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Last update 22 Oct 2026

Irinotecan Hydrochloride

Last update 22 Oct 2026

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

Drug Type

Small molecule drug

Synonyms

ASC-201 - Ascelia Pharma, IHT, irinotecan

+ [39]

Target

Top I

Action

inhibitors

Mechanism

TOP1 inhibitors(DNA topoisomerase I inhibitors)

Therapeutic Areas

NeoplasmsImmune System DiseasesDigestive System Disorders+ [8]

Active Indication

Childhood Malignant Solid NeoplasmIntestinal NeoplasmsColonic Cancer+ [38]

Inactive Indication

Adenocarcinoma of EsophagusAdenocarcinoma of small intestineAdvanced breast cancer+ [61]

Originator Organization

Yakult Honsha Co., Ltd.

Active Organization

Eli Lilly Japan KK

Yakult Honsha Co., Ltd.

Edison Oncology Holding Corp.

+ [13]

Inactive Organization

Sanofi

Bristol Myers Squibb Co.Hoffmann-La Roche, Inc.

+ [16]

License Organization

Hanmi Pharmaceutical Co., Ltd.

Dr. Reddy's Laboratories Ltd.

SAM CHUN DANG PHARM Co., Ltd.

+ [7]

Drug Highest PhaseApproved

First Approval Date

Japan (19 Jan 1994),

Non-Small Cell Lung CancerOvarian CancerPancreatic Cancer+ [3]

RegulationAccelerated Approval (United States), Priority Review (China)

Structure/Sequence

Molecular FormulaC33H45ClN4O9

InChIKeyKLEAIHJJLUAXIQ-JDRGBKBRSA-N

CAS Registry136572-09-3

1,858

Clinical Trials associated with Irinotecan Hydrochloride

JPRN-UMIN000006999

/ CompletedPhase 2IIT

Phase II randomized trail of Gemcitabine and Irinotecan in Patients with Platinum Refractory and Resistant Mullerian carcinoma (tailored dose vs optimal dose chemotherapy)

Start Date31 Dec 2046

Sponsor / Collaborator-

NCT07748611

/ Not yet recruitingPhase 2IIT

SMART-PANC: SMAD4 Tailored Neoadjuvant Therapy for Pancreatic Cancer, A Phase ll Non-Randomized, Single Center Pilot Study

This phase II trial tests the impact of using SMAD4-mutant status to personalize chemotherapy in treating patients with pancreatic ductal adenocarcinoma (PDAC) that can be removed by surgery (resectable) or that may be between resectable and unresectable (borderline resectable) before undergoing surgery (neoadjuvant). PDAC is one of the most aggressive and fastest growing tumors. SMAD4, a tumor suppressor gene, acts like a brake on cell growth and helps to prevent tumor cells from growing. However, losing it makes the tumor more aggressive and often resistant to standard of care (SOC) treatments regimens, such as gemcitabine with nab-paclitaxel and fluorouracil, leucovorin, irinotecan, and oxaliplatin (FOLFIRINOX). Gemcitabine is a chemotherapy drug that blocks the cells from making deoxyribonucleic (DNA) and may kill tumor cells. Paclitaxel is in a class of medications called antimicrotubule agents. It stops tumor cells from growing and dividing and may kill them. Nab-paclitaxel is an albumin-stabilized nanoparticle formulation of paclitaxel which may have fewer side effects and work better than other forms of paclitaxel. Fluorouracil, a type of antimetabolite, stops cells from making DNA and it may kill tumor cells. Leucovorin is a form of folic acid. It is a type of chemoprotective agent and a type of chemosensitizing agent. Irinotecan is in a class of antineoplastic medications called topoisomerase I inhibitors. It blocks a certain enzyme needed for cell division and DNA repair and may kill tumor cells. Oxaliplatin is in a class of medications called platinum-containing antineoplastic agents. It damages the cell's DNA and may kill tumor cells. Instead of a one-sized fits all treatment approach with SOC therapies, matching therapy based on SMAD4 mutation status may use this specific genetic weakness against the tumor. This approach to neoadjuvant therapy may dramatically alter the path of treatment and improve outcomes, including complete resection rates, in patients with resectable or borderline resectable PDAC.

Start Date03 Feb 2027

Sponsor / Collaborator

Northwestern University

[+1]

NCT07363408

/ Not yet recruitingPhase 1IIT

A Phase I Clinical Trial of Ivonescimab and ADG126, Alone, and in Combination With 5-FU/LV or FOLFIRI in MSS Advanced/Metastatic Colorectal Cancer

This phase I trial studies the safety, side effects, and best dose of ADG126, in combination with ivonescimab alone, in combination with ivonescimab, leucovorin, and fluorouracil, or in combination with ivonescimab and leucovorin, fluorouracil, and irinotecan (FOLFIRI regimen) in treating patients with microsatellite stable (MSS) colorectal cancer that may have spread from where it first started to nearby tissue, lymph nodes, or distant parts of the body (advanced) or has spread from where it first started (primary site) to other places in the body (metastatic). Immunotherapy with monoclonal antibodies, such as ivonescimab and ADG126, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Ivonescimab may also stop or slow the cancer by blocking the growth of new blood vessels necessary for tumor growth. Leucovorin calcium is a type of drug called a folic acid analog, which means it is similar to the vitamin folic acid. It is used in combination with certain chemotherapy drugs to enhance their ability to kill tumor cells or to lessen their harmful side effects. Fluorouracil is a type of chemotherapy called an antimetabolite, which is a drug that mimics a natural chemical and prevents its use in cells. It interferes with the production of a key component of deoxyribonucleic acid (DNA), which prevents the DNA from copying itself. This causes tumor cells and other rapidly dividing cells to die. Fluorouracil also gets incorporated into ribonucleic acid (RNA) and DNA, disrupting critical cell functions. Irinotecan is in a class of antineoplastic medications called topoisomerase I inhibitors. It blocks a certain enzyme needed for cell division and DNA repair and may kill cancer cells. Giving ADG126 with ivonescimab, with or without leucovorin and fluorouracil or FOLFIRI regimen, may be safe in treating patients with MSS advanced/metastatic colorectal cancer.

Start Date01 Dec 2026

Sponsor / Collaborator

City of Hope National Medical Center

[+1]

100 Clinical Results associated with Irinotecan Hydrochloride

100 Translational Medicine associated with Irinotecan Hydrochloride

100 Patents (Medical) associated with Irinotecan Hydrochloride

8,325

Literatures (Medical) associated with Irinotecan Hydrochloride

01 Nov 2026·INTERNATIONAL JOURNAL OF BIOLOGICAL MACROMOLECULES

Angelica sinensis polysaccharide ameliorates chemotherapy-induced intestinal mucositis by regulating gut microbiota-mediated innate immunity and short-chain fatty acid production in Drosophila and mice

Article

Author: He, Jianzheng ; Wu, Jinhan ; Liu, Menglu ; Wang, Junlong ; Wang, Yan ; Xiu, Minghui ; Li, Kexin ; Zhang, Yongxuan ; Yue, Shiqi

Intestinal mucositis is one of the most debilitating side effects of chemotherapeutic agents. Angelica sinensis polysaccharide (ASP), the crucial active ingredient of Angelica sinensis, has been reported to possess anti-colitis activity. However, the efficacy of ASP against chemotherapy-induced intestinal mucositis (CIM) remain to be clarified. The aim of this study using Drosophila melanogaster and mouse models was to investigate the potential effect of ASP on intestinal mucositis and its underlying mechanism. ASP significantly alleviated overall physiological and intestinal damage caused by CPT-11 in adult flies, including increased survival rate and intestinal length, improved digestive capacity, restored intestinal acid-base balance and reduced death of intestinal epithelial cells. Near Infrared (NIR) imaging indicated that ASP was absorbed through the intestine and metabolized via the hepatic and renal systems in mice. Furthermore, ASP reduced intestinal damage and restored the intestinal barrier function in CPT-11 treated mice, including increased intestinal length, elevated levels of ZO-1 and an increased number of goblet cells. Mechanistically, ASP markedly down-regulated the over-activated innate immunity by inhibiting the Toll-IMD and TLR4/NF-κB/MyD88 signaling pathways in CPT-11 induced flies and mice. Besides, ASP also exerted a protective effect against structural damage to the spleen induced by CPT-11. Moreover, ASP ameliorated gut microbiota imbalances and increased the levels of short-chain fatty acids (SCFAs), particularly propionate and butyrate. Fecal microbiota transplantation (FMT) further confirmed that ASP could modulate gut microbiota and protect against intestinal mucositis in mice. Collectively, these results demonstrate that ASP effectively ameliorates CIM and has the potential to serve as a novel adjunctive therapy to CPT-11.

01 Oct 2026·PHYTOMEDICINE

Xiao-Chai-Hu-Tang alleviates irinotecan-induced diarrhea via promoting bile acid reabsorption and decreasing focal adhesion kinase signaling revealed by metabolomics and proteomics

Article

Author: Zhu, Lijun ; Rao, Jinsong ; He, Zhuoru ; Cai, Yu ; Qiu, Huawei ; Liu, Zhongqiu ; Chen, Yongxv ; Yao, Ye ; Zhou, Runze ; Ma, Tongyang ; Zhou, Jialin

BACKGROUND:

Irinotecan (CPT-11) is widely used for colorectal cancer treatment, with delayed-onset diarrhea as its primary side effect. Xiao-Chai-Hu-Tang (XCHT) has been clinically observed to alleviate chemotherapy-induced diarrhea, but its underlying mechanisms remain unclear.

PURPOSE:

This study aimed to elucidate the regulatory mechanisms through which XCHT alleviated CPT-11-induced diarrhea using an integrated multi-omics approach.

METHODS:

The chemical components of XCHT were detected using UHPLC-QE-Orbitrap-MS, and 12 active components were quantified via UHPLC-QQQ-MS/MS to ensure quality stability. CPT-11-induced diarrheal mice were established to evaluate the therapeutic effects of XCHT. Differential metabolites in liver and intestinal tissues among control, CPT-11, and XCHT mice were analyzed by untargeted metabolomics, followed by molecular network analysis using IPA. Bile acids were quantified using targeted UHPLC-QQQ-MS/MS method. Proteomics of colon tissues identified differentially expressed proteins, with functional enrichment conducted via GO and KEGG. Western blot and flow cytometry were used to validate the potential biopathway.

RESULTS:

Total 1108 chemical components were identified in XCHT, mainly including flavonoids, terpenoids and phenylpropanoids. XCHT significantly improved diarrhea symptoms and reduced intestinal inflammation. Metabolomics revealed 117 and 53 differential metabolites in the liver and intestine, respectively, with both tissues showing alterations in bile acid metabolism. Targeted bile acid analysis showed that CPT-11 inhibited the synthesis and uptake of bile acids in the liver and enhanced bile excretion, resulting in an increase in bile acids in the gallbladder. Meanwhile, CPT-11 impaired the reabsorption of bile acids in the intestine, leading to a decrease in total bile acid levels, reduction of circulating bile acids, and accumulation of conjugated bile acids in the colon. XCHT reversed these abnormal phenomena, restoring the intestinal bile acid homeostasis. Proteomics identified 86 differentially expressed proteins in colon tissues, with significant enrichment in 'Focal adhesion' pathway. Further verification indicated that CPT-11 abnormally activated the focal adhesion kinase (FAK) and its downstream RhoA/ROCK pathways, then reduced tight junction proteins ZO-1 and Occludin, damaging the intestinal barrier. XCHT inhibited this pathway, restored the expression of tight junction proteins, balanced Th1/Th2 differentiation, and alleviated intestinal inflammation. Cholestyramine, a bile acid chelator, also alleviated CPT-11-induced diarrhea by reducing the accumulation of bile acids in the intestine, confirming the key role of bile acids in the treatment of CPT-11-induced diarrhea.

CONCLUSION:

This study revealed that the combined administration of XCHT could restore bile acid reabsorption, reduced the accumulation of conjugated bile acids in the colon, inhibited the abnormally activated FAK-RhoA/ROCK pathway, improved intestinal epithelial barrier function and alleviate diarrhea. These findings provide a new entry point for the treatment of chemotherapy-induced diarrhea (CID) and offer data support for the combined medication regimen of XCHT and CPT-11.

03 Jul 2026·Expert Opinion On Drug Safety

Drug-induced nasal septum perforation: a disproportionality analysis of the FDA adverse event reporting system database

Article

Author: Xie, Chubo ; Wang, Yuewu ; Qiu, Qianhui ; Liu, Yitong ; Jiang, Zhilin ; Cui, Yi ; Sun, Fang ; Zhou, Suizi ; Wang, Keshuang

BACKGROUND:

Nasal septum perforation represents a significant clinical concern, with limited investigations into the role of medications in its etiology. This study utilizes the FDA Adverse Event Reporting System (FAERS) database to identify the drugs associated with nasal septum perforation and assess their risk.

RESEARCH DESIGN AND METHODS:

This retrospective pharmacovigilance study analyzed drug-induced nasal septum perforation data from January 2004 to December 2023. Disproportionality analysis using reporting odds ratio (ROR) assessed drug associations with nasal septum perforation.

RESULTS:

For 552 identified cases, the most commonly reported drugs were bevacizumab (n = 56), fluticasone propionate (n = 50), methotrexate (n = 34), hydrocodone and acetaminophen (n = 22), and paclitaxel (n = 17). Twenty-six drugs showed positive risk signals, with the top five being azelastine hydrochloride and fluticasone propionate (ROR = 173.82), beclomethasone dipropionate (ROR = 90.91), oxymetazoline (ROR = 53.77), desmopressin (ROR = 51.43), and leucovorin (ROR = 42.83). Intriguingly, 18 of these drugs did not list nasal septum perforation as a known side effect.

CONCLUSION:

This study provides a comprehensive overview of drug-induced nasal septum perforation from a pharmacovigilance perspective, highlighting the need for further research to clarify these associations and update drug safety information to reduce patient risk.

630

News (Medical) associated with Irinotecan Hydrochloride

20 Oct 2026

·www.globenewswire.com

Oncolytics Biotech® to Present REO 033 Trial in Progress Abstract at ESMO Showcasing Pelareorep’s Potential in Colorectal Cancer

Study launched earlier this year is expected to confirm previous efficacy signal in 2L KRAS-mutant MSS colorectal cancer study that saw 17 months PFS vs. 6 months for the historical standard of careSAN DIEGO, Oct. 20, 2026 (GLOBE NEWSWIRE) -- Oncolytics Biotech® Inc. (Nasdaq: ONCY) (“Oncolytics” or the “Company”), a clinical-stage immunotherapy company developing pelareorep, today announced the Company is presenting a trial-in-progress abstract and e-poster discussing the REO 033 study at the European Society for Medical Oncology (“ESMO”) Congress 2026. REO 033 is a randomized study evaluating pelareorep in combination with folinic acid, fluorouracil, and irinotecan (“FOLFIRI”) and bevacizumab versus standard-of-care FOLFIRI and bevacizumab as second-line treatment for RAS-mutant, microsatellite-stable (“MSS”) metastatic colorectal cancer (“mCRC”) patients (link to study on ClinicalTrials.gov). The study is designed to validate the encouraging efficacy signals observed in REO 022 while generating the controlled clinical data necessary to support future regulatory interactions and potential registration. Abstract Title: Phase 2 randomized, controlled study of pelareorep combined with 5-fluorouracil/leucovorin/ irinotecan (FOLFIRI) + bevacizumab (bev) for the second-line treatment of RAS-mutated, microsatellite-stable (MSS) metastatic colorectal cancerPresentation Type: e-PosterPresentation Number: 994eTiP Highlights from the REO 033 trial-in-progress abstract include: The randomized, controlled, open-label study will evaluate patients with RAS-mutated, MSS mCRC who have progressed on first-line oxaliplatin-based therapy.Thirty patients per arm will be randomized to receive either: 1) pelareorep + FOLFIRI + bevacizumab, or 2) FOLFIRI + bevacizumab.The primary endpoint is objective response rate (“ORR”). Secondary endpoints include other efficacy assessments, such as overall survival (“OS”), progression-free survival (“PFS”), disease control rate, duration of response (“DOR”), and safety and tolerability.Blood and tumor samples are being collected for biomarker evaluations. Recent U.S. Food and Drug Administration interactions have provided the opportunity to initiate a pivotal expansion of the REO 033 study (Part B) that would build upon the trial currently enrolling (Part A) and could support an accelerated approval submission based on ORR and a full approval based on PFS. A previous study, REO 022, evaluated second-line KRAS-mutant, MSS mCRC patients with treatment histories similar to those of patients enrolled in the current study. In that study, the ORR, median PFS, median OS, and median DOR were more than doubled in patients treated with pelareorep + FOLFIRI/bevacizumab compared to historical outcomes for patients treated with FOLFIRI and bevacizumab alone.1-4 “I am thrilled to see the recent momentum in the REO 033 study among sites, physicians, and patients,” said Sanjay Goel, M.D., M.S., FASCO, Professor of Medicine at Rutgers Cancer Institute, and Lead Investigator. “Having been involved in the REO 022 study, I have a strong conviction that pelareorep can positively impact patients in a meaningful way, and this controlled randomized study will help prove that out.” Jared Kelly, Chief Executive Officer of Oncolytics, commented, “Second-line mCRC development is generally stalling because emerging therapies are not working well, especially in RAS-mutant MSS patients. With mCRC growing faster than almost every cancer in the U.S. and only minimally effective options for second-line patients, pelareorep has the potential in this controlled study to change the standard of care by improving what has been in place for the better part of a decade. It’s a multi-billion-dollar market that is being largely ignored because nothing works better than the standard of care. Adding pelareorep to the standard of care could double the efficacy while maintaining an acceptable safety profile. This is what drug development is all about.” About PelareorepPelareorep is an intravenously delivered, systemically active, investigational immunotherapy with a dual mechanism of action that selectively replicates in tumor cells while activating both innate and adaptive anti-tumor immune responses, including the upregulation of key inflammatory cytokines resulting in the formation of tertiary lymphoid structures and the expansion of tumor-infiltrating lymphocytes. It has been administered to over 1,200 patients, and clinical studies have demonstrated pelareorep’s potential to enhance the activity of checkpoint inhibitors and other anti-cancer therapies across multiple solid tumor types. About Oncolytics Biotech Inc.Oncolytics is a clinical-stage biotechnology company developing pelareorep, an investigational intravenously delivered double-stranded RNA immunotherapeutic agent. Pelareorep has demonstrated encouraging results in multiple first-line pancreatic cancer studies, two randomized Phase 2 studies in metastatic breast cancer, and early-phase studies in anal and colorectal cancer. It is designed to induce anti-cancer immune responses by converting immunologically inactive tumors to active through the activation of innate and adaptive immune responses. The Company is advancing pelareorep in combination with chemotherapy and/or checkpoint inhibitors in metastatic gastrointestinal cancers, where pelareorep has received Fast Track designation from the U.S. Food and Drug Administration for colorectal, anal, and pancreatic cancer. Oncolytics is actively pursuing strategic partnerships to accelerate development and maximize commercial impact. For more about Oncolytics, please visit: www.oncolyticsbiotech.com or follow the Company on LinkedIn and on X @oncolytics. References Goel S, et al. Elucidation of Pelareorep Pharmacodynamics in A Phase I Trial in Patients with KRAS-Mutated Colorectal Cancer. Mol Cancer Ther. 2020 May;19(5):1148-1156. doi: 10.1158/1535-7163.MCT-19-1117.Bennouna J. Lancet Oncol (14):29-37, 2013Iwamoto S. Ann Oncol. Jul;26(7):1427-33, 2015FDA grants accelerated approval to adagrasib with cetuximab for KRAS G12C–mutated colorectal cancer. Published June 21, 2024. Accessed April 28, 2026. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-adagrasib-cetuximab-kras-g12c-mutated-colorectal-cancer Forward-looking statementsThis press release contains forward-looking statements, within the meaning of Section 21E of the U.S. Securities Exchange Act of 1934, as amended, and forward-looking information under applicable Canadian securities laws (such forward-looking statements and forward-looking information are collectively referred to herein as “forward-looking statements”). Forward-looking statements contained in this press release include statements regarding beliefs as to the potential, registration, mechanism of action and benefits of pelareorep as a cancer therapeutic; the Company’s goals, strategies, and objectives; expectations around the design, milestones, anticipated timelines and expected outcomes for current and future studies; its belief in the clinical promise of pelareorep in anal, colorectal, pancreatic and other gastrointestinal cancers; U.S. Food and Drug Administration interactions and its anticipated outcomes; the Company’s goals and expectations for its potential registrational development path for pelareorep in multiple gastrointestinal cancers; and plans for future disclosure of clinical trial results. In any forward-looking statement in which Oncolytics expresses an expectation or belief as to future results, such expectations or beliefs are expressed in good faith and are believed to have a reasonable basis, but there can be no assurance that the statement or expectation or belief will be achieved. These statements involve known and unknown risks and uncertainties that may cause actual results to differ materially from those anticipated. These risks include, but are not limited to, regulatory outcomes, trial execution, financial resources, access to capital markets, and market dynamics. Please refer to Oncolytics’ public filings with securities regulators in the United States and Canada for more information. The Company assumes no obligation to update forward-looking statements, except as required by law. Company Contact Jon PattonDirector of IR & Communicationjpatton@oncolytics.com

ImmunotherapyClinical ResultPhase 2Fast TrackPhase 1

19 Oct 2026

·www.biospace.com

BioNTech to Showcase Oncology Late-Stage Pipeline Progress and Pumitamig Novel-Novel Combination Data at ESMO 2026

Late-breaking oral presentation will feature first data from the global ROSETTA Lung-107 Phase 2 clinical trial evaluating pumitamig, a PD-L1×VEGF-A bispecific immunomodulator , plus docetaxel in patients with metastatic non-small cell lung cancer following prior immunotherapy plus chemotherapy Global Phase 2 data for pumitamig plus elfetabart drozuntecan, a B7-H3-targeting antibody-drug conjugate, expand the body of evidence for this novel-novel combination in multiple advanced solid tumors Updates from across the pumitamig clinical development program underscore its pan-tumor potential, including in lung and breast cancer patients with brain metastases and patients with neuroendocrine neoplasms MAINZ, Germany, October 19, 2026 – BioNTech SE (Nasdaq: BNTX, “BioNTech” or “the Company”) will present new clinical data from its late-stage oncology pipeline with 16 abstracts spanning the Company’s tumor focus areas, including lung and breast cancer, at the European Society for Medical Oncology (“ESMO”) 2026 Congress held in Madrid, Spain, from October 23–27, 2026. Key oral presentations will focus on clinical data for pumitamig (BNT327/BMS986545), a PD-L1×VEGF-A bispecific immunomodulator being jointly developed with Bristol Myers Squibb (“BMS”), plus docetaxel in second-line metastatic non-small cell lung cancer (“NSCLC”) as well as pumitamig in combination with elfetabart drozuntecan (elfe-D or BNT324/DB-1311), a B7-H3-targeting antibody-drug conjugate (“ADC”) being jointly developed with Duality Biologics (Suzhou) Co. Ltd. (“DualityBio”), across several advanced solid tumors – complementing the data in lung cancer presented earlier this year. This new clinical data advances BioNTech’s differentiated novel-novel combination approach. Additional presentations for pumitamig and ADCs reflect BioNTech’s tumor-focused strategy aimed at addressing significant unmet medical needs across various stages of disease. “Sixteen abstracts spanning ten tumor types – this is our significant data presence at this year’s ESMO. It demonstrates momentum for our late-stage oncology pipeline across strategic key assets and tumor areas such as lung and breast cancer,” said Prof. Özlem Türeci, M.D., Co-Founder and Chief Medical Officer at BioNTech . “For our PD-L1xVEGF immunomodulator pumitamig, we will be sharing new data that highlights its pan-tumor potential, including in challenging treatment settings where patients still show limited benefit from current standard of care therapies. Our goal is to establish pumitamig as a potential backbone for future combination regimens across multiple tumor types, including novel-novel treatment combinations. At this year's conference, we will showcase the potential of this approach in two presentations featuring ADC combinations, aimed to further enhance outcomes for patients.” Highlights from BioNTech’s presentations at ESMO 2026: Pumitamig – an investigational PD-L1×VEGF-A bispecific immunomodulator: NSCLC: First data from the global ROSETTA Lung-107 Phase 2 clinical trial ( NCT06841055 ) evaluating pumitamig plus docetaxel in patients with metastatic NSCLC whose disease progressed after immunotherapy plus chemotherapy will be presented in a late-breaking oral presentation. This clinical trial extends evidence for pumitamig’s non-small cell lung cancer development program into the second-line setting where there remains high unmet medical need, representing the third global Phase 2 dataset in lung cancer. SCLC and TNBC with brain metastases: Results from a subgroup analysis of two Phase 2 clinical trials ( NCT06449209 ; NCT06449222 ) evaluating pumitamig plus chemotherapy in patients with small cell lung cancer (“SCLC”) or triple-negative breast cancer (“TNBC”) showed systemic efficacy generally consistent in patients with and without brain metastases. These early data suggest intracranial efficacy and add to the clinical evidence for pumitamig plus chemotherapy in tumor types where brain metastases often occur and remain challenging to treat as many systemic therapies have limited ability to cross the blood-brain barrier. 2L neuroendocrine neoplasms: First results from a Phase 2 clinical trial conducted in China ( NCT05879055 ) evaluating pumitamig plus FOLFIRI (folinic acid, fluorouracil, and irinotecan) chemotherapy in patients with unresectable neuroendocrine neoplasms after first-line platinum-based chemotherapy showed encouraging preliminary antitumor activity and a manageable safety profile. These data broaden the clinical evidence for pumitamig across diverse tumor types. Novel-novel combination of pumitamig and elfetabart drozuntecan, an investigational B7-H3-targeted ADC: Advanced solid tumors: First results from the global Phase 2 clinical trial ( NCT06953089 ) evaluating pumitamig in combination with elfetabart drozuntecan in patients with advanced solid tumors showed encouraging preliminary antitumor activity across multiple tumor types and a manageable safety profile, with minimal overlapping toxicities observed. These data support the potential of pumitamig as a backbone in novel treatment combinations, furthering BioNTech’s combination strategy across multiple tumor types with high unmet medical need. BioNTech is advancing a diversified oncology pipeline spanning next-generation immunomodulators, ADCs, and mRNA cancer immunotherapies, both as monotherapies and novel-novel combination approaches. With 13 ongoing Phase 3 trials as well as multiple novel-novel combination trials, BioNTech is focused on developing innovative approaches to address the challenges of cancer treatment among the Company’s tumor focus areas from early- to late-stage conditions. All abstracts are available through the ESMO website . Further information on BioNTech’s oncology pipeline can be accessed here . Full presentation details: Candidate Abstract Title Abstract Number/Presentation Details Pumitamig A global phase 2, open-label trial of pumitamig (PD-L1 × VEGF-A bsAb) + docetaxel post-chemoimmunotherapy in mNSCLC: First data from ROSETTA Lung-107 Abstract #LBA76 Rapid Oral Presentation NSCLC, Metastatic Oct 25, 2026: 8:30 - 10:00am CET Efficacy of pumitamig (PD-L1 × VEGF-A bsAb) plus chemotherapy in patients with SCLC or TNBC with brain metastases: A subgroup analysis of two phase 2 trials Abstract #1951RO Rapid Oral Presentation Investigational Immunotherapy Oct 24, 2026: 8:30 - 10:00am CEST First results from a phase 2 trial of second-line pumitamig (PD-L1 × VEGF-A bsAb) plus chemotherapy in unresectable neuroendocrine neoplasms Abstract #2393O Proffered Paper Presentation NETs and Endocrine Tumours Oct 26, 2026: 10:15 - 11:45am CET ROSETTA RCC-208: A phase 1/2, open-label, randomized study of pumitamig alone or in combination with ipilimumab or cabozantinib in advanced renal cell carcinoma (RCC) Abstract #3639TiP Poster Presentation Renal Cancer Oct 23, 2026: 3:15 - 4:00pm CEST ROSETTA Gastric-204: A randomized, phase 2/3 study of first-line (1L) pumitamig + chemotherapy versus nivolumab (NIVO) + chemotherapy in advanced or metastatic gastric cancer (GC), gastroesophageal junction adenocarcinoma (GEJC), or esophageal adenocarcinoma (EAC) Abstract #3081eTiP ePoster Presentation Oesophagogastric Cancer Oct 23 - 27, 2026 A phase 1/2 trial of first-line pumitamig monotherapy or pumitamig plus ipilimumab in advanced or unresectable hepatocellular carcinoma (ROSETTA HCC-206) Abstract #1762eTiP ePoster Presentation Hepatocellular Carcinoma Oct 23 - 27, 2026 Pumitamig + elfetabart drozuntecan Elfetabart drozuntecan (Elfe-D; DB-1311/BNT324 [B7H3 ADC]) + pumitamig (PD-L1 x VEGF-A bsAb) in patients with advanced solid tumors: First results from a global phase 2 trial Abstract #999O Proffered Paper Presentation Developmental Therapeutics Oct 24, 2026: 2:45 - 4:15pm CEST Pumitamig + trastuzumab pamirtecan Pumitamig + trastuzumab pamirtecan (T-Pam) in patients (pts) with HR+, HER2-low advanced/metastatic breast cancer (a/mBC): Phase 1 results of a phase 1/2, open-label trial (BNT323-03) Abstract #1777P Poster Presentation HER2+ Breast Cancer Oct 24, 2026: 12:00 - 12:45pm CEST Elfetabart drozuntecan Elfetabart drozuntecan (Elfe-D; DB-1311/BNT324 [B7H3 ADC]) in patients with platinum-resistant ovarian cancer (PROC): Efficacy and safety data from a Phase 2 dose optimization cohort Abstract #1255P Poster Presentation Gynaecological Cancers Oct 26, 2026: 12:00 - 12:45pm CET BNT324-03: A phase 3, randomized, open-label trial comparing elfetabart drozuntecan, a B7H3 antibody-drug conjugate (ADC), with docetaxel as treatment of patients (pts) with taxane-naïve metastatic castration-resistant prostate cancer (mCRPC) Abstract #2300TiP Poster Presentation Metastatic Prostate Cancer Oct 23, 2026: 3:15 - 4:00pm CEST Gotistobart Gotistobart plus pembrolizumab demonstrates durable antitumor activity in advanced melanoma refractory or resistant to both prior anti-CTLA-4 and anti-PD-(L)1 therapies Abstract #2106P Poster Presentation Melanoma Oct 24, 2026: 12:00 - 12:45 pm CEST BNT317 BNT317: An anti–PD-1 × CD39 bispecific antibody demonstrating preclinical antitumor activity Abstract #420P Poster Presentation Basic Science Oct 23, 2026: 3:15 - 4pm CEST Beruzatatug pelitecan (YL202/BNT326) Clinical outcomes of YL202/BNT326 (HER3 ADC) in advanced/metastatic HR+/HER2-null/low breast cancer (BC) in chemotherapy (chemo)-naïve and exposed patients (pts) Abstract #1881P Poster Presentation HR+ Breast Cancer Oct 24, 2026: 12:00 - 12:45pm CEST BNT329 A first-in-human phase 1/2 dose-escalation and expansion trial of BNT329 (an anti-carbohydrate antigen 19-9 antibody-drug conjugate [CA19-9 ADC]) in patients (pts) with advanced solid tumors that express CA19-9 Abstract #1076TiP Poster Presentation Developmental Therapeutics Oct 23, 2026: 3:15 - 4:00 pm CEST Health Economics and Outcomes Research (HEOR) Real-world progression-free survival (rwPFS) from chemotherapy initiation in patients with HR+/HER2– metastatic breast cancer (mBC) in the US Abstract #1880P Poster Presentation HR+ Breast Cancer Oct 24, 2026: 12:00 - 12:45pm CEST Real-world outcomes with first-line pembrolizumab-based chemoimmunotherapy in patients with advanced/metastatic non-small cell lung cancer in the USA Abstract #2705P Poster Presentation NSCLC, Metastatic Oct 26, 2026: 12:00 - 12:45pm CET About BioNTech BioNTech is a global next generation biopharmaceutical company pioneering novel investigative therapies for cancer and other serious diseases. In oncology, BioNTech is committed to transforming how cancer is treated. Its ambition is to develop innovative medicines with pan-tumor or synergistic potential to address cancer from multiple angles and across the full continuum of the disease from early- to late-stage. Its growing late-stage oncology pipeline comprises complementary treatment approaches spanning immunomodulators, antibody drug conjugates, and mRNA cancer immunotherapies. BioNTech has partnered with multiple global and specialized pharmaceutical collaborators leveraging complementary expertise and resources to accelerate innovation and drive progress, including Bristol Myers Squibb, Duality Biologics, Genentech, a member of the Roche Group, Genmab, MediLink, OncoC4, and Pfizer. For more information, please visit . BioNTech Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, but not limited to, statements concerning: the initiation, timing, progress and results of BioNTech’s research and development programs in oncology, including the targeted timing and number of additional potentially registrational trials; BioNTech’s and its collaborators’ current and future preclinical and clinical trials in oncology, including the investigational bispecific immunomodulator pumitamig (BNT327/BMS986545), the investigational anti-CTLA-4 antibody gotistobart (BNT316/ONC-392), the investigational B7H3-targeting ADC elfetabart drozuntecan (BNT324/DB-1311) alone and in combination with pumitamig, the investigational HER2-targeting ADC trastuzumab pamirtecan (BNT323/DB-1303) in combination with pumitamig, the investigational anti-PD-1×CD39 antibody BNT317, the investigational HER3-targeting ADC Beruzatatug pelitecan (Beru-Pel/YL202/BNT326), and the investigational CA19-9-targeting ADC BNT329; the nature and characterization of and timing for release of clinical data across BioNTech’s platforms, which is subject to peer review, regulatory review and market interpretation; the planned next steps in BioNTech’s pipeline programs, including, but not limited to, statements regarding timing or plans for initiation or enrollment of clinical trials, or submission for and receipt of product approvals and potential commercialization with respect to BioNTech’s product candidates; and the potential safety and efficacy of BioNTech’s product candidates. In some cases, forward-looking statements can be identified by terminology such as “will,” “may,” “should,” “expects,” “intends,” “plans,” “aims,” “anticipates,” “believes,” “estimates,” “predicts,” “potential,” “continue,” or the negative of these terms or other comparable terminology, although not all forward-looking statements contain these words. The forward-looking statements in this press release are based on BioNTech’s current expectations and beliefs of future events and are neither promises nor guarantees. You should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, many of which are beyond BioNTech’s control, and which could cause actual results to differ materially and adversely from those expressed or implied by these forward-looking statements. These risks and uncertainties include, but are not limited to: the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for clinical trials, projected data release timelines, regulatory submission dates, regulatory approval dates and/or launch dates, as well as risks associated with preclinical and clinical data, including the data discussed in this release, and including the possibility of unfavorable new preclinical, clinical or safety data and further analyses of existing preclinical, clinical or safety data; the nature of the clinical data, which is subject to ongoing peer review, regulatory review and market interpretation; the ability to produce comparable clinical results in future clinical trials; the timing of and BioNTech’s ability to obtain and maintain regulatory approval for its product candidates; discussions with regulatory agencies regarding timing and requirements for additional clinical trials; the impact of tariffs and escalations in trade policy; BioNTech’s ability to identify research opportunities and discover and develop investigational medicines; the ability and willingness of BioNTech’s third-party collaborators to continue research and development activities relating to BioNTech’s development candidates and investigational medicines; unforeseen safety issues and potential claims that are alleged to arise from the use of products and product candidates developed or manufactured by BioNTech; BioNTech’s and its collaborators’ ability to commercialize and market its product candidates, if approved; BioNTech’s ability to manage its development and related expenses; regulatory and political developments; BioNTech’s ability to effectively scale its production capabilities and manufacture its products and product candidates; risks relating to the global financial system and markets; and other factors not known to BioNTech at this time. You should review the risks and uncertainties described under the heading “Risk Factors” in BioNTech’s Report on Form 6-K for the period ended June 30, 2026 and in subsequent filings made by BioNTech with the SEC, which are available on the SEC’s website at . These forward-looking statements speak only as of the date hereof. Except as required by law, BioNTech disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. CONTACTS Media Relations Jasmina Alatovic Media@BioNTech.de Investor Relations Douglas Maffei, Ph.D. Investors@BioNTech.de

Clinical ResultPhase 2ImmunotherapyASCOADC

19 Oct 2026

·www.biospace.com

Bristol Myers Squibb to Showcase Next-Generation Cancer Modalities and Leadership Across Solid Tumors at ESMO 2026

Late-breaking Phase 3 disclosures for izalontamab brengitecan (iza-bren), a potential first-in-class EGFR × HER3 bispecific ADC, in EGFR-mutated non-small cell lung cancer (PANKU-Lung01) and HR+/HER2- breast cancer (PANKU-Breast01) Late-breaking Phase 2 global data presentation for pumitamig, a PD-L1 × VEGF-A bispecific immunomodulator, from ROSETTA Lung-107 in metastatic non-small cell lung cancer PRINCETON, N.J.--(BUSINESS WIRE)-- $BMY #ESMO -- Bristol Myers Squibb (NYSE: BMY) today announced it will present new data from its oncology portfolio and pipeline across a broad range of solid tumors at the European Society for Medical Oncology (ESMO) 2026 Congress to be held October 23-27 in Madrid, Spain. Across 12 oral presentations and nearly 60 disclosures, the company will highlight progress across differentiated therapeutic approaches, including: Late-breaking global Phase 2 data for pumitamig, an investigational PD-L1 x VEGF-A bispecific immunomodulator, being jointly developed with BioNTech Two late-breaking, Phase 3 studies of izalontamab brengitecan (iza-bren), a potential first-in-class EGFR x HER3 bispecific antibody drug conjugate (ADC), sponsored by SystImmune’s parent company, Sichuan Biokin Pharmaceutical Co., Ltd. (Biokin) Long-term survival results evaluating Opdivo ® in combination with Yervoy ® in a Phase 3 late-breaking oral presentation “BMS is advancing a new generation of oncology medicines designed to address some of the most persistent challenges faced by patients with cancer,” said Cristian Massacesi, MD , executive vice president, chief medical officer and head of Development, Bristol Myers Squibb. “At ESMO 2026, we will share important data across both emerging and established modalities. From new Phase 3 results for iza-bren and new findings for pumitamig, to long-term survival data for Opdivo ® plus Yervoy ® , these results reflect the breadth of our oncology pipeline, and our focus on translating differentiated science into meaningful, durable outcomes for patients.” Key data highlights across diverse modalities and scientific approaches to be presented at the ESMO 2026 Congress: Abstract Title Author Presentation Type/# Session Title Session Date/Time (Local Time (Madrid) Thoracic Cancer A Global Phase 2, Open-label Trial of Pumitamig (PD-L1 × VEGF-A bsAb) + Docetaxel Post-chemoimmunotherapy in mNSCLC: First Data from ROSETTA Lung-107 Cho Rapid Oral #LBA76 Rapid Oral 2: NSCLC, Metastatic Sunday, October 25, 8:30 – 10:00 AM PANKU-Lung01: Randomized Phase 3 Study of Izalontamab Brengitecan (iza-bren) in EGFR-Mutant Non-Squamous Non-Small Cell Lung Cancer (NSCLC) After Progression on a Third Generation EGFR TKI Zhang Proffered Paper #LBA71 Proffered Paper 2: NSCLC, Metastatic Monday, October 26, 8:30 – 10:00 AM Phase 2 Study of Izalontamab Brengitecan (iza-bren) Plus Osimertinib in Patients with EGFR Mutant Locally Advanced or Metastatic NSCLC Zhou Poster #2575P Poster Display Session Monday, October 26, 12:00 – 12:45 PM Breast/Thoracic Cancer Efficacy of Pumitamig (PD-L1 × VEGF-A bsAb) Plus Chemotherapy in Patients with SCLC and TNBC with Brain Metastases: Subgroup Analyses of Two Phase 2 Trials Pavlakis Rapid Oral #1951RO Rapid Oral: Investigational Immunotherapy Saturday, October 24, 8:30 – 10:00 AM Breast Cancer PANKU-Breast01: Randomized Phase 3 Study of Izalontamab Brengitecan (iza-bren) in Previously Treated Unresectable Locally Advanced or Metastatic (LA/M) Hormone Receptor-Positive/HER2- negative (HR+/HER2-) Breast Cancer (BC) Lan Proffered Paper #LBA50 Proffered Paper 1: Breast Cancer, Early and Advanced Stage Friday October 23, 1:30 – 3:00 PM Head and Neck Cancer Phase 2 Study of Izalontamab Brengitecan (iza-bren) Plus Pembrolizumab in Patients with Recurrent or Metastatic Head and Neck Squamous Cell Carcinoma Ji Rapid Oral #1542RO Rapid Oral: Head and Neck Cancer Sunday, October 25, 2:45 – 4:15 PM Gastrointestinal Cancer Prevalence, Prognosis, and Impact on Tumor Microenvironment of Homozygous MTAP Deletion in Advanced Solid Tumors Rodón Poster #4445P Poster Display Session Friday, October 23, 3:15 – 4:00 PM Final Overall Survival Results from CheckMate 8HW: Nivolumab plus Ipilimumab vs Nivolumab or Chemotherapy in MSI-H/dMMR Metastatic Colorectal Cancer Lonardi Rapid Oral #LBA32 Rapid Oral: Gastrointestinal Tumours, Lower Digestive Saturday, October 24, 2:45 – 4:15 PM Genitourinary Cancer Results From a Phase 3 Trial of Neoadjuvant Chemotherapy Alone vs in Combination with Nivolumab Followed by Postsurgical Adjuvant Nivolumab in Patients with Muscle-Invasive Bladder Cancer Necchi Proffered Paper #4736O Proffered Paper 1: Genitourinary Tumour, Renal and Urothelial Friday, October 23, 1:30 – 3:00 PM Pan Tumor Cancer First Results from a Phase 2 Trial of Second-Line Pumitamig (PD-L1 × VEGF-A bsAb) Plus Chemotherapy in Unresectable Neuroendocrine Neoplasms Xu Proffered Paper #2393O Proffered Paper: NETs and Endocrine Tumours Monday, October 26, 10:15 – 11:45 AM When Overall Survival Is Not Enough: The Emerging Need for Context-Sensitive Endpoint Frameworks in Oncology Cardone Proffered Paper #3315O Proffered Paper: Policy Sunday, October 25, 10:15 – 11:45 AM INDICATIONS OPDIVO ® (nivolumab), as a single agent, is indicated for the treatment of adult and pediatric patients 12 years and older with unresectable or metastatic melanoma. OPDIVO ® (nivolumab), in combination with YERVOY ® (ipilimumab), is indicated for the treatment of adult and pediatric patients 12 years and older with unresectable or metastatic melanoma. OPDIVO ® (nivolumab) is indicated for the adjuvant treatment of adult and pediatric patients 12 years and older with completely resected Stage IIB, Stage IIC, Stage III, or Stage IV melanoma. OPDIVO ® (nivolumab), in combination with platinum-doublet chemotherapy, is indicated as neoadjuvant treatment of adult patients with resectable (tumors ≥4 cm or node positive) non-small cell lung cancer (NSCLC). OPDIVO ® (nivolumab) in combination with platinum-doublet chemotherapy, is indicated for neoadjuvant treatment of adult patients with resectable (tumors ≥4 cm or node positive) non-small cell lung cancer (NSCLC) and no known epidermal growth factor receptor (EGFR) mutations or anaplastic lymphoma kinase (ALK) rearrangements, followed by single-agent OPDIVO ® as adjuvant treatment after surgery. OPDIVO ® (nivolumab), in combination with YERVOY ® (ipilimumab), is indicated for the first-line treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) whose tumors express PD-L1 (≥1%) as determined by an FDA-authorized test, with no EGFR or ALK genomic tumor aberrations. OPDIVO ® (nivolumab), in combination with YERVOY® (ipilimumab) and 2 cycles of platinum-doublet chemotherapy, is indicated for the first-line treatment of adult patients with metastatic or recurrent non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations. OPDIVO ® (nivolumab) is indicated for the treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) with progression on or after platinum-based chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving OPDIVO. OPDIVO ® (nivolumab), in combination with YERVOY ® (ipilimumab), is indicated for the first-line treatment of adult patients with unresectable malignant pleural mesothelioma (MPM). OPDIVO ® (nivolumab), in combination with YERVOY ® (ipilimumab), is indicated for the first-line treatment of adult patients with intermediate or poor risk advanced renal cell carcinoma (RCC). OPDIVO ® (nivolumab), in combination with cabozantinib, is indicated for the first-line treatment of adult patients with advanced renal cell carcinoma (RCC). OPDIVO ® (nivolumab) is indicated for the treatment of adult patients with advanced renal cell carcinoma (RCC) who have received prior anti-angiogenic therapy. OPDIVO ® (nivolumab), as a single agent, is indicated for the treatment of adult patients with classical Hodgkin lymphoma (cHL) that has relapsed or progressed after autologous hematopoietic stem cell transplantation (HSCT) and brentuximab vedotin or after 3 or more lines of systemic therapy that includes autologous HSCT. OPDIVO ® (nivolumab), in combination with doxorubicin, vinblastine, and dacarbazine (AVD), is indicated for the treatment of adult and pediatric patients 12 years and older with previously untreated, Stage III or IV classical Hodgkin lymphoma (cHL). OPDIVO ® (nivolumab) is indicated for the treatment of adult patients with recurrent or metastatic squamous cell carcinoma of the head and neck (SCCHN) with disease progression on or after platinum-based therapy. OPDIVO ® (nivolumab), as a single agent, is indicated for the adjuvant treatment of adult patients with urothelial carcinoma (UC) who are at high risk of recurrence after undergoing radical resection of UC. OPDIVO ® (nivolumab), in combination with cisplatin and gemcitabine, is indicated for the first-line treatment for adult patients with unresectable or metastatic urothelial carcinoma. OPDIVO ® (nivolumab) is indicated for the treatment of adult patients with locally advanced or metastatic urothelial carcinoma who have disease progression during or following platinum-containing chemotherapy or have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy. OPDIVO ® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of adult and pediatric patients 12 years and older with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) unresectable or metastatic colorectal cancer (CRC) as determined by an FDA-authorized test. OPDIVO ® (nivolumab), as a single agent, is indicated for the treatment of adult and pediatric (12 years and older) patients with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. OPDIVO ® (nivolumab), in combination with YERVOY ® (ipilimumab), is indicated for the first-line treatment of adult patients with unresectable or metastatic hepatocellular carcinoma (HCC). OPDIVO ® (nivolumab), in combination with YERVOY ® (ipilimumab), is indicated for the treatment of adult patients with unresectable or metastatic hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. OPDIVO ® (nivolumab) is indicated for the adjuvant treatment of completely resected esophageal or gastroesophageal junction cancer with residual pathologic disease in adult patients who have received neoadjuvant chemoradiotherapy (CRT). OPDIVO ® (nivolumab), in combination with fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the first-line treatment of adult patients with unresectable advanced or metastatic esophageal squamous cell carcinoma (ESCC) whose tumors express PD-L1 (≥1). OPDIVO ® (nivolumab), in combination with YERVOY ® (ipilimumab), is indicated for the first-line treatment of adult patients with unresectable advanced or metastatic esophageal squamous cell carcinoma (ESCC) whose tumors express PD-L1 (≥1). OPDIVO ® (nivolumab) is indicated for the treatment of adult patients with unresectable advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC) after prior fluoropyrimidine- and platinum-based chemotherapy. OPDIVO ® (nivolumab), in combination with fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the treatment of adult patients with advanced or metastatic gastric cancer, gastroesophageal junction cancer, and esophageal adenocarcinoma whose tumors express PD-L1 (≥1). IMPORTANT SAFETY INFORMATION Severe and Fatal Immune-Mediated Adverse Reactions Immune-mediated adverse reactions listed herein may not include all possible severe and fatal immune-mediated adverse reactions. Immune-mediated adverse reactions, which may be severe or fatal, can occur in any organ system or tissue. While immune-mediated adverse reactions usually manifest during treatment, they can also occur after discontinuation of OPDIVO or YERVOY. Early identification and management are essential to ensure safe use of OPDIVO and YERVOY. Monitor for signs and symptoms that may be clinical manifestations of underlying immune-mediated adverse reactions. Evaluate clinical chemistries including liver enzymes, creatinine, adrenocorticotropic hormone (ACTH) level, and thyroid function at baseline and periodically during treatment with OPDIVO and before each dose of YERVOY. In cases of suspected immune-mediated adverse reactions, initiate appropriate workup to exclude alternative etiologies, including infection. Institute medical management promptly, including specialty consultation as appropriate. Withhold or permanently discontinue OPDIVO and YERVOY depending on severity (please see section 2 Dosage and Administration in the accompanying Full Prescribing Information). In general, if OPDIVO or YERVOY interruption or discontinuation is required, administer systemic corticosteroid therapy (1 to 2 mg/kg/day prednisone or equivalent) until improvement to Grade 1 or less. Upon improvement to Grade 1 or less, initiate corticosteroid taper and continue to taper over at least 1 month. Consider administration of other systemic immunosuppressants in patients whose immune-mediated adverse reactions are not controlled with corticosteroid therapy. Toxicity management guidelines for adverse reactions that do not necessarily require systemic steroids (e.g., endocrinopathies and dermatologic reactions) are discussed below. Immune-Mediated Pneumonitis OPDIVO and YERVOY can cause immune-mediated pneumonitis. The incidence of pneumonitis is higher in patients who have received prior thoracic radiation. In patients receiving OPDIVO monotherapy, immune-mediated pneumonitis occurred in 3.1% (61/1994) of patients, including Grade 4 (<0.1%), Grade 3 (0.9%), and Grade 2 (2.1%). In patients receiving OPDIVO 1 mg/kg with YERVOY 3 mg/kg every 3 weeks, immune-mediated pneumonitis occurred in 7% (31/456) of patients, including Grade 4 (0.2%), Grade 3 (2.0%), and Grade 2 (4.4%). In patients receiving OPDIVO 3 mg/kg with YERVOY 1 mg/kg every 3 weeks, immune-mediated pneumonitis occurred in 3.9% (26/666) of patients, including Grade 3 (1.4%) and Grade 2 (2.6%). In NSCLC patients receiving OPDIVO 3 mg/kg every 2 weeks with YERVOY 1 mg/kg every 6 weeks, immune-mediated pneumonitis occurred in 9% (50/576) of patients, including Grade 4 (0.5%), Grade 3 (3.5%), and Grade 2 (4.0%). Four patients (0.7%) died due to pneumonitis. Immune-Mediated Colitis OPDIVO and YERVOY can cause immune-mediated colitis, which may be fatal. A common symptom included in the definition of colitis was diarrhea. Cytomegalovirus (CMV) infection/reactivation has been reported in patients with corticosteroid-refractory immune-mediated colitis. In cases of corticosteroid-refractory colitis, consider repeating infectious workup to exclude alternative etiologies. In patients receiving OPDIVO monotherapy, immune-mediated colitis occurred in 2.9% (58/1994) of patients, including Grade 3 (1.7%) and Grade 2 (1%). In patients receiving OPDIVO 1 mg/kg with YERVOY 3 mg/kg every 3 weeks, immune-mediated colitis occurred in 25% (115/456) of patients, including Grade 4 (0.4%), Grade 3 (14%) and Grade 2 (8%). In patients receiving OPDIVO 3 mg/kg with YERVOY 1 mg/kg every 3 weeks, immune-mediated colitis occurred in 9% (60/666) of patients, including Grade 3 (4.4%) and Grade 2 (3.7%). Immune-Mediated Hepatitis and Hepatotoxicity OPDIVO and YERVOY can cause immune-mediated hepatitis. In patients receiving OPDIVO monotherapy, immune-mediated hepatitis occurred in 1.8% (35/1994) of patients, including Grade 4 (0.2%), Grade 3 (1.3%), and Grade 2 (0.4%). In patients receiving OPDIVO 1 mg/kg with YERVOY 3 mg/kg every 3 weeks, immune-mediated hepatitis occurred in 15% (70/456) of patients, including Grade 4 (2.4%), Grade 3 (11%), and Grade 2 (1.8%). In patients receiving OPDIVO 3 mg/kg with YERVOY 1 mg/kg every 3 weeks, immune-mediated hepatitis occurred in 7% (48/666) of patients, including Grade 4 (1.2%), Grade 3 (4.9%), and Grade 2 (0.4%). OPDIVO in combination with cabozantinib can cause hepatic toxicity with higher frequencies of Grade 3 and 4 ALT and AST elevations compared to OPDIVO alone. Consider more frequent monitoring of liver enzymes as compared to when the drugs are administered as single agents. In patients receiving OPDIVO and cabozantinib, Grades 3 and 4 increased ALT or AST were seen in 11% (35/320) of patients. Immune-Mediated Endocrinopathies OPDIVO and YERVOY can cause primary or secondary adrenal insufficiency, immune-mediated hypophysitis, immune-mediated thyroid disorders, and Type 1 diabetes mellitus, which can present with diabetic ketoacidosis. Withhold OPDIVO and YERVOY depending on severity (please see section 2 Dosage and Administration in the accompanying Full Prescribing Information). For Grade 2 or higher adrenal insufficiency, initiate symptomatic treatment, including hormone replacement as clinically indicated. Hypophysitis can present with acute symptoms associated with mass effect such as headache, photophobia, or visual field defects. Hypophysitis can cause hypopituitarism; initiate hormone replacement as clinically indicated. Thyroiditis can present with or without endocrinopathy. Hypothyroidism can follow hyperthyroidism; initiate hormone replacement or medical management as clinically indicated. Monitor patients for hyperglycemia or other signs and symptoms of diabetes; initiate treatment with insulin as clinically indicated. In patients receiving OPDIVO monotherapy, adrenal insufficiency occurred in 1% (20/1994), including Grade 3 (0.4%) and Grade 2 (0.6%). In patients receiving OPDIVO 1 mg/kg with YERVOY 3 mg/kg every 3 weeks, adrenal insufficiency occurred in 8% (35/456) of patients, including Grade 4 (0.2%), Grade 3 (2.4%), and Grade 2 (4.2%). In patients receiving OPDIVO 3 mg/kg with YERVOY 1 mg/kg every 3 weeks, adrenal insufficiency occurred in 7% (48/666) of patients, including Grade 4 (0.3%), Grade 3 (2.5%), and Grade 2 (4.1%). In patients receiving OPDIVO and cabozantinib, adrenal insufficiency occurred in 4.7% (15/320) of patients, including Grade 3 (2.2%) and Grade 2 (1.9%). In patients receiving OPDIVO monotherapy, hypophysitis occurred in 0.6% (12/1994) of patients, including Grade 3 (0.2%) and Grade 2 (0.3%). In patients receiving OPDIVO 1 mg/kg with YERVOY 3 mg/kg every 3 weeks, hypophysitis occurred in 9% (42/456) of patients, including Grade 3 (2.4%) and Grade 2 (6%). In patients receiving OPDIVO 3 mg/kg with YERVOY 1 mg/kg every 3 weeks, hypophysitis occurred in 4.4% (29/666) of patients, including Grade 4 (0.3%), Grade 3 (2.4%), and Grade 2 (0.9%). In patients receiving OPDIVO monotherapy, thyroiditis occurred in 0.6% (12/1994) of patients, including Grade 2 (0.2%). In patients receiving OPDIVO 3 mg/kg with YERVOY 1 mg/kg every 3 weeks, thyroiditis occurred in 2.7% (22/666) of patients, including Grade 3 (4.5%) and Grade 2 (2.2%). In patients receiving OPDIVO monotherapy, hyperthyroidism occurred in 2.7% (54/1994) of patients, including Grade 3 (<0.1%) and Grade 2 (1.2%). In patients receiving OPDIVO 1 mg/kg with YERVOY 3 mg/kg every 3 weeks, hyperthyroidism occurred in 9% (42/456) of patients, including Grade 3 (0.9%) and Grade 2 (4.2%). In patients receiving OPDIVO 3 mg/kg with YERVOY 1 mg/kg every 3 weeks, hyperthyroidism occurred in 12% (80/666) of patients, including Grade 3 (0.6%) and Grade 2 (4.5%). In patients receiving OPDIVO monotherapy, hypothyroidism occurred in 8% (163/1994) of patients, including Grade 3 (0.2%) and Grade 2 (4.8%). In patients receiving OPDIVO 1 mg/kg with YERVOY 3 mg/kg every 3 weeks, hypothyroidism occurred in 20% (91/456) of patients, including Grade 3 (0.4%) and Grade 2 (11%). In patients receiving OPDIVO 3 mg/kg with YERVOY 1 mg/kg every 3 weeks, hypothyroidism occurred in 18% (122/666) of patients, including Grade 3 (0.6%) and Grade 2 (11%). In patients receiving OPDIVO monotherapy, diabetes occurred in 0.9% (17/1994) of patients, including Grade 3 (0.4%) and Grade 2 (0.3%), and 2 cases of diabetic ketoacidosis. In patients receiving OPDIVO 3 mg/kg with YERVOY 1 mg/kg every 3 weeks, diabetes occurred in 2.7% (15/666) of patients, including Grade 4 (0.6%), Grade 3 (0.3%), and Grade 2 (0.9%). Immune-Mediated Nephritis with Renal Dysfunction OPDIVO and YERVOY can cause immune-mediated nephritis. In patients receiving OPDIVO monotherapy, immune-mediated nephritis and renal dysfunction occurred in 1.2% (23/1994) of patients, including Grade 4 (<0.1%), Grade 3 (0.5%), and Grade 2 (0.6%). In patients receiving OPDIVO 3 mg/kg with YERVOY 1 mg/kg every 3 weeks, immune-mediated nephritis with renal dysfunction occurred in 4.1% (27/666) of patients, including Grade 4 (0.6%), Grade 3 (1.1%), and Grade 2 (2.2%). Immune-Mediated Dermatologic Adverse Reactions OPDIVO and YERVOY can cause immune-mediated rash or dermatitis. Exfoliative dermatitis, including Stevens-Johnson syndrome (SJS), toxic epidermal necrolysis (TEN), and drug rash with eosinophilia and systemic symptoms (DRESS) has occurred with PD-1/PD-L1 blocking antibodies. Topical emollients and/or topical corticosteroids may be adequate to treat mild to moderate non-exfoliative rashes. Withhold or permanently discontinue OPDIVO and YERVOY depending on severity (please see section 2 Dosage and Administration in the accompanying Full Prescribing Information). In patients receiving OPDIVO monotherapy, immune-mediated rash occurred in 9% (171/1994) of patients, including Grade 3 (1.1%) and Grade 2 (2.2%). In patients receiving OPDIVO 1 mg/kg with YERVOY 3 mg/kg every 3 weeks, immune-mediated rash occurred in 28% (127/456) of patients, including Grade 3 (4.8%) and Grade 2 (10%). In patients receiving OPDIVO 3 mg/kg with YERVOY 1 mg/kg every 3 weeks, immune-mediated rash occurred in 16% (108/666) of patients, including Grade 3 (3.5%) and Grade 2 (4.2%). Other Immune-Mediated Adverse Reactions The following clinically significant immune-mediated adverse reactions occurred at an incidence of <1% (unless otherwise noted) in patients who received OPDIVO monotherapy or OPDIVO in combination with YERVOY, or were reported with the use of other PD-1/PD-L1 blocking antibodies. Severe or fatal cases have been reported for some of these adverse reactions: cardiac/vascular: myocarditis, pericarditis, vasculitis; nervous system: meningitis, encephalitis, myelitis and demyelination, myasthenic syndrome/myasthenia gravis (including exacerbation), Guillain-Barré syndrome, nerve paresis, autoimmune neuropathy; ocular: uveitis, iritis, and other ocular inflammatory toxicities can occur; gastrointestinal: pancreatitis to include increases in serum amylase and lipase levels, gastritis, duodenitis; musculoskeletal and connective tissue: myositis/polymyositis, rhabdomyolysis, and associated sequelae including renal failure, arthritis, polymyalgia rheumatica; endocrine: hypoparathyroidism; hematologic/immune: hemolytic anemia, aplastic anemia, hemophagocytic lymphohistiocytosis (HLH), systemic inflammatory response syndrome, histiocytic necrotizing lymphadenitis (Kikuchi lymphadenitis), sarcoidosis, immune thrombocytopenic purpura, solid organ transplant rejection, other transplant (including corneal graft) rejection; other: Myocarditis-Myositis-Myasthenia Gravis (or Myasthenia-Like) Overlap Syndrome, reporting as the co-occurrence of either two or all three adverse reactions. Contacts Bristol Myers Squibb Media Inquiries: media@bms.com Investors: investor.relations@bms.com Read full story here

Phase 2Phase 3Clinical ResultDrug ApprovalAccelerated Approval

100 Deals associated with Irinotecan Hydrochloride

External Link

KEGG	Wiki	ATC	Drug Bank
D01061	Irinotecan Hydrochloride	L01CE02	DBSALT000103

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Childhood Malignant Solid Neoplasm	Japan	Yakult Honsha Co., Ltd.	25 Mar 2013
Childhood Malignant Solid Neoplasm	Japan	Daiichi Sankyo Co., Ltd.	25 Mar 2013
Intestinal Neoplasms	China	Jiangsu Hengrui Pharmaceuticals Co., Ltd.	01 Jan 1998
Colonic Cancer	United States	Pfizer Inc.	14 Jun 1996
Rectal Cancer	United States	Pfizer Inc.	14 Jun 1996
Breast Cancer	Japan	Yakult Honsha Co., Ltd.	29 Sep 1995
Breast Cancer	Japan	Alfresa Pharma Corp.	29 Sep 1995
Breast Cancer	Japan	Daiichi Sankyo Co., Ltd.	29 Sep 1995
Colorectal Cancer	Japan	Yakult Honsha Co., Ltd.	29 Sep 1995
Colorectal Cancer	Japan	Daiichi Sankyo Co., Ltd.	29 Sep 1995
Colorectal Cancer	Japan	Alfresa Pharma Corp.	29 Sep 1995
Lymphoma	Japan	Alfresa Pharma Corp.	29 Sep 1995
Non-Hodgkin Lymphoma	Japan	Alfresa Pharma Corp.	29 Sep 1995
Non-Hodgkin Lymphoma	Japan	Yakult Honsha Co., Ltd.	29 Sep 1995
Non-Hodgkin Lymphoma	Japan	Daiichi Sankyo Co., Ltd.	29 Sep 1995
Squamous Cell Carcinoma	Japan	Alfresa Pharma Corp.	29 Sep 1995
Squamous Cell Carcinoma	Japan	Daiichi Sankyo Co., Ltd.	29 Sep 1995
Squamous Cell Carcinoma	Japan	Yakult Honsha Co., Ltd.	29 Sep 1995
Stomach Cancer	Japan	Yakult Honsha Co., Ltd.	29 Sep 1995
Stomach Cancer	Japan	Alfresa Pharma Corp.	29 Sep 1995

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Advanced gastric carcinoma	Phase 3	Japan	Eli Lilly Japan KK	22 Feb 2017
Gastroesophageal junction adenocarcinoma	Phase 3	Japan	Eli Lilly Japan KK	22 Feb 2017
Hypersensitivity	Phase 3	Japan	Eli Lilly Japan KK	22 Feb 2017
RAS Wild Type Colorectal Cancer	Phase 3	France	Hoffmann-La Roche, Inc.	30 Oct 2013
RAS Wild Type Colorectal Cancer	Phase 3	Ireland	Hoffmann-La Roche, Inc.	30 Oct 2013
RAS Wild Type Colorectal Cancer	Phase 3	Israel	Hoffmann-La Roche, Inc.	30 Oct 2013
Esophageal Carcinoma	Phase 3	Canada	Pfizer Inc.	01 Apr 2009
recurrent gastric cancer	Phase 3	Japan	Yakult Honsha Co., Ltd.	01 Aug 2007
Colorectal Liver Metastases	Phase 3	France	Pfizer Inc.	01 Apr 2004
Colorectal Liver Metastases	Phase 3	France	Sanofi	01 Apr 2004

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


EANO2026	Not Applicable	Glioblastoma	152	Bevacizumab-Irinotecan	jdcqvercxq(bwjyofvnrv) = latdeyecgg zczmscguns (pwvzyfnglj ) View more	Positive	24 Sep 2026
				Bevacizumab-Lomustine	jdcqvercxq(bwjyofvnrv) = njoyyojldd zczmscguns (pwvzyfnglj ) View more
ESMO_GI2026	Not Applicable	Adenocarcinoma of small intestine Second line	34	Irinotecan	wubzttasjm(gtsaemlwnb) = diarrhoea (23.5%), neutropenia (14.7%), and febrile neutropenia (14.7%). cymtdxjowd (bhsfkwffxg ) View more	Positive	02 Jul 2026
NCT07381764 (ROMANCE, ESMO_GI2026)	Phase 2	Metastatic Colorectal Carcinoma Third line RAS/BRAF wild-type \| pMMR	150	Irinotecan plus cetuximab	drmpjtjjsn(toemexekto) = wgrrsmtlkm dvnqemejcb (nbxdurkijj ) View more	Positive	02 Jul 2026
				trifluridine/tipiracil plus bevacizumab	drmpjtjjsn(toemexekto) = uvebbqqksq dvnqemejcb (nbxdurkijj )
ESMO_GI2026 \| ESMO_GCC2026	Not Applicable	Metastatic Pancreatic Cancer First line BRCA1 \| BRCA2	26	Gemcitabine plus nab-paclitaxel (GnP)	lmvmzqvote(foxihioomu) = amymnreuef xgvvzdcawg (iafobrayab ) View more	Positive	02 Jul 2026
				modified FOLFIRINOX (mFFX)	lmvmzqvote(foxihioomu) = alcijvgcfr xgvvzdcawg (iafobrayab ) View more
ESMO_GI2026	Not Applicable	Colorectal Liver Metastases	30	TACE using a combination of oxaliplatin and irinotecan	sjqqjfbumw(vqsttxsmbm) = Most adverse events were mild and consistent with post-embolization syndrome ykxdmitvhp (swbsbcrplz )	Positive	02 Jul 2026
ESMO_GI2026	Not Applicable	Metastatic Colorectal Carcinoma	16	oral capecitabine + irinotecan (CapIRI)	kuqwwbsjef(ucbboywggr): HR = 1.03 (95.0% CI, 0.81 - 1.3) View more	Similar	02 Jul 2026
				5-fluorouracil + irinotecan (FOLFIRI)
NCT04251715 (HELIX-1, CTgov)	Phase 2	Unresectable Intrahepatic Cholangiocarcinoma	5	Quality-of-Life Assessment+leucovorin+Dexamethasone+Floxuridine+Irinotecan+oxaliplatin	cfnnsuopzd = ohygwrzglz dogoxkcywu (iufyyazkck, ftdgwyhkvw - dzxjasasni) View more	-	25 Jun 2026
NCT04067115 (SARC037, Pubmed \| Nat Med)	Phase 1/2	Ewing Sarcoma	37	Trabectedin 1.0 mg m-2 + irinotecan 25 mg m-2	tugybuxmoo(lbtqcdctzl) = rbvimqbymr tdbiwcyvfi (qqfvalzegw ) View more	Positive	16 Apr 2026
NCT02143219 (PAMELA70, CTgov)	Phase 2	Pancreatic adenocarcinoma metastatic \| Toxicity \| Metastatic Pancreatic Cancer	72	Folinic acid+Irinotecan+Oxaliplatine+5-FU	oxpsmhscko = xrqyuqbazu jyuitqfpyc (seboxxihmp, rctcxcosku - khitrxsstk) View more	-	15 Apr 2026
NCT05440786 (CTgov)	Phase 2	Neoplasm Metastasis \| Recurrent Ewing Sarcoma	46	Temozolomide+abemaciclib+Irinotecan (Abemaciclib + Irinotecan +Temozolomide)	ntznqxceil(vxmymgtwnw) = fmxdsqceqn iziwcafkdr (ejhhksnhwa, jtheguekff - gfqsuzpqvd) View more	-	12 Mar 2026
				Temozolomide+Irinotecan (Irinotecan +Temozolomide)	ntznqxceil(vxmymgtwnw) = heazaqycha iziwcafkdr (ejhhksnhwa, ozupffurfu - srlbxmfsnu) View more

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