辉瑞公司提供了癌症恶病质患者Ponsegromab 2期研究的阳性数据-动脉网

Pfizer Presents Positive Data from Phase 2 Study of Ponsegromab in Patients with Cancer Cachexia

辉瑞等信源发布 2024-09-14 21:31

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Study met primary endpoint of change from baseline in body weight for ponsegromab compared to placebo across all ponsegromab doses tested, reaching 5.6% mean increase at the highest dose evaluated at 12 weeks; ponsegromab was generally considered safe and well-tolerated at all dose levels.

在所有测试的ponsegromab剂量中，与安慰剂相比，研究达到了ponsegromab体重从基线变化的主要终点，在12周时评估的最高剂量下达到5.6%的平均增加；在所有剂量水平下，ponsegromab通常被认为是安全且耐受性良好的。

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At the highest dose evaluated, improvements were seen from baseline in appetite and cachexia symptoms, physical activity, and muscle mass

在评估的最高剂量下，食欲和恶病质症状、体力活动和肌肉质量均较基线有所改善

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Based on positive Phase 2 results, registration-enabling studies will start in 2025

根据第二阶段的积极成果，登记启用研究将于2025年开始

NEW YORK--(BUSINESS WIRE)--

纽约--（商业新闻）--

Pfizer Inc.

辉瑞公司。

(NYSE: PFE) today announced its Phase 2 study of ponsegromab, a monoclonal antibody directed against growth differentiation factor-15 (GDF-15), met its primary endpoint of change from baseline in body weight compared to placebo in people with cancer cachexia and elevated levels of GDF-15.

（纽约证券交易所：PFE）今天宣布了针对生长分化因子-15（GDF-15）的单克隆抗体ponsegromab的2期研究，与安慰剂相比，癌症恶病质患者的体重与基线相比发生了变化，并且GDF-15水平升高。

Cachexia is a common, life-threatening wasting condition that can significantly impact quality of life in affected patients with cancer..

恶病质是一种常见的危及生命的消耗性疾病，可显着影响受影响癌症患者的生活质量。。

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Results will be presented today as a late-breaking Proffered Paper Presentation (LBA82) at the European Society for Medical Oncology (ESMO) 2024 Congress and have simultaneously been published in

研究结果将于今天在欧洲肿瘤内科学会（ESMO）2024年大会上以最新的发表论文（LBA82）形式发表，并同时发表在

The

New England Journal of Medicine

新英格兰医学杂志

(NEJM)

（奈姆）

“Cachexia is a common condition in cancer patients, associated with weight loss, functional decline, and ultimately poor outcomes. Despite the number of people suffering from cachexia, there are no available options for us to help treat patients,” said Jeffrey Crawford, M.D., George Barth Geller Professor for Research at Duke Cancer Institute, and principal investigator.

杜克癌症研究所（DukeCancerInstitute）研究教授、首席研究员杰弗里·克劳福德（JeffreyCrawford）医学博士（M.D.）表示：“恶病质是癌症患者的常见病，与体重减轻、功能下降以及最终的不良后果有关。尽管有许多人患有恶病质，但我们没有可用的选择来帮助治疗患者。”。

“This study showed us those who received ponsegromab had improvement in body weight, muscle mass, quality of life, and physical function. These findings offer hope that a breakthrough targeted treatment is potentially on the horizon for our patients.”.

“这项研究表明，接受ponsegromab治疗的患者体重、肌肉质量、生活质量和身体机能均有改善。这些发现为我们的患者提供了突破性靶向治疗的希望。”。

The Phase 2 study included 187 participants with non-small cell lung cancer, pancreatic cancer, or colorectal cancer. Ponsegromab demonstrated significant and robust increases in body weight after 12 weeks across all doses: 2.02% (95% confidence interval (CI), -0.97 to 5.01%) in the 100 mg treatment group, 3.48% (95% CI, 0.54 to 6.42%) in the 200 mg group, and 5.61% (95% CI, 2.56 to 8.67%) in the 400 mg group, compared to placebo.

第二阶段研究包括187名非小细胞肺癌，胰腺癌或结直肠癌患者。在所有剂量下，Ponsegromab在12周后显示出显着且稳健的体重增加：100 mg治疗组为2.02%（95%置信区间（CI），-0.97至5.01%），200 mg组为3.48%（95%CI，0.54至6.42%），400 mg组为5.61%（95%CI，2.56至8.67%）。

Improvements across multiple domains of the cachexia phenotype were observed in the 400 mg group including in measures of appetite and cachexia symptoms, physical activity and skeletal muscle index. No clinically significant adverse trends were noted with ponsegromab administration. Treatment related adverse events occurred in 8.9% of patients taking placebo and 7.7% of patients taking ponsegromab..

在400 mg组中观察到恶病质表型多个领域的改善，包括食欲和恶病质症状，体力活动和骨骼肌指数的测量。使用ponsegromab没有发现临床上显着的不良趋势。服用安慰剂的患者中有8.9%发生了与治疗相关的不良事件，服用ponsegromab的患者中有7.7%发生了与治疗相关的不良事件。。

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“Discovered and developed in-house at Pfizer, ponsegromab represents our ability to translate deep scientific expertise into patient benefit,” said Charlotte Allerton, Head of Discovery and Early Development, Pfizer. “These results provide strong evidence that we have unlocked a mechanism to interrupt a critical driver of cachexia, GDF-15, which has the potential to impact patients with cancer cachexia and other life-threatening conditions.

辉瑞公司发现和早期开发负责人夏洛特·阿勒顿（Charlotte Allerton）表示：“在辉瑞公司内部发现和开发的ponsegromab代表了我们将深入的科学专业知识转化为患者利益的能力。”。“这些结果提供了强有力的证据，表明我们已经解锁了一种机制，可以中断恶病质的关键驱动因素GDF-15，它有可能影响癌症恶病质和其他危及生命的疾病患者。

We look forward to advancing this program as part of our broader cardiometabolic portfolio to address weight management across the spectrum of patient need.”.

我们期待着推进这一计划，作为我们更广泛的心脏代谢组合的一部分，以解决患者需求范围内的体重管理问题。”。

Based on these positive results, Pfizer is discussing late-stage development plans with regulators with the goal of starting registration-enabling studies in 2025. Ponsegromab is also being investigated in a Phase 2 study in patients with heart failure (HF) and elevated serum GDF-15 concentrations (.

基于这些积极的结果，辉瑞正在与监管机构讨论后期发展计划，目标是在2025年开始进行注册研究。Ponsegromab也正在进行心力衰竭（HF）和血清GDF-15浓度升高患者的2期研究。

NCT05492500

时间05492500

About the Phase 2 Trial in Cancer Cachexia

关于癌症恶病质的2期试验

The primary objective of the Phase 2

第二阶段的主要目标

study

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(NCT05546476) is to assess the effect of ponsegromab on body weight in patients with cancer (non-small cell lung cancer, pancreatic cancer or colorectal cancer), cachexia and elevated serum GDF-15 concentrations (

（NCT05546476）是为了评估ponsegromab对癌症（非小细胞肺癌、胰腺癌或结直肠癌）、恶病质和血清GDF-15浓度升高患者体重的影响(

1500 pg/mL). Secondary and exploratory endpoints objectives included measures such as: Change from baseline in appetite and cachexia symptoms, digital measures of physical activity, and changes in lumbar skeletal muscle index (LSMI). Patients (n=187) received either ponsegromab (100 mg, 200 mg or 400 mg) or placebo once every four weeks subcutaneously for 12 weeks..

1500微克/毫升）。次要和探索性终点目标包括以下措施：食欲和恶病质症状从基线的变化，身体活动的数字测量以及腰椎骨骼肌指数（LSMI）的变化。患者（n=187）每四周皮下注射一次ponsegromab（100 mg，200 mg或400 mg）或安慰剂，持续12周。。

About Cachexia

关于恶病质

Cachexia is a complex, disabling, and life-threatening metabolic condition that is estimated to affect about 9 million people worldwide.

恶病质是一种复杂的，致残的和危及生命的代谢疾病，估计会影响全世界约900万人。

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Symptoms such as weight and muscle loss can reduce patients’ ability to tolerate treatment for their underlying chronic diseases, such as cancer and heart failure, and can severely impact quality of life.

体重和肌肉减轻等症状会降低患者对癌症和心力衰竭等潜在慢性疾病的耐受能力，并可能严重影响生活质量。

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In cancer, cachexia can diminish the efficacy of cancer treatments and is thought to contribute to decreased survival rates and may cause up to 30% of cancer-related deaths.

在癌症中，恶病质会降低癌症治疗的效果，并被认为有助于降低生存率，并可能导致多达30%的癌症相关死亡。

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Despite its severity, there are no FDA-approved treatments for cachexia.

尽管其严重程度，但尚无FDA批准的恶病质治疗方法。

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About Ponsegromab

关于Ponsegromab

Ponsegromab is an investigational monoclonal antibody designed to treat cachexia by targeting GDF-15. Prior Phase 1b data in participants with cancer cachexia demonstrated proof-of-mechanism for ponsegromab with robust suppression of unbound circulating GDF15 levels .

Ponsegromab是一种研究性单克隆抗体，旨在通过靶向GDF-15治疗恶病质。癌症恶病质参与者的先前1b期数据证明了ponsegromab的机制，并强烈抑制了未结合的循环GDF15水平。

observed

观察到

. The results showed encouraging signals of efficacy that included increases in body weight and encouraging improvements in patient reported outcomes.

结果显示令人鼓舞的疗效信号，包括体重增加和患者报告结果的改善。

About Pfizer: Breakthroughs That Change Patients’ Lives

关于辉瑞：改变患者生活的突破

At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines.

在辉瑞，我们运用科学和全球资源为人们带来治疗方法，延长并显着改善他们的生活。我们努力为包括创新药物和疫苗在内的保健产品的发现、开发和制造制定质量、安全和价值标准。

Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world.

辉瑞公司的同事们每天都在发达市场和新兴市场开展工作，促进健康、预防、治疗和治愈，挑战我们这个时代最可怕的疾病。作为世界首屈一指的创新生物制药公司之一，我们与医疗保健提供者、政府和当地社区合作，支持和扩大全球可靠、负担得起的医疗保健服务。

For 175 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at .

175年来，我们一直致力于为所有依赖我们的人带来改变。我们经常在我们的网站上发布对投资者可能很重要的信息。

www.Pfizer.com

. In addition, to learn more, please visit us on

。此外，要了解更多信息，请访问我们

www.Pfizer.com

and follow us on X at

并在X上跟踪我们

@Pfizer

@辉瑞公司

and

和

@Pfizer News

@辉瑞新闻

YouTube

and like us on Facebook at

就像我们在脸书上

Facebook.com/Pfizer

Disclosure Notice

披露通知

The information contained in this release is as of September 14, 2024. Pfizer assumes no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments.

本版本中包含的信息截至2024年9月14日。辉瑞没有义务因新信息或未来事件或发展而更新本版本中包含的前瞻性声明。

This release contains forward-looking information about ponsegromab, an investigational monoclonal antibody designed to treat cachexia, including its potential benefits and late-stage development planning, and Pfizer’s cardiometabolic portfolio, that involve substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements.

本版本包含关于ponsegromab的前瞻性信息，ponsegromab是一种旨在治疗恶病质的研究性单克隆抗体，包括其潜在益处和晚期发展计划，以及辉瑞公司的心脏代谢组合，这些组合涉及重大风险和不确定性，可能导致实际结果与此类声明所表达或暗示的结果存在实质性差异。

Risks and uncertainties include, among other things, the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for our clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as the possibility of unfavorable new clinical data and further analyses of existing clinical data, including results from the Phase 2 study of ponsegromab in patients with heart failure; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities; whether regulatory authorities will be satisfied with the design of and results from our clinical studies; whether and when drug applications may be filed in any jurisdictions for any potential indication for ponsegromab or any other cardiometabolic product candidates; whether and when any such applications that may be filed for ponsegromab or any other such product candidates may be approved by regulatory authorities, which will depend on myriad factors, including making a determination as to whether the product's benefits outweigh its known risks and determination of the product's efficacy and, if approved, whether ponsegromab or any such other product candidates will be commercially successful; decisions by regulatory author.

风险和不确定性包括研究和开发固有的不确定性，包括满足预期临床终点的能力，我们临床试验的开始和/或完成日期，监管提交日期，监管批准日期和/或发布日期，以及不利的新临床数据和现有临床数据的进一步分析的可能性，包括心力衰竭患者ponsegromab第二阶段研究的结果；临床试验数据受到监管机构不同解释和评估的风险；监管机构是否会对我们临床研究的设计和结果感到满意；是否以及何时可以在任何司法管辖区申请ponsegromab或任何其他心脏代谢产品候选药物的任何潜在适应症；监管机构是否以及何时可以批准针对ponsegromab或任何其他此类候选产品提交的任何此类申请，这将取决于多种因素，包括确定产品的益处是否超过其已知风险，以及确定产品的功效，如果批准，ponsegromab或任何其他此类候选产品是否会取得商业成功；监管机构的决定。

A further description of risks and uncertainties can be found in Pfizer’s Annual Report on Form 10-K for the fiscal year ended December 31, 2023, and in its subsequent reports on Form 10-Q, including in the sections thereof captioned “Risk Factors” and “Forward-Looking Information and Factors That May Affect Future Results”, as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S.

有关风险和不确定性的进一步说明，请参阅辉瑞公司截至2023年12月31日的10-K表年度报告，以及随后的10-Q表报告，包括标题为“风险因素”和“前瞻性信息和可能影响未来结果的因素”的部分，以及随后的8-K表报告，所有这些报告均已提交给美国。

Securities and Exchange Commission and available at .

证券交易委员会，网址为。

www.sec.gov

and

和

www.pfizer.com

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Groarke J, et al. Ponsegromab for the Treatment of Cancer Cachexia.

Groarke J等人。Ponsegromab治疗癌症恶病质。

The New England Journal of Medicine