Randomized Phase III Study of Second-Line Chemotherapy With or Without Panitumumab for KRAS Wild Type, Locally Advanced or Metastatic Pancreatic Adenocarcinoma

This phase III trial compares the effect of adding panitumumab to standard chemotherapy (with nanoliposomal Irinotecan, leucovorin, and 5-fluorouracil [5-FU] or irinotecan, leucovorin, and 5-FU or nab-paclitaxel and gemcitabine) versus standard chemotherapy alone in treating patients with KRAS wild type (WT) pancreatic ductal adenocarcinoma that cannot be removed by sugery (unresectable) or that has spread to nearby tissue or lymph nodes (locally advanced) or that has spread from where it first started (primary site) to other places in the body (metastatic). Panitumumab is a monoclonal antibody that may interfere with the ability of tumor cells to grow and spread. A monoclonal antibody is a type of protein that can bind to certain targets in the body, such as molecules that cause the body to make an immune response (antigens). Chemotherapy drugs, such as nanoliposomal irinotecan, leucovorin, 5-FU, irinotecan, nab-paclitaxel and gemcitabine, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Adding panitumumab to standard chemotherapy may be effective in treating patients with unresectable, locally advanced, or metastatic KRAS WT pancreatic ductal adenocarcinoma.

Start Date06 Jan 2026

Sponsor / Collaborator

SWOG

[+1]

NCT07033689

/ Not yet recruitingEarly Phase 1

Open-label, Non-randomized, Single-center Study Investigating the Feasibility, Safety, Tolerability and Efficacy of Suizenji in Unresectable Pancreatic Ductal Adenocarcinoma Patients

This is a feasibility, safety, tolerability, and efficacy research study of an investigational device called "Suizenji", an ultrasound-guided high-intensity focused ultrasound (HIFU) therapy system for the treatment of unresectable pancreatic ductal adenocarcinoma. Focused ultrasound therapy uses a number of small ultrasound generators attached to a bowl-shaped ultrasound generator to emit ultrasound waves from outside to inside the body and focus them on a single point where cancerous cells in the pancreas are located. The targeted area is then heated, which kills the pancreatic cancer cells, and as a result, the patient's life may be prolonged. Experience with Suizenji for pancreatic cancer patients has shown that the "heating" is only a warm feeling in the abdomen during the treatment.

Start Date01 Dec 2025

Sponsor / Collaborator

Sonire Therapeutics Inc

NCT06821997

/ RecruitingPhase 2IIT

A Phase 2 Clinical Trial of Nalirifox as Neoadjuvant Treatment for Patients With Borderline Resectable Pancreatic Ductal Adenocarcinoma (Nectar Study)

This phase II trial tests how well liposomal irinotecan, oxaliplatin, 5-fluorouracil and leucovorin (NALIRIFOX) before surgery works in treating patients with pancreatic ductal adenocarcinoma that is close to major blood vessels, but is still potentially removable by surgery (borderline resectable). Irinotecan is in a class of antineoplastic medications called topoisomerase I inhibitors. It blocks a certain enzyme needed for cell division and deoxyribonucleic acid (DNA) repair and may kill tumor cells. Liposomal irinotecan is a form of the anticancer drug irinotecan that is contained inside very tiny, fat-like particles. Liposomal irinotecan may have fewer side effects and work better than other forms of the drug. Oxaliplatin is in a class of medications called platinum-containing antineoplastic agents. It damages the cell's DNA and may kill tumor cells. 5-fluorouracil, a type of antimetabolite, stops cells from making DNA and it may kill tumor cells. Leucovorin, a form of folic acid, is used to lessen the toxic effects of substances that block the action of folic acid. It is a type of chemoprotective agent and a type of chemosensitizing agent. Giving NALIRIFOX before surgery may improve the chance of successful surgery and decrease the chance of the cancer returning after surgery in patients with borderline resectable pancreatic ductal adenocarcinoma.

Start Date01 Aug 2025

Sponsor / Collaborator

Roswell Park Comprehensive Cancer Center

100 Clinical Results associated with Irinotecan Hydrochloride Lioposome

100 Translational Medicine associated with Irinotecan Hydrochloride Lioposome

100 Patents (Medical) associated with Irinotecan Hydrochloride Lioposome

9,250

Literatures (Medical) associated with Irinotecan Hydrochloride Lioposome

31 Dec 2025·AUTOIMMUNITY

Deciphering distinct pathogenic mechanisms of ankylosing spondylitis and systemic sclerosis via shared biomarkers ZSWIM6 and CCL3L3: Insights from an integrative bioinformatics approach

Article

Author: Zhou, Chenxing ; Chen, Jiarui ; Zhan, Xinli ; Xue, Jiang ; Shi, Jianjun ; Zhou, Zhongxian ; Yang, Renbang ; Huang, Liangyu ; Huang, Chengqian ; Liu, Chong ; Chen, Tianyou ; Feng, Sitan ; Huang, Jieping ; Wu, Shaofeng ; Zhu, Jichong

Ankylosing Spondylitis (AS) and Systemic Sclerosis (SSc) are both autoimmune diseases, albeit with distinct anatomical targets. AS primarily affects the spine and sacroiliac joints, triggering inflammation and eventual fusion of the vertebrae. SSc predominantly impacts the skin and connective tissues, leading to skin fibrosis, thickening, and potential damage to vital organs such as the lungs, heart, and kidneys. Despite their differing anatomical manifestations, inflammation serves as a pivotal factor in both conditions. Exploring the causes of the different pathogenesis of inflammation in AS and SSc could provide new insights into their treatment. We selected RNA-seq profiles of peripheral blood mononuclear cells (PBMCs) from the GEO datasets GSE73754 and GSE19617. DEGs were identified using the Limma R package with an adjusted p-value cutoff of < 0.05. Gene Ontology pathway analysis, SVM recursive feature elimination, and Gene Set Enrichment Analysis (GSEA) were conducted to analyze the DEGs. CIBERSORT was applied to estimate immune cell composition and its correlation with hub genes. Single-cell RNA sequencing data from peripheral blood mononuclear cells in the GSE194315 dataset were included to support differential expression analysis and biomarker identification. Additionally, single-cell RNA sequencing data from bone marrow blood samples were utilized to further validate these findings, offering complementary insights into biomarker expression across distinct sample types. A total of 762 DEGs were identified between AS patients and controls, and 441 DEGs between SSc patients and controls. Both conditions showed enrichment in the Natural killer cell mediated cytotoxicity pathway. ZSWIM6 and CCL3L3 were identified as potential biomarkers in AS and SSc, with significant diagnostic capabilities demonstrated by ROC analysis. Correlation analysis revealed associations between these biomarkers and specific immune cell types. The study utilizing ZSWIM6 and CCL3L3 as potential biomarkers provides deep insights into the distinct molecular mechanisms of SSc and AS. These findings lay the foundation for future research on targeted therapies and enhance our understanding of immune cell interactions in these autoimmune diseases.

01 Aug 2025·INTERNATIONAL JOURNAL OF PHARMACEUTICS

Local drug delivery of irinotecan with phosphatidyldiglycerol-based thermosensitive liposomes reduces systemic exposure and increases therapeutic efficacy compared to systemic drug and Onivyde®

Article

Author: Wedmann, Barbara ; Lindner, Lars H ; Pointner, Lisa ; Hossann, Martin ; Winter, Gerhard ; Kort, Simone

Thermosensitive Liposomes (TSL) represent a promising tool for targeted drug delivery in combination with local hyperthermia (HT). Irinotecan (CPT-11) is approved for the treatment of various solid tumors but is rapidly metabolized after i.v. injection impairing intratumoral uptake. CPT-11 was therefore encapsulated in phosphatidyldiglycerol-based TSL (DPPG₂-TSL-CPT-11) to extend its blood stability and to intravascularly release CPT-11 in the tumor. In vitro, DPPG₂-TSL-CPT-11 demonstrated efficient drug retention at ≤ 39 °C and fast CPT-11 release at > 40 °C in presence of serum. DPPG₂-TSL-CPT-11 showed a CPT-11 plasma half-life of 1.57 h in rats compared to 0.71 h for non-liposomal CPT-11 and 18.70 h for PEGylated non-thermosensitive CPT-11 (Onivyde®), respectively. Systemic exposure of the active metabolite SN-38 was reduced by DPPG₂-TSL-CPT-11 compared to Onivyde®. One hour of HT treatment in combination with DPPG₂-TSL-DOX delivered 24-fold and 36-fold more CPT-11 into tumors than Onivyde® and non-liposomal CPT-11, respectively. DPPG₂-TSL-DOX and Onivyde® showed a comparable overall survival, superior (p < 0.005) to non-liposomal CPT-11. DPPG₂-TSL-DOX was the only formulation that led to a tumor size reduction. In vivo data indicated an advantage of the intravascular CPT-11 release over systemic drug application or passive tumor accumulation of liposomes.

01 Aug 2025·JOURNAL OF PHARMACEUTICAL AND BIOMEDICAL ANALYSIS

Simultaneous analysis of various anticancer drugs by supercritical fluid chromatography-mass spectrometry. Part I: Optimization of chromatographic separation

Article

Author: Guillarme, Davy ; Rudaz, Serge ; Fleury-Souverain, Sandrine ; Nguyen, Nathalie ; Bonnabry, Pascal

This work presents a generic SFC-MS method for the simultaneous analysis of 22 anticancer drugs (fluorouracil, busulfan, cyclophosphamide, cytarabine, dacarbazine, daunorubicin, docetaxel, doxorubicin, epirubicin, etoposide, gemcitabine, idarubicin, ifosfamide, irinotecan, methotrexate, paclitaxel, pemetrexed, raltitrexed, topotecan, treosulfan, vinblastine, vincristine). The separation conditions were optimized by screening nine stationary phases (2-picolylamine, bare hybrid silica, 2-ethylpyridine, fluoro-phenyl, octadecyl, diethylamine, diol, 1-aminoanthracene, zwitterionic modification), evaluating additives effects (2-5 % water, 20-50 mM ammonium formate, 0-1 mM ammonium fluoride), and adjusting the organic modifier composition (methanol, ethanol, isopropanol, acetonitrile). The optimized SFC-MS method successfully analyzed 22 anticancer drugs, along with 5 additional challenging compounds (azacitidine, mitomycin, cisplatin, oxaliplatin, carboplatin), in 12 min, using a diol column (100 × 3 mm, 1.7 µm) and a gradient of 2-100 % methanol containing 2 % water and 50 mM ammonium formate. To overcome overpressure generated by high organic solvent content, a backpressure gradient (110-150 bar) and a flow rate gradient (0.6-1.5 mL/min) were applied. The diol column was selected as the most promising based on five predefined chromatographic criteria. Additives with 5 % water or ammonium fluoride were excluded due to overpressure and signal loss, respectively. Increasing ammonium formate concentration improved peak symmetry by 29 %. For the organic modifier, pure methanol was chosen since ternary mixtures led to system overpressure without improving separation. Comparison with the LC-MS method using real samples confirmed the potential applicability of the SFC method, as the same trace compounds were detected with comparable concentrations. Sensitivity optimization and method validation will be discussed separately in a later paper.

122

News (Medical) associated with Irinotecan Hydrochloride Lioposome

04 Jun 2025

·www.globenewswire.com

Late-breaking analysis demonstrates characteristics associated with long-term overall survival with Onivyde® regimen in metastatic pancreatic adenocarcinoma

Phase III NAPOLI 3 trial is the largest and has the longest follow-up for an interventional study in metastatic pancreatic adenocarcinoma1 Post-hoc analysis of NAPOLI 3 study determined characteristics associated with long-term survival, with median overall survival of 19.5 months amongst long-term survivors receiving Onivyde® plus oxaliplatin, fluorouracil and leucovorin (NALIRIFOX) regimen as a first-line therapy2 Dose reductions and/or treatment delays for the management of adverse events enabled patients to stay on treatment longer and achieve high cumulative doses of liposomal irinotecan and oxaliplatin2 31 May 2025 – Late-breaking (LBA4175) post-hoc analysis data from the Phase III NAPOLI 3 study were presented today at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting. These results found a median overall survival (mOS) of 19.5 months among long-term survivors (n=15) with metastatic pancreatic adenocarcinoma (mPDAC) treated with the Onivyde® (irinotecan liposome injection) plus oxaliplatin, fluorouracil and leucovorin (NALIRIFOX) regimen as a first-line treatment (n=120), with younger age at diagnosis, and certain tumor and metastasis locations associated with long-term survivorship.2 Pancreatic adenocarcinoma (PDAC) is the most common type of cancer that forms in the pancreas, with more than 60,000 people diagnosed annually in the U.S. and nearly 500,000 people globally.3,4 It is often detected after the disease has spread to other parts of the body (metastatic or stage IV)5 and fewer than 20% of people diagnosed with metastatic pancreatic adenocarcinoma (mPDAC) survive longer than one year.5,6 Overall, pancreatic cancer has the lowest five-year survival rate of all cancer types globally and in the U.S.5,6 “When people are diagnosed with metastatic pancreatic adenocarcinoma, the most important question remains: how long will they have with their loved ones,” said Dr. Vincent Chung, Medical Oncologist, City of Hope. “Findings from the NAPOLI 3 post-hoc analysis provide important context on long-term overall survival with the Onivyde (NALIRIFOX) treatment regimen.” The analysis included patients who survived for 18 months or longer (N=15), with findings showing long-term survivors living with mPDAC had a mOS of 19.5 months (interquartile range [IQR]: 18.8–22.6).2 Clinical and pathological factors of long-term survivors included younger than average age at time of diagnosis (median age 61.0 (IQR: 49.0–70.5) as well as tumor location.2 Fewer patients had tumors in the head or tail of the pancreas (53.3% had the main pancreatic tumor located in the body of the pancreas), a substantial proportion had liver metastasis (66.7%) and ≥3 metastatic sites (53.3%).2 Additionally, findings indicate dose reduction and treatment delays resulted in prolonged exposure and higher cumulative doses of the Onivyde (NALIRIFOX) regimen.1 Liver metastasis and ≥3 metastatic sites, dose modifications and an otherwise good clinical profile enabled people to achieve a long mOS.2 Consideration should be taken when interpreting these results as a post-hoc analysis with a small sample size. "Data from the Phase III NAPOLI 3 trial were the first positive data of its kind in a decade and continue to reinforce the potential for long-term outcomes with the Onviyde (NALIRIFOX) regimen,” said Sandra Silvestri, MD, PhD, Executive Vice President, Chief Medical Officer, Ipsen. “With people on average living just 4-6 months following diagnosis with pancreatic adenocarcinoma, these data help us to understand the characteristics associated with long-term survival seen in the NAPOLI trial, an important advancement for this difficult-to-treat cancer where data of this kind are scarce.” Onivyde is a long-circulating liposomal topoisomerase inhibitor. In Onivyde, irinotecan is enclosed in tiny fat particles called liposomes which accumulate in the tumor and release slowly over time. Onivyde is administered via intravenous infusion over 90 minutes every two weeks with recommendations on dosing modifications. Onivyde, as part of the NALIRIFOX regimen (combined with oxaliplatin, fluorouracil (FU) and leucovorin (LV)), is for people living with mPDAC who are treatment naïve or used in combination with FU and LV following gemcitabine-based therapy. Onivyde is not indicated as a single agent for the treatment of adult patients with metastatic pancreatic adenocarcinoma. Ipsen has exclusive commercialization rights for the current and potential future indications for Onivyde in the U.S. Servier, an independent international pharmaceutical company governed by a foundation and with an international presence in 140 countries, is responsible for the commercialization of Onivyde outside of the U.S., Taiwan and Canada. PharmaEngine is a commercial stage oncology company headquartered in Taipei and is responsible for the commercialization of Onivyde in Taiwan. NAPOLI 3 is a randomized, open-label Phase III trial of an Onivyde treatment regimen (NALIRIFOX) in treatment-naïve mPDAC. NAPOLI 3 enrolled 770 patients across 187 trial site locations in 18 countries across Europe, North America, South America, Asia, and Australia. Patients were randomized to receive Onivyde plus oxaliplatin, fluorouracil and leucovorin (NALIRIFOX regimen; n=383) twice in a month (days 1 and 15 of 28-day cycle) compared to an injection of nab-paclitaxel and gemcitabine (n=387) administered three times a month (days 1, 8, 15 of a 28-day cycle). 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 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. References 1 Wainberg et al. NALIRIFOX versus nab-paclitaxel and gemcitabine in treatment-naive patients with metastatic pancreatic ductal adenocarcinoma (NAPOLI 3): a randomised, open-label, phase 3 trial. Lancet. 2023 Oct 7;402(10409):1272-1281. 2 Chung et al. NAPOLI 3 phase 3 study of NALIRIFOX in patients with metastatic pancreatic ductal adenocarcinoma (mPDAC): final overall survival (OS) analysis and characteristics of the long-term survivors. As presented at ASCO Congress 2025 Chicago, USA 3 American Cancer Society – Cancer Facts and Figures 2024. Available : https://www.cancer.org/content/dam/cancer-org/research/cancer-facts-and-statistics/annual-cancer-facts-and-figures/2024/2024-cancer-facts-and-figures-acs.pdf 4 https://www.cancer.net/cancer-types/pancreatic-cancer/statistics 5 Orth, M., Metzger, P., Gerum, S. et al. Pancreatic ductal adenocarcinoma: biological hallmarks, current status, and future perspectives of combined modality treatment approaches. Radiat Oncol 14, 141 (2019). https://doi-org.libproxy1.nus.edu.sg/10.1186/s13014-019-1345-6 The content above comes from the network. if any infringement, please contact us to modify.

Clinical ResultASCO

16 May 2025

·www.fiercepharma.com

Fierce Pharma Asia—Pfizer partner's trial win; Roche's $300M China plant; Hengrui's HK IPO plan

Positive data from a Pfizer-partnered antibody-drug conjugate, Roche's new investment in China and Hengrui's upcoming dual listing in Hong Kong made our news this week. Pfizer and RemeGen's HER2 antibody-drug conjugate delivered positive phase 3 results in a bladder cancer trial in China. Roche plans to invest nearly $300 million to build a new Chinese plant to produce Vabysmo. China's Hengrui Pharma is looking to raise up to $1.27 billion in its Hong Kong IPO. And more.1. Pfizer partner RemeGen says HER2 ADC prolongs life in newly diagnosed bladder cancerRemeGen’s Pfizer-partnered HER2 antibody-drug conjugate, disitamab vedotin, was successful in a phase 3 study among Chinese patients with first-line HER2-expressing bladder cancer. The ADC, used alongside Junshi Biosciences’ PD-1 inhibitor toripalimab, outperformed chemotherapy regardless of patients' eligibility for cisplatin or HER2 expression levels. The trial win bodes well for Pfizer’s global DV-001 study, also called Keynote-D74.2. Roche pledges nearly $300M to establish new Vabysmo production plant in ChinaRoche is investing about 2 billion Chinese yuan ($282 million) to build a new biologics plant in Shanghai. The plant will occupy about 25,000 square meters (269,000 square feet) of real estate and will mainly be used for domestic production of the eye med Vabysmo. Construction is expected to finish in 2029, and production could kick off by 2031, according to Roche.3. Hengrui looks to raise up to $1.27B in Hong Kong IPO (Morningstar)Jiangsu Hengrui Pharmaceutical, already listed in Shanghai, is planning to raise up to HK$9.89 billion ($1.27 billion) in a Hong Kong IPO. The plan involves selling 224.52 million shares at a price range of HK$41.45 to HK$44.05 each. The Chinese pharma giant expects to finalize pricing by May 21 and start trading from May 23.4. Qilu Pharmaceutical pays $38M upfront for greater China rights to Minghui's ADCThe B7-H3 target has yielded another deal. Qilu Pharmaceutical is paying 280 million Chinese yuan ($38 million) upfront for greater China rights to Minghui Pharmaceutical’s B7-H3 ADC, MHB088C. Also in the B7-H3 ADC field, Merck & Co. has licensed ifinatamab deruxtecan from Daiichi Sankyo, GSK got a candidate from Hansoh and BioNTech partnered with Duality Bio.5. CSPC licenses Onivyde copycat to Cipla despite Ipsen patent dispute (release, PDF)Despite a pending patent infringement lawsuit from Ipsen, CSPC Pharmaceutical has penned a deal to license its irinotecan liposome injection in the U.S. to Cipla for $15 million upfront. Milestone payments from the pact could exceed $1 billion. In a lawsuit filed in August 2024, Ipsen alleges that the CSPC candidate infringes a patent for the French pharma’s irinotecan liposome product Onivyde.6. Novartis cancer drugs Kisqali, Scemblix win China approvals (releases, Chinese)In two approvals for Novartis’ business in China, local authorities approved the company’s Kisqali for the adjuvant treatment of early-stage HR-positive, HER2-negative breast cancer, as well as Scemblix for newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia. Both indications have been cleared in the U.S. and are considered major growth drivers for the Swiss pharma.Other News of Note:7. Merck KGaA drops China surcharge on orders after US-China tariff deal (Reuters)8. Sino-American siRNA biotech Sirius Therapeutics raises $50M (release)

Drug ApprovalPhase 3ADCClinical Result

13 Feb 2025

·www.ipsen.com

Ipsen delivers solid results in 2024, driven by strong performance across all therapeutic areas, and provides guidance for 2025

FY 2024 total sales growth of 9.9% at CER1, or 8.7% as reported, with growth driven by strong performance across all therapeutic areas, including a 67.4% increase in the Rare Diseases portfolio, 9.2% in Neuroscience, and 7.3% in Oncology; Somatuline® (lanreotide) sales grew by 5.6%, while all other products, excluding Somatuline, achieved double-digit sales growth at 12.2% FY 2024 core operating income of €1,109m, growing 10.8% as reported, with core operating margin of 32.6% of total sales Continued pipeline expansion in 2024, with significant regulatory approvals, addition of several preclinical therapies with global rights and innovative modalities, and a late-stage asset Four key regulatory and clinical milestones expected in 2025, including the Proof-of-Concept data readout for the Long-Acting Neurotoxin (LANT) Financial guidance2 for 2025 including total sales growth greater than 5.0%3 at CER, and core operating margin greater than 30.0% of total sales, based on accelerated sales growth of the ex-Somatuline portfolio and assuming negative impact on Somatuline sales due to increased generic competition in the U.S. and Europe PARIS, FRANCE, 13 February 2025 – Ipsen (Euronext: IPN; ADR: IPSEY), a global specialty-care biopharmaceutical company, today presents its financial results for the full year 2024. “Ipsen delivered solid results and advanced its pipeline in 2024, laying a strong foundation for sustained growth,” said David Loew, Chief Executive Officer, Ipsen. “With the successful global rollout of Iqirvo and Bylvay, and the U.S. launch of Onivyde, alongside multiple business development deals adding several innovative assets, we are well positioned to execute our strategic roadmap. This year, we look forward to achieving key milestones, including the first data readout for the Long-Acting Neurotoxin (LANT), and further expand and progress our pipeline across all three therapeutic areas to bring promising new medicines to patients.” Pipeline Progress Significant regulatory milestones were achieved in 2024, including FDA approval of Onivyde® (irinotecan) for first-line pancreatic ductal adenocarcinoma (PDAC), along with accelerated U.S. approval and European approval for Iqirvo® (elafibranor), respectively. Additionally, Kayfanda® (odevixibat) was approved for Alagille syndrome (ALGS) in the E.U. The company also opted-in for the CABINET Phase III study of Cabometyx® (cabozantinib) in patients with advanced neuroendocrine tumors (NETs), with study results presented at the 2024 European Society for Medical Oncology (ESMO) Congress and published in the New England Journal of Medicine. An IND application was filed for IPN01194, an ERK inhibitor, advancing the potential medicine into clinical development with a Phase I/IIa trial in advanced solid tumors. Ipsen improved further the depth and breadth of its pipeline by adding five preclinical innovative therapies with global rights and new modalities, and an ex-U.S. licensing agreement with DayOne Biopharmaceuticals for the late-stage oncology asset tovorafenib, an oral RAF inhibitor for pediatric low-grade glioma. Two global licensing agreements for Antibody Drug Conjugate (ADC) in oncology with Sutro Biopharma and Foreseen Biotechnology were signed. An extension of the oncology partnership with Marengo Therapeutics to include TriSTAR, a next-generation precision T-cell engager was completed, as well as more recently, in the fourth quarter, a global licensing agreement with Biomunex for a preclinical novel T-cell engager (TCE). A collaboration with Skyhawk Therapeutics to develop RNA-modulating small molecules for rare neurological diseases was also signed during the year. Ipsen executed several divestments in 2024, including the sale of Increlex® (mecasermin injection) to Eton Pharmaceuticals and the sale of its rare pediatric disease Priority Review Voucher. Environmental, Social and Governance Ipsen took important steps in 2024 in delivering its ambitious sustainability strategy. The company continued to integrate sustainability across its operations. From reducing its environmental footprint to advancing patient access and fostering a strong workplace culture, the company increased its commitment to driving progress for patients, employees, communities, and the planet. Our sustainability efforts were recognized across multiple environment initiatives. The company achieved a 45% reduction in Scopes 1 & 2 greenhouse gas emissions and a 25% reduction in Scope 3, fully in line with its 2030 targets (versus 2019 baseline). Significant efforts were made to engage suppliers and third parties in Ipsen’s sustainability roadmap including the first-ever “Ipsen Supplier Sustainability Day”. Following an intensive transformation project, 99.8% of Ipsen’s global electricity now comes from renewable sources. Through the Fleet for Future project, the company continues to advance sustainable transportation, with 43% of its total company’s fleet now electric vehicles as of 2024. We remain committed to gender balance in leadership, with women now representing 55% of the Global Leadership Team. 2025 Upcoming Milestones Ipsen anticipates several key milestones across its portfolio in 2025, including: Cabometyx (CABINET trial) – Regulatory decision in Europe for advanced neuroendocrine tumors (NETs), including pancreatic (pNETs) and extra-pancreatic (epNETs) neuroendocrine tumors Tovorafenib (FIREFLY-1 trial) – Regulatory submission in Europe for pediatric low-grade glioma Fidrisertib (FALKON trial) – Readout of the pivotal Phase IIb trial in fibrodysplasia ossificans progressiva (FOP) LANT88(LANTIC trial) – Proof-of-concept data readout, evaluating its potential in aesthetics These milestones reinforce Ipsen’s commitment to advancing innovative therapies and expanding treatment options for patients worldwide. 2025 Financial Guidance Ipsen has set for FY 2025 the following financial guidance, which excludes any impact from potential late-stage (Phase III clinical development or later) business development transactions: Total sales growth greater than 5.0%, at constant currency. Based on the average level of exchange rates in January 2025, a favorable effect on total sales of around 1% 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 on total sales and core operating margin is based on accelerated sales growth of the ex-Somatuline portfolio and assumes negative impact on Somatuline sales due to increased generic competition in the U.S. and Europe. Consolidated financial statements The Board of Directors approved the consolidated financial statements on 12 February 2025. The consolidated financial statements have been audited and the Statutory Auditors’ report is in the process of being published. Ipsen’s comprehensive audited financial statements will be available in due course on ipsen.com (regulated-information section). Conference call A conference call and webcast for investors and analysts will begin today at 1pm CET. Participants can access the call and its details by registering here; webcast details can be found here. Calendar Ipsen intends to publish its Q1 2025 sales on April 16th, 2025. Notes All financial figures are in € millions (€m), unless otherwise noted. The performance shown in this announcement covers the twelve-month period to 31 December 2024 (FY 2024) and the three-month period to 31 December 2024 (Q4 2024), compared to the twelve-month period to 31 December 2023 (FY 2023) and the three-month period to 31 December 2023 (Q4 2023), respectively, unless stated otherwise. The commentary is based on the performance in FY 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 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 Investors Media 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 Excluding any impact from potential late-stage (Phase III clinical development or later) external-innovation transactions.3 Based on the average level of exchange rates in Jan 2024, a favorable effect on total sales of about 1% from currencies is expected. 4 Extract of consolidated results. The Company’s auditors performed a limited review of the condensed consolidated financial statements.5 Including an impairment loss of €279m (or €2,33 /share) related to Sohonos, reflecting lower revised sales following lower patient uptake.6 Dividend related to the current financial year to be paid the following year.7 Decided by the Ipsen S.A. Board of Directors and to be proposed at the annual shareholders’ meeting on 21 May 2025. 8 Long-acting neurotoxin Attachment Ipsen PR_FY 2024 – Results announcement_13022025 FY 2024 total sales growth of 9.9% at CER1, or 8.7% as reported, with growth driven by strong performance across all therapeutic areas, including a 67.4% increase in the Rare Diseases portfolio, 9.2% in Neuroscience, and 7.3% in Oncology; Somatuline® (lanreotide) sales grew by 5.6%, while all other products, excluding Somatuline, achieved double-digit sales growth at 12.2% FY 2024 core operating income of €1,109m, growing 10.8% as reported, with core operating margin of 32.6% of total sales Continued pipeline expansion in 2024, with significant regulatory approvals, addition of several preclinical therapies with global rights and innovative modalities, and a late-stage asset Four key regulatory and clinical milestones expected in 2025, including the Proof-of-Concept data readout for the Long-Acting Neurotoxin (LANT) Financial guidance2 for 2025 including total sales growth greater than 5.0%3 at CER, and core operating margin greater than 30.0% of total sales, based on accelerated sales growth of the ex-Somatuline portfolio and assuming negative impact on Somatuline sales due to increased generic competition in the U.S. and Europe PARIS, FRANCE, 13 February 2025 – Ipsen (Euronext: IPN; ADR: IPSEY), a global specialty-care biopharmaceutical company, today presents its financial results for the full year 2024. “Ipsen delivered solid results and advanced its pipeline in 2024, laying a strong foundation for sustained growth,” said David Loew, Chief Executive Officer, Ipsen. “With the successful global rollout of Iqirvo and Bylvay, and the U.S. launch of Onivyde, alongside multiple business development deals adding several innovative assets, we are well positioned to execute our strategic roadmap. This year, we look forward to achieving key milestones, including the first data readout for the Long-Acting Neurotoxin (LANT), and further expand and progress our pipeline across all three therapeutic areas to bring promising new medicines to patients.” Pipeline Progress Significant regulatory milestones were achieved in 2024, including FDA approval of Onivyde® (irinotecan) for first-line pancreatic ductal adenocarcinoma (PDAC), along with accelerated U.S. approval and European approval for Iqirvo® (elafibranor), respectively. Additionally, Kayfanda® (odevixibat) was approved for Alagille syndrome (ALGS) in the E.U. The company also opted-in for the CABINET Phase III study of Cabometyx® (cabozantinib) in patients with advanced neuroendocrine tumors (NETs), with study results presented at the 2024 European Society for Medical Oncology (ESMO) Congress and published in the New England Journal of Medicine. An IND application was filed for IPN01194, an ERK inhibitor, advancing the potential medicine into clinical development with a Phase I/IIa trial in advanced solid tumors. Ipsen improved further the depth and breadth of its pipeline by adding five preclinical innovative therapies with global rights and new modalities, and an ex-U.S. licensing agreement with DayOne Biopharmaceuticals for the late-stage oncology asset tovorafenib, an oral RAF inhibitor for pediatric low-grade glioma. Two global licensing agreements for Antibody Drug Conjugate (ADC) in oncology with Sutro Biopharma and Foreseen Biotechnology were signed. An extension of the oncology partnership with Marengo Therapeutics to include TriSTAR, a next-generation precision T-cell engager was completed, as well as more recently, in the fourth quarter, a global licensing agreement with Biomunex for a preclinical novel T-cell engager (TCE). A collaboration with Skyhawk Therapeutics to develop RNA-modulating small molecules for rare neurological diseases was also signed during the year. Ipsen executed several divestments in 2024, including the sale of Increlex® (mecasermin injection) to Eton Pharmaceuticals and the sale of its rare pediatric disease Priority Review Voucher. Environmental, Social and Governance Ipsen took important steps in 2024 in delivering its ambitious sustainability strategy. The company continued to integrate sustainability across its operations. From reducing its environmental footprint to advancing patient access and fostering a strong workplace culture, the company increased its commitment to driving progress for patients, employees, communities, and the planet. Our sustainability efforts were recognized across multiple environment initiatives. The company achieved a 45% reduction in Scopes 1 & 2 greenhouse gas emissions and a 25% reduction in Scope 3, fully in line with its 2030 targets (versus 2019 baseline). Significant efforts were made to engage suppliers and third parties in Ipsen’s sustainability roadmap including the first-ever “Ipsen Supplier Sustainability Day”. Following an intensive transformation project, 99.8% of Ipsen’s global electricity now comes from renewable sources. Through the Fleet for Future project, the company continues to advance sustainable transportation, with 43% of its total company’s fleet now electric vehicles as of 2024. We remain committed to gender balance in leadership, with women now representing 55% of the Global Leadership Team. 2025 Upcoming Milestones Ipsen anticipates several key milestones across its portfolio in 2025, including: Cabometyx (CABINET trial) – Regulatory decision in Europe for advanced neuroendocrine tumors (NETs), including pancreatic (pNETs) and extra-pancreatic (epNETs) neuroendocrine tumors Tovorafenib (FIREFLY-1 trial) – Regulatory submission in Europe for pediatric low-grade glioma Fidrisertib (FALKON trial) – Readout of the pivotal Phase IIb trial in fibrodysplasia ossificans progressiva (FOP) LANT88(LANTIC trial) – Proof-of-concept data readout, evaluating its potential in aesthetics These milestones reinforce Ipsen’s commitment to advancing innovative therapies and expanding treatment options for patients worldwide. 2025 Financial Guidance Ipsen has set for FY 2025 the following financial guidance, which excludes any impact from potential late-stage (Phase III clinical development or later) business development transactions: Total sales growth greater than 5.0%, at constant currency. Based on the average level of exchange rates in January 2025, a favorable effect on total sales of around 1% 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 on total sales and core operating margin is based on accelerated sales growth of the ex-Somatuline portfolio and assumes negative impact on Somatuline sales due to increased generic competition in the U.S. and Europe. Consolidated financial statements The Board of Directors approved the consolidated financial statements on 12 February 2025. The consolidated financial statements have been audited and the Statutory Auditors’ report is in the process of being published. Ipsen’s comprehensive audited financial statements will be available in due course on ipsen.com (regulated-information section). Conference call A conference call and webcast for investors and analysts will begin today at 1pm CET. Participants can access the call and its details by registering here; webcast details can be found here. Calendar Ipsen intends to publish its Q1 2025 sales on April 16th, 2025. Notes All financial figures are in € millions (€m), unless otherwise noted. The performance shown in this announcement covers the twelve-month period to 31 December 2024 (FY 2024) and the three-month period to 31 December 2024 (Q4 2024), compared to the twelve-month period to 31 December 2023 (FY 2023) and the three-month period to 31 December 2023 (Q4 2023), respectively, unless stated otherwise. The commentary is based on the performance in FY 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 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 Investors Media 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 Excluding any impact from potential late-stage (Phase III clinical development or later) external-innovation transactions.3 Based on the average level of exchange rates in Jan 2024, a favorable effect on total sales of about 1% from currencies is expected. 4 Extract of consolidated results. The Company’s auditors performed a limited review of the condensed consolidated financial statements.5 Including an impairment loss of €279m (or €2,33 /share) related to Sohonos, reflecting lower revised sales following lower patient uptake.6 Dividend related to the current financial year to be paid the following year.7 Decided by the Ipsen S.A. Board of Directors and to be proposed at the annual shareholders’ meeting on 21 May 2025. 8 Long-acting neurotoxin Attachment Ipsen PR_FY 2024 – Results announcement_13022025

Phase 2Drug ApprovalLicense out/inPhase 3

100 Deals associated with Irinotecan Hydrochloride Lioposome

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KEGG	Wiki	ATC	Drug Bank
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R&D Status

Approved

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Indication	Country/Location	Organization	Date
Pancreatic adenocarcinoma metastatic	Australia	Servier Laboratories (Australia) Pty Ltd.	19 Dec 2016
Pancreatic adenocarcinoma	European Union	Les Laboratoires Servier SAS	14 Oct 2016
Pancreatic adenocarcinoma	Iceland	Les Laboratoires Servier SAS	14 Oct 2016
Pancreatic adenocarcinoma	Liechtenstein	Les Laboratoires Servier SAS	14 Oct 2016
Pancreatic adenocarcinoma	Norway	Les Laboratoires Servier SAS	14 Oct 2016
Pancreatic Cancer	United States	Ipsen Biopharmaceuticals, Inc.	22 Oct 2015

Developing

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Indication	Highest Phase	Country/Location	Organization	Date
Metastatic Pancreatic Ductal Adenocarcinoma	NDA/BLA	United States	Ipsen SA	14 Jun 2023
Extensive stage Small Cell Lung Cancer	Phase 3	United States	Ipsen SA	25 Apr 2018
Extensive stage Small Cell Lung Cancer	Phase 3	China	Ipsen SA	25 Apr 2018
Extensive stage Small Cell Lung Cancer	Phase 3	Australia	Ipsen SA	25 Apr 2018
Extensive stage Small Cell Lung Cancer	Phase 3	Belgium	Ipsen SA	25 Apr 2018
Extensive stage Small Cell Lung Cancer	Phase 3	Brazil	Ipsen SA	25 Apr 2018
Extensive stage Small Cell Lung Cancer	Phase 3	France	Ipsen SA	25 Apr 2018
Extensive stage Small Cell Lung Cancer	Phase 3	Germany	Ipsen SA	25 Apr 2018
Extensive stage Small Cell Lung Cancer	Phase 3	Hungary	Ipsen SA	25 Apr 2018
Extensive stage Small Cell Lung Cancer	Phase 3	Italy	Ipsen SA	25 Apr 2018

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT06766266 (Pubmed) Manual	Phase 1	Non-Muscle Invasive Bladder Urothelial Carcinoma	-	Irinotecan Liposome+Epirubicin	zlcvaireat(jfgboyvtjq) = feasible vddlfluvjp (giuznpbqci ) View more	Positive	10 Jul 2025
NAPOLI-1 (ESMO_GI2025)	Not Applicable	Pancreatic Cancer Second line	27	Nanoliposomal Irinotecan + Fluorouracil	lksxggqhpm(doixawkkzd) = 52% evltfbkvws (jnkqgazklo ) View more	Positive	03 Jul 2025
TriNetX Global Collaborative Network (ESMO_GI2025)	Not Applicable	Pancreatic Cancer Second line	1,520	Nanoliposomal irinotecan plus fluorouracil (NI)	abjwkdxdpq(csweeelwdk) = qbgiztzwpq hgixrfrxgf (reafobernc )	Negative	03 Jul 2025
TriNetX Global Collaborative Network (ESMO_GI2025)	Not Applicable	Pancreatic Cancer Second line	1,520	Fluoropyrimidine-based chemotherapy plus oxaliplatin (OF)	abjwkdxdpq(csweeelwdk) = hzvcxffykx hgixrfrxgf (reafobernc )	Negative	03 Jul 2025
ESMO_GI2025	Not Applicable	Metastatic Pancreatic Ductal Adenocarcinoma Second line	332	FOLFOX/XELOX	xdgemfluwx(ketonnglsu) = more frequent in the 5-FU+Nal-IRI group tuvlpobfib (vifhhblmbn ) View more	Positive	03 Jul 2025
NAPOLI-1 (ESMO_GI2025)	Phase 3	Second line	55	Nal-IRI + 5-FU/LV	qizfcaytny(vovykdzhej) = occurred in 33% (n = 6) of pts without MA and 100% (n = 7) of pts with ascites grade 3 zfribnydsb (mqeibullce ) View more	Negative	03 Jul 2025
NCT04617457 (HOLIPANC, ASCO2025) Manual	Phase 2	Pancreatic Cancer Neoadjuvant	56	Nal-IRIFOX (liposomal irinotecan, oxaliplatin, 5-fluorouracil/folinic acid)	pnadyybzdf(jkxzizzplr) = fksspmorxt utdejtcpvn (rqaqynatbg ) View more	Positive	30 May 2025
NCT06434090 (Phase I/II trial, ASCO2025)	Phase 1/2	Metastatic Colorectal Carcinoma	12	Liposomal irinotecan 70 mg/m2	qzabwdumna(rwncamfifl) = dhhindjifu myxnrhxeip (zgfdvkoxdl, 34.9% - 96.8%) View more	Positive	30 May 2025
NCT06434090 (Phase I/II trial, ASCO2025)	Phase 1/2	Metastatic Colorectal Carcinoma	12	Liposomal irinotecan 80 mg/m2	bmtnocnzhr(xygezoiwli) = rjdmtqrvjp qkcgfuavxr (dmklbspeqm )	Positive	30 May 2025
NAPOLI (ASCO2025)	Phase 3	Metastatic Pancreatic Ductal Adenocarcinoma	1,006	nanoliposomal irinotecan (Nal-IRI + FU + LV)	cncnsswgof(hzdtgnodox) = lcenoeibnt vsuwnhqjwb (jyohafuegc ) View more	Positive	30 May 2025
NAPOLI (ASCO2025)	Phase 3	Metastatic Pancreatic Ductal Adenocarcinoma	1,006	nanoliposomal irinotecan (FU + LV)	cncnsswgof(hzdtgnodox) = cncdmzwkuz vsuwnhqjwb (jyohafuegc ) View more	Positive	30 May 2025
ChiCTR2400087003 (ASCO2025)	Phase 2	Extensive stage Small Cell Lung Cancer Second line	13	Liposomal irinotecan + Cadonilimab	jcckfhhqxv(yrhlfaakfe) = ptkoxnolvx twfedxrrtc (olmyfwezqz ) View more	Positive	30 May 2025
NAPOLI-1 (AACR2025)	Phase 3	Pancreatic adenocarcinoma Adjuvant	-	Nanoliposomal irinotecan plus 5-fluorouracil/leucovorin (nal-IRI/5-FU/LV)	dfpcnbawvm(volmnslmgb) = yjwqdbrzkr qgnbnqdupg (pffadrshvv )	Positive	29 Apr 2025
NAPOLI-1 (AACR2025)	Phase 3	Pancreatic adenocarcinoma Adjuvant	-	Non-liposomal irinotecan	dfpcnbawvm(volmnslmgb) = jwbgaexdow qgnbnqdupg (pffadrshvv )	Positive	29 Apr 2025

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