Breakthrough Therapy Designation granted for investigational therapy IPN60340 in combination with venetoclax and azacitidine in first line unfit acute myeloid leukemia

授予研究性疗法IPN60340联合维奈托克和阿扎胞苷在一线治疗不适合的急性髓系白血病中的突破性疗法认定

PARIS, FRANCE, 13 JANUARY 2026

法国巴黎，2026年1月13日

– Ipsen (Euronext: IPN; ADR: IPSEY) announced today that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) for IPN60340 in combination with venetoclax and azacitidine (Ven-Aza) in first line unfit acute myeloid leukemia, an aggressive blood cancer affecting older adults.

– 益普生（Euronext：IPN；ADR：IPSEY）今天宣布，美国食品药品监督管理局（FDA）已授予IPN60340联合维奈托克和阿扎胞苷（Ven-Aza）用于一线治疗不适合的急性髓系白血病的突破性疗法认定（BTD），这是一种侵袭性的血液癌症，主要影响老年人。

IPN60340 is an investigational first-in-class monoclonal antibody targeting BTN3A, a key immune-regulatory molecule broadly expressed across cancer. Breakthrough Therapy Designation is intended to expedite the development and review of medicines for serious or life-threatening conditions with evidence of a substantial clinical improvement.

IPN60340 是一种研究性首创单克隆抗体，靶向 BTN3A，这是一种在多种癌症中广泛表达的关键免疫调节分子。突破性疗法认定旨在加速用于治疗严重或危及生命疾病的药物的开发和审评，这些药物已展现出显著的临床改善证据。

IPN60340 previously received Orphan Drug Designations from the U.S. Food and Drug Administration and European Medicines Agency in July 2025..

IPN60340 于2025年7月获得了美国食品和药物管理局以及欧洲药品管理局的孤儿药资格认定。

“This Breakthrough Therapy Designation recognizes both the urgent need for new treatment options for people living with acute myeloid leukemia and the promising data seen so far in the development program for IPN60340,” said Christelle Huguet, PhD, EVP and Head of R&D, Ipsen. “We look forward to working closely with the FDA as we advance to the next stage of clinical development and continue to deliver medicines with the potential to be transformative to people living with cancer.” .

“这一突破性疗法认定不仅认可了急性髓系白血病患者对新治疗方案的迫切需求，也反映了IPN60340开发项目迄今为止展现的令人鼓舞的数据，”益普生公司执行副总裁兼研发主管克里斯特尔·乌戈博士表示。“我们期待在进入下一阶段临床开发时与FDA密切合作，继续为癌症患者提供具有变革潜力的药物。”

This Breakthrough Therapy Designation is based on data from the Phase I/II EVICTION trial. Updated clinical data orally presented at the American Society of Hematology (n=57)

这一突破性疗法认定是基于I/II期EVICTION试验的数据。在美国血液学会上口头报告的更新临床数据（n=57）

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from the EVICTION trial showed treatment with IPN60340 in combination with Ven-Aza achieved very encouraging high responses. In this single-arm trial, treatment with IPN60340 and Ven-Aza (n=38), resulted in a near doubling of the complete response relative to those seen in historical standard of care data across all molecular subtypes in newly diagnosed patients including sub-types typically less responsive to standard of care (Ven-Aza)..

来自EVICTION试验的数据显示，IPN60340与Ven-Aza联合治疗取得了非常令人鼓舞的高反应率。在这项单臂试验中，使用IPN60340和Ven-Aza治疗（n=38）使得完全缓解率几乎翻倍，相较于历史标准治疗数据，在所有分子亚型的新诊断患者中均表现出优异效果，包括那些通常对标准治疗（Ven-Aza）反应较差的亚型。

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IPN60340 in combination with Ven-Aza was also shown to be well tolerated, underscoring IPN60340’s potential as a novel immunotherapy to improve outcomes for patients with AML. Based on these preliminary data, we look forward to discussing the design of the Phase II/III development plans with the FDA for IPN60340 in H1 2026..

IPN60340 与 Ven-Aza 联合使用也显示出良好的耐受性，突显了 IPN60340 作为一种新型免疫疗法在改善 AML 患者预后方面的潜力。基于这些初步数据，我们期待在 2026 年上半年与 FDA 讨论 IPN60340 的 II/III 期开发计划的设计。

About the EVICTION trial

关于EVICTION试验

EVICTION is a first-in-human, dose-escalation (Part 1) and cohort-expansion (Part 2) clinical trial of IPN60340 (ICT01) in patients with various advanced relapsed or refractory solid or hematologic cancers that have exhausted standard-of-care treatment options, as well as newly-diagnosed AML. More information on the EVICTION trial can be found at clinicaltrials.gov (NCT04243499)..

EVICTION是一项针对IPN60340（ICT01）的首次人体试验，包含剂量递增（第一部分）和队列扩展（第二部分），适用于多种晚期复发或难治性实体瘤或血液癌症患者，这些患者已穷尽标准治疗方案，同时也包括新诊断的急性髓系白血病（AML）。有关EVICTION试验的更多信息，请访问clinicaltrials.gov（NCT04243499）。

About IPN60340 (ICT01)

关于 IPN60340 (ICT01)

IPN60340 is a humanized, anti-BTN3A (also known as CD277) monoclonal antibody that promotes the recognition and elimination of tumor cells by γ9δ2 T cells, which are responsible for immunosurveillance of malignancy and infections. The three isoforms of BTN3A targeted by IPN60340 are overexpressed on many solid tumors (e.g., melanoma, urothelial cell, colorectal, ovarian, pancreatic, and lung cancer) and hematologic malignancies (e.g., leukemia and lymphomas) and are also expressed on the surface of innate (e.g., γδ T cells and NK cells) and adaptive immune cells (T cells and B cells).

IPN60340是一种人源化的抗BTN3A（也称为CD277）单克隆抗体，能够促进γ9δ2 T细胞对肿瘤细胞的识别和清除。γ9δ2 T细胞负责对恶性肿瘤和感染进行免疫监视。IPN60340所靶向的BTN3A的三种异构体在许多实体瘤（例如黑色素瘤、尿路上皮癌、结直肠癌、卵巢癌、胰腺癌和肺癌）以及血液系统恶性肿瘤（例如白血病和淋巴瘤）中过度表达，并且还表达在先天性免疫细胞（例如γδ T细胞和NK细胞）以及适应性免疫细胞（T细胞和B细胞）的表面。

Binding to BTN3A is essential for the activation of the anti-tumor immune response of γ9δ2 T cells. By altering the conformation of BTN3A, IPN60340 promotes this binding, thereby selectively activating circulating γ9δ2 T cells. This leads to migration of γ9δ2 T cells out of the circulation and into the tumor tissue, and triggers a downstream immunological cascade through secretion of pro-inflammatory cytokines, including but not limited to IFNγ and TNFα, further augmenting the anti-tumor immune response.

与BTN3A的结合对于γ9δ2 T细胞的抗肿瘤免疫反应激活至关重要。通过改变BTN3A的构象，IPN60340促进这种结合，从而选择性激活循环中的γ9δ2 T细胞。这导致γ9δ2 T细胞从循环系统迁移到肿瘤组织，并通过分泌促炎性细胞因子（包括但不限于IFNγ和TNFα）触发下游免疫级联反应，进一步增强抗肿瘤免疫反应。

Anti-tumor activity and efficacy of IPN60340 have been shown in patients across several cancer indications. IPN60340 is an investigational therapy under evaluation for people 75 years or older living with acute myeloid leukemia who due to comorbidities are prevented from receiving treatment with intensive chemotherapy.

IPN60340在多种癌症适应症的患者中显示出抗肿瘤活性和疗效。IPN60340是一种在研疗法，正被评估用于75岁及以上因合并症而无法接受强化疗的急性髓系白血病患者。

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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. Our pipeline is fueled by internal and external innovation and supported by nearly 100 years of development experience and global hubs in the U.S., France and the U.K.

我们是一家全球生物制药公司，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。我们的研发管线由内部和外部创新推动，并得到近百年开发经验以及美国、法国和英国的全球中心的支持。

Our teams in more than 40 countries and our partnerships around the world enable us to bring medicines to patients in more than 100 countries..

我们在40多个国家的团队和全球范围内的合作伙伴关系使我们能够将药品带给100多个国家的患者。

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

益普生在巴黎（泛欧证券交易所：IPN）和美国通过一级赞助美国存托凭证计划（ADR：IPSEY）上市。欲了解更多信息，请访问

www.Ipsen.com

www.Ipsen.com

Ipsen Contacts

益普生联系方式

Investors

投资者

Henry Wheeler

亨利·惠勒

henry.wheeler@ipsen.com

henry.wheeler@ipsen.com

+33 7 66 47 11 49

+33 7 66 47 11 49

Khalid Deojee

哈利德·德欧吉

khalid.deojee@ipsen.com

khalid.deojee@ipsen.com

+33 6 66 01 95 26

+33 6 66 01 95 26

Media

媒体

Sally Bain

萨莉·贝恩

sally.bain@ipsen.com

sally.bain@ipsen.com

+1 857 320 0517

+1 857 320 0517

Anne Liontas

安妮·莱昂塔斯

anne.liontas.ext@ipsen.com

anne.liontas.ext@ipsen.com

+33 7 67 34 72 96

+33 7 67 34 72 96

Disclaimers and/or forward-looking statements

免责声明和/或前瞻性陈述

The forward-looking statements, objectives and targets contained herein are based on Ipsen’s management strategy, current views and assumptions. Such statements involve known and unknown risks and uncertainties that may cause actual results, performance or events to differ materially from those anticipated herein.

本文件中包含的前瞻性声明、目标和指标基于益普生的管理战略、当前观点和假设。这些声明涉及已知和未知的风险与不确定性，可能导致实际结果、业绩或事件与此处预期的内容存在重大差异。

All of the above risks could affect Ipsen’s future ability to achieve its financial targets, which were set assuming reasonable macroeconomic conditions based on the information available today. Use of the words ‘believes’, ‘anticipates’ and ‘expects’ and similar expressions are intended to identify forward-looking statements, including Ipsen’s expectations regarding future events, including regulatory filings and determinations.

所有上述风险可能会影响益普生未来实现其财务目标的能力，这些目标是在假设合理的宏观经济条件的基础上根据今天可获得的信息设定的。使用“相信”、“预期”和“预计”等词语及其类似表达旨在识别前瞻性陈述，包括益普生对未来事件的预期，包括监管文件提交和决定。

Moreover, the targets described in this document were prepared without taking into account external-growth assumptions and potential future acquisitions, which may alter these parameters. These objectives are based on data and assumptions regarded as reasonable by Ipsen. These targets depend on conditions or facts likely to happen in the future, and not exclusively on historical data.

此外，本文件中描述的目标是在未考虑外部增长假设和潜在未来收购的情况下制定的，这些假设和收购可能会改变这些参数。这些目标基于益普生认为合理的数据和假设。这些目标取决于未来可能发生的条件或事实，而不仅仅依赖于历史数据。

Actual results may depart significantly from these targets given the occurrence of certain risks and uncertainties, notably the fact that a promising medicine in early development phase or clinical trial may end up never being launched on the market or reaching its commercial targets, notably for regulatory or competition reasons.

鉴于某些风险和不确定性的发生，特别是处于早期开发阶段或临床试验中的有希望的药物可能最终无法上市或达到其商业目标，尤其是由于监管或竞争原因，实际结果可能与这些目标存在显著差异。

Ipsen must face or might face competition from generic medicine that might translate into a loss of market share. Furthermore, the research and development process involves several stages each of which involves the substantial risk that Ipsen may fail to achieve its objectives and be forced to abandon its efforts with regards t.

益普生可能面临来自仿制药的竞争，这可能会导致市场份额的损失。此外，研发过程包含多个阶段，每个阶段都存在重大风险，即益普生可能无法实现其目标，并被迫放弃相关努力。

ipsen.com

易普森公司

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References

参考文献

Garciaz et al. γ9δ2 T-cell (γ9δ2TC) activation with ICT01 and azacitidine-venetoclax (Aza-Ven) induces high rates of remission and overall survival in patients with newly diagnosed (ND) acute myeloid leukemia (AML): Results from the phase 1/2 study eviction.

Garciaz 等人研究表明，使用 ICT01 和阿扎胞苷-维奈托克（Aza-Ven）激活 γ9δ2 T 细胞（γ9δ2TC）在新诊断（ND）急性髓系白血病（AML）患者中诱导了高缓解率和总体生存率：来自 1/2 期研究 eviction 的结果。

https://ashpublications.org/blood/article/146/Supplement%201/652/549979/9-2-T-cell-9-2TC-activation-with-ICT01-and

https://ashpublications.org/blood/article/146/Supplement%201/652/549979/9-2-T-cell-9-2TC-activation-with-ICT01-and

Dumas et al. γ9δ2 T-cell activation with ICT01 combined with azacitidine-venetoclax for older/unfit adults with newly diagnosed AML: preliminary efficacy and dose selection in Phase 1/2 study EVICTION. ASCO 2025. Available here:

Dumas 等人。使用 ICT01 联合阿扎胞苷-维奈托克激活 γ9δ2 T 细胞治疗新诊断的不适合强化疗的老年 AML 患者：1/2 期研究 EVICTION 的初步疗效与剂量选择。ASCO 2025。可在此处获取：

https://www.ImChecktherapeutics.com/fileadmin/Posters_Prez/ASCO2025-ICT01-AzaVen.pdf

https://www.ImChecktherapeutics.com/fileadmin/Posters_Prez/ASCO2025-ICT01-AzaVen.pdf

Kone AS, Ait Ssi S, Sahraoui S, Badou A. BTN3A: A Promising Immune Checkpoint for Cancer Prognosis and Treatment.

Kone AS, Ait Ssi S, Sahraoui S, Badou A. BTN3A：一个有前景的癌症预后和治疗的免疫检查点。

Int J Mol Sci

国际分子科学杂志

. 2022;23(21):13424. Published 2022 Nov 3. doi:10.3390/ijms232113424

. 2022;23(21):13424. 发表于2022年11月3日. doi:10.3390/ijms232113424

Attachment

附件

Ipsen PR_IPN60340 FDA Breakthrough Designation_13102026

益普生 PR_IPN60340 FDA 突破性疗法认定_13102026