挑战，从确保及时诊断到最佳治疗选择，以满足全球越来越多受这种癌症影响的人的需求。”。

“These latest data reaffirm the possibilities of continuing scientific advancements in neuroendocrine tumors, offering the potential for new therapies which could significantly impact people’s everyday lives as they navigate this complex and life altering diagnosis.”.

“这些最新数据重申了神经内分泌肿瘤持续科学进步的可能性，为新疗法提供了潜力，这些疗法可能会对人们的日常生活产生重大影响，因为他们可以驾驭这种复杂且改变生活的诊断。”。

Final results demonstrated progression-free survival (PFS) benefits in favor of Cabometyx versus placebo by blinded independent central review (BICR).1,2 In the pNET cohort, at a median follow-up of 13.8 months, median PFS was 13.8 months for Cabometyx versus 4.4 months for placebo (HR 0.23 [95% confidence interval (CI) 0.12-0.42] p<0.0001).1,2 In the epNET cohort, at a median follow-up of 10.2 months, median PFS was 8.4 months for Cabometyx versus 3.9 months for placebo (HR 0.38 [95% CI 0.25-0.59] p<0.0001).1,2 The safety profile of Cabometyx observed in each cohort was consistent with its known safety profile; no new safety signals were identified.1,2.

1,2在pNET队列中，中位随访时间为13.8个月，Cabometyx的中位PFS为13.8个月，安慰剂组为4.4个月（HR 0.23[95%置信区间（CI）0.12-0.42]p<0.0001）。1,2在epNET队列中，中位随访时间为10.2个月，Cabometyx的中位PFS为8.4个月，安慰剂组为3.9个月（HR 0.38[95%CI 0.25-0.59]p＜0.0001）。1,2在每个队列中观察到的Cabometyx的安全性与其已知的安全性一致；没有发现新的安全信号。

“These latest data reinforce the potential of Cabometyx to deliver significant efficacy benefits at an advanced stage of disease,” said Christelle Huguet, EVP and Head of Research and Development at Ipsen. “Through our submission to the EMA, it is our ambition to evolve the treatment paradigm for people living with neuroendocrine tumors, harnessing our longstanding heritage in this area to deliver an effective new therapy where options are notably limited.”.

“这些最新数据增强了Cabometyx在疾病晚期提供显着疗效益处的潜力，”Ipsen研究与开发主管兼执行副总裁ChristelleHuguet说。“通过我们提交给EMA，我们的目标是发展神经内分泌肿瘤患者的治疗模式，利用我们在这一领域的长期遗产，在选择明显有限的情况下提供有效的新疗法。”。

The number of people newly diagnosed with NETs is believed to be rising due to increasing awareness and better methods of diagnosis, with approximately 35 in every 100,000 people currently living with NETs globally.5,6 However, despite increasing awareness, the non-specific nature of NET symptoms often leads patients to be seen by multiple specialists and to undergo various forms of testing before an accurate diagnosis is achieved.5 As a result, almost a third of people take at least five years to be diagnosed with NETs, contributing to poorer patient outcomes.5 Most forms of NETs are indolent in nature and can develop in any part of the body,7 requiring multiple lines of therapy as people progress.3,4 Treatment options upon progression are often limited dependent on primary site of disease, resulting in challenges in identifying optimal care pathways specific to patients’ circumstances.3,4,8.

据信，由于认识的提高和更好的诊断方法，新诊断出患有NETs的人数正在增加，目前全球每10万人中约有35人患有NETs。5,6然而，尽管认识有所提高，但NETs症状的非特异性常常导致患者被多名专家看到，并在获得准确诊断之前接受各种形式的检测。因此，几乎三分之一的人至少需要五年才能被诊断出患有NETs，导致患者预后较差。5大多数形式的NETs本质上是惰性的，可以在身体的任何部位发展，7随着人们的进步，需要多种治疗方案。3,4进展后的治疗选择通常有限，取决于疾病的原发部位因此，在确定针对患者情况的最佳护理途径方面存在挑战。3,4,8。

About Cabometyx

关于Cabometyx

Cabometyx (cabozantinib) is a small molecule that inhibits multiple receptor tyrosine kinases, including VEGFRs, MET, RET and the TAM family (TYRO3, MER, AXL).9 These receptor tyrosine kinases are involved in both normal cellular function and pathologic processes such as oncogenesis, metastasis, tumor angiogenesis (the growth of new blood vessels that tumors need to grow), drug resistance, modulation of immune activities and maintenance of the tumor microenvironment.9,10,11,12.

Cabometyx（cabozantinib）是一种抑制多种受体酪氨酸激酶的小分子，包括VEGFRs，MET，RET和TAM家族（TYRO3，MER，AXL）[9]。这些受体酪氨酸激酶参与正常细胞功能和病理过程，如肿瘤发生，转移，肿瘤血管生成（肿瘤需要生长的新血管的生长），耐药性，免疫活性的调节和肿瘤微环境的维持[9,10,11,12]。

Exelixis granted Ipsen exclusive rights for the commercialization and further clinical development of Cabometyx outside of the U.S. and Japan. Exelixis granted exclusive rights to Takeda Pharmaceutical Company Limited (Takeda) for the commercialization and further clinical development of Cabometyx for all future indications in Japan.

Exelixis授予Ipsen在美国和日本以外的Cabometyx商业化和进一步临床开发的专有权。Exelixis授予武田制药有限公司（武田）专有权，用于Cabometyx在日本所有未来适应症的商业化和进一步临床开发。

Exelixis holds the exclusive rights to develop and commercialize Cabometyx in the U.S..

Exelixis拥有在美国开发和商业化Cabometyx的独家权利。。

In over 65 countries outside of the United States and Japan, including in the European Union, Cabometyx is currently indicated as:10

在美国和日本以外的65多个国家，包括欧盟，Cabometyx目前表示为：10

Monotherapy for advanced renal cell carcinoma (aRCC).

单药治疗晚期肾细胞癌（aRCC）。

as first-line treatment of adults with intermediate- or poor-risk disease.

作为中危或低危成年人的一线治疗。

in adults following prior VEGFR-targeted therapy.

先前接受VEGFR靶向治疗的成年人。

A combination with nivolumab for the first-line treatment of aRCC in adults.

与nivolumab联合用于成人aRCC的一线治疗。

Monotherapy for the treatment of adults living with locally advanced or metastatic differentiated thyroid carcinoma, refractory or not eligible to radioactive iodine who have progressed during or after prior systemic therapy.

用于治疗在先前全身治疗期间或之后进展的局部晚期或转移性分化型甲状腺癌，难治性或不符合放射性碘条件的成年人的单一疗法。

Monotherapy for the treatment of hepatocellular carcinoma in adults who have previously been treated with sorafenib.

单药治疗先前接受索拉非尼治疗的成人肝细胞癌。

About neuroendocrine tumors

关于神经内分泌肿瘤

NETs are relatively uncommon and develop from cells of the neuroendocrine system; thus, can arise from a variety of locations throughout the body.5,7 The most common sites of NETs include the gastrointestinal (GI) tract, lungs and pancreas.7,13 Most NETs take years to develop and grow slowly, however some NETs can be fast-growing.7 The five-year survival rate is dependent on the primary site of disease.

NETs相对不常见，由神经内分泌系统的细胞发育而来；因此，NETs可能来自全身的各种部位[5,7]。NETs最常见的部位包括胃肠道（GI），肺和胰腺[7,13]。大多数NETs需要数年才能发育并缓慢生长，但一些NETs可能会快速增长[7]。五年生存率取决于疾病的原发部位。

For advanced GI-NET and lung NETs, where the cancer has spread to distant parts of the body, the five-year survival rates are 68% and 55%, respectively.14,15 For people diagnosed with advanced pNET, however, the prognosis is poor, with a five-year survival rate of 23%.16.

对于癌症扩散到身体远端的晚期GI-NET和肺NET，五年生存率分别为68%和55%[14,15]。然而，对于诊断为晚期pNET的患者，预后较差，五年生存率为23%[16]。

About CABINET (Alliance A021602)

关于内阁（联盟A021602）

CABINET (randomized, double-blinded Phase III trial of CABozantinib versus placebo In patients with advanced NEuroendocrine Tumors after progression on prior therapy) is sponsored by the National Cancer Institute (NCI), part of the National Institutes of Health, and is being led and conducted by the NCI-funded Alliance for Clinical Trials in Oncology with participation from the NCI-funded National Clinical Trials Network, as part of Exelixis’ collaboration through a Cooperative Research and Development Agreement with the NCI’s Cancer Therapy Evaluation Program..

CABINET（卡博替尼与安慰剂治疗晚期神经内分泌肿瘤患者先前治疗进展后的随机双盲III期临床试验）由美国国立卫生研究院（National Institutes of Health）下属的国家癌症研究所（National Cancer Institute，NCI）赞助，由NCI资助的肿瘤学临床试验联盟（Alliance for Clinical Trials In Oncology）领导和进行，由NCI资助的国家临床试验网络（National Clinical Trials Network）参与，作为Exelixis通过与NCI癌症治疗评估计划的合作研发协议的一部分。。

The multicenter, Phase III CABINET pivotal trial enrolled a total of 298 patients in the US at the time of the final analysis. Patients were randomized 2:1 to Cabometyx or placebo in two separately powered cohorts (pNET, n=95; epNET, n=203). The epNET cohort included patients with the following primary tumor sites: gastrointestinal tract, lung, unknown primary sites and other.

在最终分析时，这项多中心的III期内阁关键试验在美国共招募了298名患者。患者以2:1的比例随机分为两组（pNET，n=95；epNET，n=203）。epNET队列包括具有以下原发肿瘤部位的患者：胃肠道，肺，未知原发部位和其他。

Each cohort was randomized separately and had its own statistical analysis plan. Patients must have had measurable disease per RECIST 1.1 criteria and must have experienced disease progression or intolerance after at least one U.S. Food and Drug Administration-approved line of prior therapy other than somatostatin analogues.

每个队列分别随机分组，并有自己的统计分析计划。根据RECIST 1.1标准，患者必须患有可测量的疾病，并且必须在至少一种美国食品和药物管理局批准的生长抑素类似物以外的先前治疗方案后经历疾病进展或不耐受。

The primary endpoint in each cohort was PFS per RECIST 1.1 by retrospective independent central review. Upon confirmation of disease progression, patients were unblinded, and those receiving placebo were permitted to cross over to open-label therapy with Cabometyx. Secondary endpoints included overall survival, radiographic response rate and safety.

通过回顾性独立中央审查，每个队列的主要终点是每个RECIST 1.1的PFS。在确认疾病进展后，患者被揭盲，接受安慰剂的患者被允许交叉使用Cabometyx进行开放标签治疗。次要终点包括总生存率，放射学缓解率和安全性。

More information about this trial is available at ClinicalTrials.gov..

有关该试验的更多信息，请访问ClinicalTrials.gov。。

About Ipsen

关于益普生

We are a global biopharmaceutical company with a focus on bringing transformative medicines to patients in three therapeutic areas: Oncology, Rare Disease and Neuroscience.

我们是一家全球性的生物制药公司，专注于在三个治疗领域为患者带来变革性药物：肿瘤学，罕见病和神经科学。

Our pipeline is fueled by external innovation and supported by nearly 100 years of development experience and global hubs in the U.S., France and the U.K. Our teams in more than 40 countries and our partnerships around the world enable us to bring medicines to patients in more than 80 countries.

我们的渠道由外部创新推动，并由近100年的发展经验和美国、法国和英国的全球中心提供支持。我们在40多个国家的团队和我们在世界各地的合作伙伴使我们能够为80多个国家的患者提供药物。

Ipsen is listed in Paris (Euronext: IPN) and in the U.S. through a Sponsored Level I American Depositary Receipt program (ADR: IPSEY). For more information, visit ipsen.com.

Ipsen通过赞助的一级美国存托凭证计划（ADR：IPSEY）在巴黎（泛欧交易所：IPN）和美国上市。有关更多信息，请访问ipsen.com。

Ipsen contacts

Ipsen联系人

Investors

投资者

Craig Marks | +44 7584 349 193

克雷格马克|+44 7584 349 193

Media

媒体

Amy Wolf | +41 7 95 76 07 23

艾米·沃尔夫|+41 7 95 76 07 23

Emma Roper | +44 7711 766 517

艾玛·罗珀+44 7711 766 517