Lanreotide Acetate

Total sales growth of 11.6% at CER1, or 11.7% as reported, driven by all three therapeutic areas and including an increasing contribution from Iqirvo and Bylvay. Tovorafenib regulatory submission to EMA2 for pediatric low-grade glioma. Confirmation of full-year 2025 financial guidance. PARIS, FRANCE, 16 April 2025 – Ipsen (Euronext: IPN; ADR: IPSEY), a global specialty-care biopharmaceutical company, today presents sales for the first quarter of 2025. “Ipsen has delivered a strong start to 2025, building further momentum in the transformation of our company,” commented David Loew, Chief Executive Officer, Ipsen. “We continued to execute on our strategy with strong top-line growth and pipeline progression. I am pleased to see the rapid build-up of our Rare Cholestatic Liver disease franchise with two innovate medicines for five indications. 2025 is set to be an important year for Ipsen, with multiple launches underway and several milestones expected across our portfolio.” Full-year guidance Ipsen is confirming financial guidance for full-year 2025: Total sales growth greater than 5.0%, at constant currency. Based on the average level of exchange rates in March 2025, a limited effect on total sales from currencies is expected. Core operating margin greater than 30.0% of total sales, which includes additional R&D expenses from anticipated early and mid-stage external-innovation opportunities. Guidance includes expected negative impact on Somatuline sales due to increased generic competition in the U.S. and Europe. It excludes any impact from potential late-stage (Phase III clinical development or later) business development transactions. Upcoming Milestones Ipsen anticipates several key milestones across its portfolio in 2025, including: Cabometyx (CABINET trial) – Regulatory decision in the European Union for advanced pancreatic (pNETs) and extra-pancreatic (epNETs) neuroendocrine tumors (NETs). fidrisertib (FALKON trial) – Readout of the pivotal Phase IIb trial in fibrodysplasia ossificans progressiva (FOP). LANT3 (LANTIC trial) – Proof-of-concept data readout, evaluating its potential in aesthetics. Q1 pipeline progress The regulatory filing for tovorafenib was accepted by EMA for review in the European Union, marking an important step forward in the development of this potential treatment for pediatric low-grade glioma and reinforcing Ipsen’s commitment to innovation in rare and difficult-to-treat cancers. Ipsen also initiated the entry in Phase I of IPN01195, a RAF inhibitor, complementing IPN01194, an ERK inhibitor, and tovorafenib, two other assets targeting the MAPK pathway. Group refinancing Ipsen announced on March 19th the successful completion of its inaugural Rated Public Bond of €500 million with a coupon of 3.875%, maturing in March 2032. Following the disclosure of the Investment Grade ratings from S&P and Moody’s, this transaction was very well received and largely oversubscribed by a diversified and solid institutional investor base. This transaction is an important component of Ipsen’s refinancing plan which included the successful renewal of €1,5 billion syndicated Revolving Credit Facility, extending Ipsen’s debt maturity profile. Conference call A conference call and webcast for investors and analysts will begin today at 2pm CET. Participants can access the call and its details by registering here; webcast details can be found here. Calendar Ipsen intends to publish its half-year results on July 31st, 2025. Notes All financial figures are in € millions (€m). The performance shown covers the three-month period to 31 March 2025 (Q1 2025, the quarter), compared to the three-month period to 31 March 2024 (Q1 2024), unless stated otherwise. About Ipsen 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. 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. 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 contacts InvestorsKhalid Deojee +33 6 69 09 12 96 MediaSally Bain +1 857 320 0517Anne Liontas +33 7 67 34 72 96 Disclaimers and/or forward-looking statements 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. 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. 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. 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. 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 to a medicine in which it has invested significant sums. Therefore, Ipsen cannot be certain that favorable results obtained during preclinical trials will be confirmed subsequently during clinical trials, or that the results of clinical trials will be sufficient to demonstrate the safe and effective nature of the medicine concerned. There can be no guarantees a medicine will receive the necessary regulatory approvals or that the medicine will prove to be commercially successful. If underlying assumptions prove inaccurate or risks or uncertainties materialize, actual results may differ materially from those set forth in the forward-looking statements. Other risks and uncertainties include but are not limited to, general industry conditions and competition; general economic factors, including interest rate and currency exchange rate fluctuations; the impact of pharmaceutical industry regulation and healthcare legislation; global trends toward healthcare cost containment; technological advances, new medicine and patents attained by competitors; challenges inherent in new-medicine development, including obtaining regulatory approval; Ipsen’s ability to accurately predict future market conditions; manufacturing difficulties or delays; financial instability of international economies and sovereign risk; dependence on the effectiveness of Ipsen’s patents and other protections for innovative medicines; and the exposure to litigation, including patent litigation, and/or regulatory actions. Ipsen also depends on third parties to develop and market some of its medicines which could potentially generate substantial royalties; these partners could behave in such ways which could cause damage to Ipsen’s activities and financial results. Ipsen cannot be certain that its partners will fulfil their obligations. It might be unable to obtain any benefit from those agreements. A default by any of Ipsen’s partners could generate lower revenues than expected. Such situations could have a negative impact on Ipsen’s business, financial position or performance. Ipsen expressly disclaims any obligation or undertaking to update or revise any forward-looking statements, targets or estimates contained in this press release to reflect any change in events, conditions, assumptions or circumstances on which any such statements are based, unless so required by applicable law. Ipsen’s business is subject to the risk factors outlined in its registration documents filed with the French Autorité des Marchés Financiers. The risks and uncertainties set out are not exhaustive and the reader is advised to refer to Ipsen’s latest Universal Registration Document, available on ipsen.com. 1 At constant exchange rates (CER), which exclude any foreign-exchange impact by recalculating the performance for the relevant period by applying the exchange rates used for the prior period.2 EMA: European Medicines Agency 3 Long-acting neurotoxin Attachment Ipsen PR_Q1 2025_15042025 Total sales growth of 11.6% at CER1, or 11.7% as reported, driven by all three therapeutic areas and including an increasing contribution from Iqirvo and Bylvay. Tovorafenib regulatory submission to EMA2 for pediatric low-grade glioma. Confirmation of full-year 2025 financial guidance. PARIS, FRANCE, 16 April 2025 – Ipsen (Euronext: IPN; ADR: IPSEY), a global specialty-care biopharmaceutical company, today presents sales for the first quarter of 2025. “Ipsen has delivered a strong start to 2025, building further momentum in the transformation of our company,” commented David Loew, Chief Executive Officer, Ipsen. “We continued to execute on our strategy with strong top-line growth and pipeline progression. I am pleased to see the rapid build-up of our Rare Cholestatic Liver disease franchise with two innovate medicines for five indications. 2025 is set to be an important year for Ipsen, with multiple launches underway and several milestones expected across our portfolio.” Full-year guidance Ipsen is confirming financial guidance for full-year 2025: Total sales growth greater than 5.0%, at constant currency. Based on the average level of exchange rates in March 2025, a limited effect on total sales from currencies is expected. Core operating margin greater than 30.0% of total sales, which includes additional R&D expenses from anticipated early and mid-stage external-innovation opportunities. Guidance includes expected negative impact on Somatuline sales due to increased generic competition in the U.S. and Europe. It excludes any impact from potential late-stage (Phase III clinical development or later) business development transactions. Upcoming Milestones Ipsen anticipates several key milestones across its portfolio in 2025, including: Cabometyx (CABINET trial) – Regulatory decision in the European Union for advanced pancreatic (pNETs) and extra-pancreatic (epNETs) neuroendocrine tumors (NETs). fidrisertib (FALKON trial) – Readout of the pivotal Phase IIb trial in fibrodysplasia ossificans progressiva (FOP). LANT3 (LANTIC trial) – Proof-of-concept data readout, evaluating its potential in aesthetics. Q1 pipeline progress The regulatory filing for tovorafenib was accepted by EMA for review in the European Union, marking an important step forward in the development of this potential treatment for pediatric low-grade glioma and reinforcing Ipsen’s commitment to innovation in rare and difficult-to-treat cancers. Ipsen also initiated the entry in Phase I of IPN01195, a RAF inhibitor, complementing IPN01194, an ERK inhibitor, and tovorafenib, two other assets targeting the MAPK pathway. Group refinancing Ipsen announced on March 19th the successful completion of its inaugural Rated Public Bond of €500 million with a coupon of 3.875%, maturing in March 2032. Following the disclosure of the Investment Grade ratings from S&P and Moody’s, this transaction was very well received and largely oversubscribed by a diversified and solid institutional investor base. This transaction is an important component of Ipsen’s refinancing plan which included the successful renewal of €1,5 billion syndicated Revolving Credit Facility, extending Ipsen’s debt maturity profile. Conference call A conference call and webcast for investors and analysts will begin today at 2pm CET. Participants can access the call and its details by registering here; webcast details can be found here. Calendar Ipsen intends to publish its half-year results on July 31st, 2025. Notes All financial figures are in € millions (€m). The performance shown covers the three-month period to 31 March 2025 (Q1 2025, the quarter), compared to the three-month period to 31 March 2024 (Q1 2024), unless stated otherwise. About Ipsen 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. 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. 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 contacts InvestorsKhalid Deojee +33 6 69 09 12 96 MediaSally Bain +1 857 320 0517Anne Liontas +33 7 67 34 72 96 Disclaimers and/or forward-looking statements 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. 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. 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. 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. 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 to a medicine in which it has invested significant sums. Therefore, Ipsen cannot be certain that favorable results obtained during preclinical trials will be confirmed subsequently during clinical trials, or that the results of clinical trials will be sufficient to demonstrate the safe and effective nature of the medicine concerned. There can be no guarantees a medicine will receive the necessary regulatory approvals or that the medicine will prove to be commercially successful. If underlying assumptions prove inaccurate or risks or uncertainties materialize, actual results may differ materially from those set forth in the forward-looking statements. Other risks and uncertainties include but are not limited to, general industry conditions and competition; general economic factors, including interest rate and currency exchange rate fluctuations; the impact of pharmaceutical industry regulation and healthcare legislation; global trends toward healthcare cost containment; technological advances, new medicine and patents attained by competitors; challenges inherent in new-medicine development, including obtaining regulatory approval; Ipsen’s ability to accurately predict future market conditions; manufacturing difficulties or delays; financial instability of international economies and sovereign risk; dependence on the effectiveness of Ipsen’s patents and other protections for innovative medicines; and the exposure to litigation, including patent litigation, and/or regulatory actions. Ipsen also depends on third parties to develop and market some of its medicines which could potentially generate substantial royalties; these partners could behave in such ways which could cause damage to Ipsen’s activities and financial results. Ipsen cannot be certain that its partners will fulfil their obligations. It might be unable to obtain any benefit from those agreements. A default by any of Ipsen’s partners could generate lower revenues than expected. Such situations could have a negative impact on Ipsen’s business, financial position or performance. Ipsen expressly disclaims any obligation or undertaking to update or revise any forward-looking statements, targets or estimates contained in this press release to reflect any change in events, conditions, assumptions or circumstances on which any such statements are based, unless so required by applicable law. Ipsen’s business is subject to the risk factors outlined in its registration documents filed with the French Autorité des Marchés Financiers. The risks and uncertainties set out are not exhaustive and the reader is advised to refer to Ipsen’s latest Universal Registration Document, available on ipsen.com. 1 At constant exchange rates (CER), which exclude any foreign-exchange impact by recalculating the performance for the relevant period by applying the exchange rates used for the prior period.2 EMA: European Medicines Agency 3 Long-acting neurotoxin Attachment Ipsen PR_Q1 2025_15042025

01. 背景简介2025年4月，德累斯顿卡尔·古斯塔夫·卡鲁斯大学医院（University Hospital Carl Gustav Carus, Technische Universität Dresden）核医学科的Enrico Michler为通讯作者在《European Journal of Nuclear Medicine and Molecular Imaging》期刊上正式发表名为“[203/212Pb]Pb-VMT-α-NET as a novel theranostic agent for targeted alpha radiotherapy—first clinical experience”的研究论文，详细阐述了新型治疗诊断剂[203/212Pb]Pb-VMT-α-NET在神经内分泌肿瘤患者中的首次临床研究。02. 研究内容神经内分泌肿瘤(NEN)是一类起源于肽能神经元和神经内分泌细胞，具有神经内分泌分化并表达神经内分泌标志物的少见肿瘤，可发生于全身各处，具有高度异质性，以肺及胃肠胰NEN(GEP-NEN)最常见。由于发病率低且生物学行为呈现高度异质性，NEN在临床上被归类为罕见疾病。生长抑素受体（SSTR）表达 是使用生长抑素类似物（SSA）或肽受体放射性核素治疗（PRRT）的重要基础，2016年68Ga-DOTA-TATE被美国食品药品监督管理局批准上市，2018年117Lu-DOTA-TATE又被批准用于NEN的PRRT治疗。尽管这些突破为晚期NETs患者提供了新的治疗选择，但其客观缓解率仍较为有限（仅约18%），因此针对新型治疗策略的探索仍是当前研究的热点领域。为提高生长抑素类似物（SSA）的临床应用价值，研究者通过结构修饰开发了多代长效化、高稳定性的衍生物，包括奥曲肽（Octreotide）、兰瑞肽（Lanreotide）及伐普肽（Vamorolone）等。其中，基于奥曲肽核心结构优化的SSTR2激动剂Tyr3-octreotide（TOC）、octreotate（TATE）、Tyr3-octreotate（Y3-TATE）和1-Nal3-octreotide(NOC)，展现出更强的结合能力。与传统的生长抑素受体激动剂相比，拮抗剂不具有内化作用，但拮抗剂与SSTR结合亲和力却比激动剂高8-9倍，也能够显著提高病灶的靶/非靶比值（如JR11、LM3）。本研究采用的TOC分子，在保留SSTR2高亲和特性的基础上，用铅特异性螯合剂（PSC）替代传统DOTA/TCMC结构，PSC通过优化的配体空间构象及螯合位点，能够对Pb及其子核实现高效稳定螯合，结构中引入的聚乙二醇（PEG）柔性链更可增强分子亲水性，从而优化整体的药代动力学。相关往期链接:诊疗一体化203/212Pb的新螯合剂！212Pb-PSMA靶向核药：AB001首次人体研研究结果公布！符合GMP标准的[212Pb]Pb核药！212Pb席卷全球核素资本市场，正面宣战王牌核素225Ac，凭什么？212Pb与225Ac（一）：核素性质与核资源储备Alpha核素选手212Pb已就位重磅：212Pb核药实现胰腺癌靶向治疗！JNM: 212Pb-DOTAMTATE靶向SSTR转移性神经内分泌肿瘤首次人体剂量递增临床试验203/212Pb靶向SSTR2新核药！[177Lu]ZHER2-HSA-DOTA和[212Pb]ZHER2-HSA-DOTA的合成应用这项单中心回顾性分析，纳入了12例经多线治疗（包括[177Lu]Lu-DOTATATE和[225Ac]Ac-DOTATATE治疗）后进展的转移性胃肠胰NET患者（G1-3级）。患者接受单次静脉注射4.9 MBq/kg（平均365 MBq）的[203Pb]Pb-VMT-α-NET，SPECT/CT成像于注射后10分钟、2、10、24及41小时采集。评估肿瘤摄取和药代动力学，符合条件的患者将接受单剂量1.2 MBq/kg（平均100 MBq）的[212Pb]Pb-VMT-α-NET，SPECT/CT于治疗后10分钟、2、4、20小时采集。[203Pb]Pb-VMT-α-NET和[212Pb]Pb-VMT-α-NET的平均给药间隔为42天（20-76天）。患者9的[68Ga]Ga-DOTATATE PET/CT的图像，患者在注射243 MBq [203Pb]Pb-VMT-α-NET后2.5小时的图像，以及在注射86 MBq [212Pb]Pb-VMT-α-NET后2.5小时的治疗后成像。同一患者不同时间点SPECT/CT显示的肿瘤摄取变化。总之，SPECT/CT成像显示所有患者在[203Pb]Pb-VMT-α-NET成像的早期时间点（3小时以内）肿瘤摄取高于肝/肾，与[68Ga]Ga-DOTATATE的显像一致，但肿瘤摄取异质性显著，2例患者因[203Pb]Pb-VMT-α-NET显示肿瘤摄取不足未接受后续治疗。8号患者的[68Ga]Ga-DOTATATE的PET/CT成像：左图为基线期图像，此时患者存在多处肝脏、骨和淋巴结转移；右图为静脉注射108 MBq [212Pb]Pb-VMT-α-NET后3个月后的随访图像。肿瘤负荷以及肿瘤标志物嗜铬粒蛋白A（CgA）在随访期间保持稳定（基线期数值为3246 ng/ml对比随访期数值为3317 ng/ml ）。 肿瘤代谢体积（MTV）瀑布图展示个体治疗反应差异：该瀑布图展示了8名患者接受单周期[212Pb]Pb-VMT-α-NET治疗后代谢肿瘤体积的变化情况。治疗结果显示，此次单剂量[212Pb]Pb-VMT-α-NET（1.2 MBq/kg）耐受性良好，仅2例出现3级短暂恶心/呕吐，未观察到血液学或肾功能显著损伤。治疗后3个月评估显示，所有接受治疗的8例接受治疗患者达到疾病稳定（RECIST 1.1），代谢肿瘤体积无显著变化（p=0.735），但肿瘤标志物CgA显著下降（p=0.05）。03. 小结此次研究仅单次给药，且1.2 MBq/kg给药剂量相对保守，提高给药剂量如2.5 MBq/kg，此外采用多周期给药将更有效，未来还可考虑与免疫检查点抑制剂、DNA损伤修复抑制剂联用，以增强α疗法协同效应。文末附上了原文链接，方便读者检索拜读。●JNM：蓝纳成PSMA新核药68Ga/177Lu-LNC1011及其临床转化研究结果！●NTSR1靶向新核药[177Lu]Lu-NA-ET1！●212Pb-PSMA靶向核药：AB001首次人体研研究结果公布！●新型PSMA靶向核药[18F]AlF-PSMA-N5及首次临床转化研究！声明：本文观点仅代表小核本人，视角局限，欢迎批评指正；如需转载，请务必注明文章作者和来源。如有其它问题，请在本平台留言或联系xiaoheshuoheyao777@163.com，小核将在第一时间处理。内容编辑 | 小核排版设计 | 小核媒体合作 | 科研宣传 | 转载开白xiaoheshuoheyao777（小核微信）