A Multicenter, III Stage, Randomized Controlled Trail on the Safety and Efficacy of the Combination of Tislelizumab With Cisplatin and Gemcitabine, With or Without Trilaciclib in Untreated Unresectable and Metastatic Urothelial Carcinoma.

The aim of this study is to see whether the Trilaciclib is safe and effective in slowing down the growth of bladder cancer in patients while taking chemoimmunotherapy.

Start Date01 Oct 2025

Sponsor / Collaborator

Sun Yat-Sen Memorial Hospital

NCT05700448

/ Not yet recruitingPhase 3

A Phase III, Randomized, Double-Blind, Multicenter Study of Sugemalimab (CS1001) Plus PGemOx Regimen Versus Placebo Plus PGemOx for Subjects With Relapsed or Refractory Extranodal NK/T-Cell Lymphoma (R/R ENKTL)

The purpose of this study is to evaluate the efficacy and safety of sugemalimab (CS1001) in combination with PGemOx regimen (pegaspargase, gemcitabine, oxaliplatin) in treatment of adult patients with Extranodal NK/T-Cell Lymphoma (ENKTL) who have relapsed or become refractory to asparaginase-based regimens.

Start Date01 Jun 2025

Sponsor / Collaborator

Cornerstone Pharmaceutical Co., Ltd

NCT06274879

/ Not yet recruitingPhase 2

Safety of Biliary Intraductal Radiofrequency Ablation in Patients With Unresectable Extrahepatic Biliary Tract Cancer Undergoing Standard of Care Chemo-immune Checkpoint Inhibitor -Therapy: a Phase II, Multicenter, Randomized and Controlled

The goal of this clinical trial is to provide evidence for the general tolerability of radiofrequency ablation (bRFA) in patients with unresectable bile duct cancer undergoing systemic palliative treatment consisting of chemotherapy (gemcitabine and cisplatin) plus durvalumab (immune-checkpoint-inhibitor, ICI). The main question it aims to answer is whether it is safe to combine chemotherapy (gemcitabine and cisplatin) and immunotherapy (durvalumab) - CICI therapy.
Participants will be assigned to either the control group or the experimental group. In the control group, the standard of care consists of endoscopy with stent placement in the bile duct and CICI, whereas in the experimental group, bRFA will be performed in addition to the standard of care. Participants will be followed up for 6 months, during the follow-up, the stage of the tumor, blood examination, the duration of the stent from the insertion until its failure, adverse events and quality of life will be examined.
Researchers will compare the standard of care alone to the experimental group to see if the additional bRFA procedure causes higher or no difference in adverse events rate.

Start Date01 Jun 2025

Sponsor / Collaborator

Insel Gruppe AG

[+1]

100 Clinical Results associated with Gemcitabine

100 Translational Medicine associated with Gemcitabine

100 Patents (Medical) associated with Gemcitabine

29,380

Literatures (Medical) associated with Gemcitabine

01 Dec 2025·Journal of Gastrointestinal Cancer

Impact of Retroperitoneal Lymphadenopathy (RPLN) on the Outcomes of Locally Advanced Gall Bladder Cancer (GBC) Following Chemotherapy (CT) or Chemotherapy Followed by Consolidation Chemoradiotherapy (CTRT)

Article

Author: Naik, Nagendra ; Priyanka, Parul ; Agrawal, Sushma

INTRODUCTION:

Retroperitoneal lymphadenopathy is considered a metastatic disease in GBC; however, some surgical series of radical surgery with enlarged RPLN who underwent RPLN dissection have shown results marginally inferior to those without enlarged RPLN. Radiological RPLN comprises a major proportion of advanced non-metastatic GBC. There is dilemma in the intent of treatment to be offered in such cases. We are reporting our series of outcome of GBC with RPLN treated with first-line CT followed by consolidation CTRT.

MATERIALS AND METHODS:

Non-metastatic locally advanced GBC with good performance status (KPS ≥ 80) were initiated on first-line CT (cisplatin-gemcitabine), and thereafter, responders were evaluated by CECT-angiography and PET-CT scan for resectability. If found unresectable, they were offered consolidation CTRT to a dose of 45 Gy by conventional fractionation (3D-CRT technique) along with concurrent capecitabine at 1250 mg/m² to GBC and regional lymphatics including RPLN. Thereafter, boost dose of 9 Gy/5# was given to GBC only. Response assessment was done using CECT abdomen by RECIST criteria v 1.1. Outcomes (overall survival) of the two groups (RPLN vs non-RPLN) were computed with Kaplan-Meier survival curves and chi-square tests using SPSS v 20.

RESULTS:

Among 189 patients of advanced non-metastatic GBC recruited from 2011 to 2022, 80 had RPLN. The demographic features of both groups were comparable. Overall, 68% of the patients were women, 30% underwent upfront stenting for obstructive jaundice, and 90% had T3 and T4 disease. Only 10% had undergone upfront laparoscopic staging and had pathologically proven RPLN. Forty percent of the patients received four cycles of CT only and 50% of the patients received six cycles or more and 33% received CTRT. By RECIST criteria, 10% vs 16% achieved complete response (CR), 39% vs 41% achieved partial response (PR), 16% vs 15% achieved stable disease (SD), 2.7% vs 6% had disease progression (PD), and 14.5% vs 3.7% were non-evaluable in non-RPLN group vs RPLN group, respectively. 12% vs 6% could undergo radical surgery in non-RPLN group vs RPLN group (p = 0.03). The median OS was 9 months (95% CI 7.6-10.3 months) vs 10 months (95% CI 8-9.8 months) (p = NS) in non-RPLN group vs RPLN group, respectively. In those who received CT only, the median OS was 7 months vs 8 months, while in those who received CT followed by CTRT, the median OS was 14 months vs 13 months (p = 0.65) in non-RPLN group vs RPLN group, respectively.

CONCLUSIONS:

Based on this analysis, we conclude that RPLN constitutes a major proportion of advanced non-metastatic GBC and has outcomes similar to those without RPLN if treated with radical intent. RPLN should not be considered a metastatic disease and should be treated with radical intent.

01 Dec 2025·CANCER CHEMOTHERAPY AND PHARMACOLOGY

Population pharmacokinetics and exposure-response analysis of durvalumab in combination with gemcitabine and cisplatin in patients with advanced biliary tract cancer

Article

Author: Chen, Cecil ; Zhou, Diansong ; Fan, Chunling ; Ren, Song ; Phipps, Alex ; Zotkiewicz, Magdalena ; Lim, KyoungSoo ; Wang, Julie ; Kim, Chong ; Xynos, Ioannis ; Abegesah, Aburough ; Oh, Do-Youn ; Gibbs, Megan

PURPOSE:

Durvalumab in combination with gemcitabine/cisplatin has shown a favorable benefit-risk profile in the TOPAZ-1 study for advanced biliary tract cancers (BTC). This analysis evaluated the population pharmacokinetics (PopPK) of durvalumab, and exposure-response for efficacy and safety (ERES) of TOPAZ-1.

METHODS:

The PopPK model for durvalumab was updated using data from 5 previously analysed studies and TOPAZ-1. Individual exposure metrics were derived from the individual empirical Bayes estimates as drivers for exposure-response (ER) analysis related to efficacy and safety.

RESULTS:

Consistent with previous analyses, the durvalumab pharmacokinetics in BTC followed a 2-compartment model with time-dependent clearance. The final population parameters were: CL, 0.298 L/day; V1, 3.42 L; V2, 1.99 L; Q, 0.452 L/day; and the time dependent clearance suggests that the clearance could decrease up to 39% over the time course of treatment. There were 111 patients (3.53%) with treatment-emergent ADA positive in the pooled group of 6 studies, and the exposure was comparable for ADA positive and negative patients. Covariates had minimal clinical impact on PopPK parameters. No significant associations were found between exposure and overall survival (OS), progression-free survival (PFS), using Cox proportional analysis (CPH). Logistic regression analysis indicated no significant relationship between the exposure and relevant adverse events measures of Grade 3 and higher treatment-related AE, Grade 3 and higher treatment-related AESI (AEs of special interest), or AE leading to treatment discontinuation.

CONCLUSIONS:

No dose adjustment for durvalumab is needed based on PopPK and ERES analyses. The analysis supports the TOPAZ-1 regimen for patients with advanced BTC.

01 May 2025·JOURNAL OF COLLOID AND INTERFACE SCIENCE

Mn-doped MOF nanoparticles mitigating hypoxia via in-situ substitution strategy for dual-imaging guided combination treatment of microwave dynamic therapy and chemotherapy

Article

Author: Jiang, Guihua ; Wang, Haiying ; Qu, Hong ; Hang, Lifeng ; Meng, Xianwei ; Li, Hong ; Diao, Yanzhao ; Fang, Laiping ; Sun, Hui ; Liu, Jinwu ; Liu, Wangzi ; Wang, Jizhuang

Microwave dynamic therapy (MWDT) destroy tumor cells using reactive oxygen species (ROS), but its effectiveness is limited by low ROS production and intracellular oxygen (O₂) availability. This study presents a novel strategy using manganese (II) ion (Mn²⁺) doped iron (Fe)-based metal-organic framework (Fe MOF) nanoparticles (NPs) to enhance both O₂ generation and ROS production for improved MWDT. Incorporating Mn²⁺ into Fe MOF narrows the bandgap from 0.673 eV to 0.429 eV, improving the separation of electronic-hole pair and increasing ROS yield. Meanwhile, Mn-porphyrin nanocomplexes facilitate the decomposition of hydrogen peroxide to O₂in situ. Additionally, encapsulating the chemotherapeutic drug gemcitabine (GEM) within NPs and surface-modifying with Pluronic F127 creates Mn-Fe MOF@GEM@F127 (MMGF) NPs, which are suitable for photoacoustic/magnetic resonance imaging guidance (relaxivity, r₁: 2.007 mM^-1s^-1). The microwave (MW)/pH dual responsive GEM release works synergistically with MWDT, thereby more effectively disrupting tumor cells. This strategy differs from monotherapy by using MW sensitizers to enhance O₂ production, which not only increases the efficiency of ROS generation in MWDT but also makes subsequent chemotherapy more effective while reducing the side effects of conventional chemotherapy. This combined treatment reduced HONE-1 cell proliferation and tumor growth by 89.95 % and 96.12 %, respectively. The study proposes a versatile strategy to significantly improve both MWDT and chemotherapy efficacy with potential applications to various cancers.

759

News (Medical) associated with Gemcitabine

19 Mar 2025

·ir.renovorx.com

RenovoRx Announces Abstract Presentation at the Society of Surgical Oncology (SSO) 2025

New human pharmacokinetic (PK) and other pre-clinical data supports use of RenovoRx’s novel Trans-Arterial Micro-Perfusion (TAMP™) therapy platform MOUNTAIN VIEW, Calif.--(BUSINESS WIRE)-- RenovoRx, Inc. (“RenovoRx” or the “Company”) (Nasdaq: RNXT), a life sciences company developing innovative targeted oncology therapies and commercializing RenovoCath®, a novel, FDA-cleared drug-delivery device, is proud to announce a presentation of a new pre-clinical clinical data abstract at the upcoming Society of Surgical Oncology (SSO) 2025 Annual Meeting. The abstract, titled “Pharmacodynamics of Intra-arterial vs. Intravenous Gemcitabine in Locally Advanced Pancreatic Cancer: Results of a Phase III Randomized Clinical Trial,” is co-authored by Dr. Ramtin Agah, RenovoRx’s Chief Medical Officer. The abstract supports RenovoRx’s proprietary Trans-Arterial Micro-Perfusion (TAMPTM) therapy platform via additional human PK data and pre-clinical data. TAMP is designed to ensure targeted therapeutic delivery across the arterial wall near the tumor site to bathe the target tumor, while potentially minimizing a therapy’s toxicities versus systemic intravenous therapy. RenovoRx’s novel approach to locoregional treatment offers the potential for increased safety, tolerance, and improved efficacy. RenovoRx’s ongoing Phase III TIGeR-PaC clinical trial is evaluating the Company’s novel investigational drug-device combination product candidate, (intra-arterial delivery of gemcitabine via RenovoCath) known as IAG, utilizing the TAMP drug delivery platform in patients with Locally Advanced Pancreatic Cancer (LAPC). RenovoRx currently anticipates the completion of both patient enrollment and the second interim analysis for TIGeR-PaC in mid-2025. This abstract is a sub-study of the TIGeR-PaC clinical trial. The combination product candidate (IAG), which is enabled by the FDA-cleared RenovoCath device, is currently under investigation and has not been approved for commercial sale. SSO 2025 Abstract Details: Title: Pharmacodynamics of Intra-arterial vs. Intravenous Gemcitabine in Locally Advanced Pancreatic Cancer: Results of a Phase III Randomized Clinical Trial Authors: Emmanuel Zervos MD, Paula Novelli MD, Amer Zureikat MD, Michael Pishvaian MD, Kenneth Meredith MD, Hassan Hatoum MD, Reza Nazemzadeh MD, Sandeep Loria MD, Ramtin Agah MD Location: ePoster P379 at Tampa Convention Center, Tampa, FL Dates: March 27 – 29, 2025 About RenovoCath Based on its FDA clearance, RenovoCath® is intended for the isolation of blood flow and delivery of fluids, including diagnostic and/or therapeutic agents, to selected sites in the peripheral vascular system. RenovoCath is also indicated for temporary vessel occlusion in applications including arteriography, preoperative occlusion, and chemotherapeutic drug infusion. For further information regarding our RenovoCath Instructions for Use (“IFU”), please see: IFU-10004-Rev.-F-Universal-IFU.pdf. About the TIGeR-PaC Clinical Trial TIGeR-PaC is an ongoing Phase III randomized multi-center study evaluating the proprietary TAMP™ (Trans-Arterial Micro-Perfusion) therapy platform for the treatment of LAPC. RenovoRx’s first investigational drug-device combination product candidate (intra-arterial delivery of gemcitabine via RenovoCath, known as IAG) using the TAMP therapy platform enabled with the Company’s FDA-cleared RenovoCath® device for the intra-arterial administration of chemotherapy, gemcitabine. The first interim analysis in the Phase III clinical trial was completed in March 2023, with the Data Monitoring Committee recommending a continuation of the study. The TIGeR-PaC study is investigating TAMP in LAPC. The study’s primary endpoint is an overall survival benefit with secondary endpoints including reduced side effects versus standard of care. The second interim analysis for this study will be triggered by the 52nd event (i.e., patient death), which is estimated to occur in the second quarter of 2025. The second interim data readout would follow thereafter, with the timing for such readout depending on customary factors such as time needed for analysis. RenovoRx is also aiming to complete patient enrollment in the TIGeR-PaC study in mid-2025. About RenovoRx, Inc. RenovoRx is a life sciences company developing innovative targeted oncology therapies and commercializing RenovoCath®, a novel, U.S. Food and Drug Administration (FDA)-cleared local drug delivery device, targeting high unmet medical needs. RenovoRx’s patented Trans-Arterial Micro-Perfusion (TAMP™) therapy platform is designed to ensure targeted therapeutic delivery across the arterial wall near the tumor site to bathe the target tumor, while potentially minimizing a therapy’s toxicities versus systemic intravenous therapy. RenovoRx’s novel approach to targeted treatment offers the potential for increased safety, tolerance, and improved efficacy, and its mission is to transform the lives of cancer patients by providing innovative solutions to enable targeted delivery of diagnostic and therapeutic agents. In addition to the RenovoCath device, RenovoRx is also evaluating our novel Phase III drug-device combination oncology product candidate (intra-arterial gemcitabine, known as IAG). IAG is being evaluated under a U.S. investigational new drug application that is regulated by the FDA’s 21 CFR 312 pathway. The investigational IAG utilizes RenovoCath, the Company’s FDA-cleared drug-delivery device, indicated for temporary vessel occlusion in applications including arteriography, preoperative occlusion, and chemotherapeutic drug infusion. The intra-arterial infusion of chemotherapy, gemcitabine, utilizing the RenovoCath device is currently being evaluated for the treatment of LAPC by the Center for Drug Evaluation and Research (the drug division of FDA). IAG by the RenovoCath catheter is currently under investigation and has not been approved for commercial sale. RenovoCath with gemcitabine received Orphan Drug Designation for pancreatic cancer and bile duct cancer, which provides 7 years of market exclusivity upon new drug application approval by the FDA. RenovoRx is also engaged in implementing commercialization strategies utilizing its TAMP technology and FDA-cleared RenovoCath device as stand-alone device. In December 2024, RenovoRx announced the receipt of its first commercial purchase orders for RenovoCath devices. Additionally, certain of these customers have already initiated repeat orders as RenovoRx works to expand the number medical institutions that have initiated the process for RenovoCath purchase orders, including several esteemed, high volume National Cancer Institute-designated centers. To meet and satisfy the anticipated demand, RenovoRx will continue to actively explore further revenue-generating activity either on its own or in tandem with a medical device commercial partner. For more information, visit www.renovorx.com. Follow RenovoRx on Facebook, LinkedIn, and X. Cautionary Note Regarding Forward-Looking Statements This press release and statements of the Company’s management made in connection therewith contain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, and Section 21E of the Securities Exchange Act of 1934, including but not limited to statements regarding (i) our pre-clinical and clinical trials and studies, including the overall timing and timing for additional interim data readouts for our ongoing TIGeR-PaC Phase III clinical trial study in LAPC, (ii) the potential of RenovoCath® or TAMP™ as standalone commercial products, our anticipated timing for revenue generation from RenovoCath sales, and our commercialization plans in general, (iii) the potential for our product candidates to treat or provide clinically meaningful outcomes for certain medical conditions or diseases and (iv) our efforts to explore commercialization strategies utilizing our TAMP technology. Statements that are not purely historical are forward-looking statements. The forward-looking statements contained herein are based upon our current expectations and beliefs regarding future events, many of which, by their nature, are inherently uncertain, outside of our control and involve assumptions that may never materialize or may prove to be incorrect. These may include estimates, projections and statements relating to our research and development plans, commercial plans, intellectual property development, clinical trials, our therapy platform, business plans, financing plans, objectives and expected operating results, which are based on current expectations and assumptions that are subject to significant known and unknown risks and uncertainties that may cause actual results to differ materially and adversely from those expressed or implied by these forward-looking statements. These statements may be identified using words such as “may,” “expects,” “plans,” “aims,” “anticipates,” “believes,” “forecasts,” “estimates,” “intends,” and “potential,” or the negative of these terms or other comparable terminology regarding RenovoRx’s expectations strategy, plans or intentions, although not all forward-looking statements contain these words. These forward-looking statements are subject to a number of risks, uncertainties and assumptions, that could cause actual events to differ materially from those projected or indicated by such statements, including, among other things: (i) the risk that our execution of our commercial strategy for RenovoCath or our TAMP technology may not lead to viable or repeating revenue generating operations; (ii) circumstances which would adversely impact our ability to efficiently utilize our cash resources on hand or raise additional funding, (iii) the timing of the initiation, progress and potential results (including the results of interim analyses) of TIGeR-PaC and any other preclinical studies, clinical trials and our research programs; (iv) the possibility that interim results may not be predictive of the outcome of our clinical trials, which may not demonstrate sufficient safety and efficacy to support regulatory approval of our product candidate, (v) that the applicable regulatory authorities may disagree with our interpretation of the data; research and clinical development plans and timelines, and the regulatory process for our product candidates; (vi) future potential regulatory milestones for our product candidates, including those related to current and planned clinical studies; (vii) our ability to use and expand our therapy platform to build a pipeline of product candidates; (viii) our ability to advance product candidates into, and successfully complete, clinical trials; (ix) the timing or likelihood of regulatory filings and approvals; (x) our estimates of the number of patients who suffer from the diseases we are targeting and the number of patients that may enroll in our clinical trials; (xi) the commercialization potential of our product candidates, if approved; (xii) our ability and the potential to successfully manufacture and supply our product candidates for clinical trials and for commercial use, if approved; (xiii) future strategic arrangements and/or collaborations and the potential benefits of such arrangements; (xiv) our estimates regarding expenses, future revenue, capital requirements and needs for additional financing and our ability to obtain additional capital; (xv) the sufficiency of our existing cash and cash equivalents to fund our future operating expenses and capital expenditure requirements; (xvi) our ability to retain the continued service of our key personnel and to identify, and hire and retain additional qualified personnel; (xvii) the implementation of our strategic plans for our business and product candidates; (xviii) the scope of protection we are able to establish and maintain for intellectual property rights, including our therapy platform, product candidates and research programs; (xix) our ability to contract with third-party suppliers and manufacturers and their ability to perform adequately; (xx) the pricing, coverage and reimbursement of our product candidates, if approved; and (xxi) developments relating to our competitors and our industry, including competing product candidates and therapies. Information regarding the foregoing and additional risks may be found in the section entitled “Risk Factors” in documents that we file from time to time with the Securities and Exchange Commission. Forward-looking statements included herein are made as of the date hereof, and RenovoRx does not undertake any obligation to update publicly such forward-looking statements to reflect subsequent events or circumstances, except as required by law. View source version on businesswire.com: https://www.businesswire.com/news/home/20250319438801/en/ KCSA Strategic Communications Valter Pinto or Jack Perkins T:212-896-1254 RenovoRX@KCSA.com Source: RenovoRx, Inc. Released March 19, 2025

Phase 3ASCOOrphan Drug

18 Mar 2025

·pipelinereview.com

Investigational Rinatabart Sesutecan (Rina-S®) Continues to Show Encouraging Antitumor Activity in Patients with Advanced Ovarian Cancer

COPENHAGEN, Denmark I March 17, 2025 I Genmab A/S (Nasdaq: GMAB) announced today updated data from cohort B1 of the Phase 1/2 RAINFOL-01 study of rinatabart sesutecan (Rina-S ® ), an investigational folate receptor-alpha (FRα)-targeted, TOPO1 antibody-drug conjugate (ADC) that showed Rina-S 120 mg/m 2 every 3 weeks (Q3W) resulted in a confirmed objective response rate (ORR) of 55.6% (95% CI: 30.8-78.5) in heavily pre-treated ovarian cancer (OC) patients regardless of FRα expression levels. With a median on-study follow-up of 48 weeks, 1 out of 10 patients experienced disease progression and the median duration of response (mDOR) was not reached (95% CI: 40.14-NR). The data are from the dose expansion cohort of the multi-part study evaluating the safety and efficacy of Rina-S as a single agent in solid tumors that are known to express FRα and were presented at the 2025 Society of Gynecologic Oncology Annual Meeting on Women’s Cancer ® (SGO) in Seattle, Washington. “The antitumor activity observed in the dose expansion cohort continues to demonstrate the potential for a much-needed treatment option for patients with PROC, who have historically had poor prognosis. I am hopeful that further exploration of Rina-Swill lead to advancements in the treatment landscape.” said Elizabeth Lee, M.D., a medical oncologist in the gynecologic oncology program at Dana-Farber. The B1 cohort is a dose expansion study in patients with histologically or cytologically confirmed advanced OC (epithelial ovarian cancer, primary peritoneal cancer or fallopian tube cancer). Rina-S 120 mg/m 2 Q3W at a median on-study follow-up of 48 weeks showed encouraging antitumor activity; the confirmed ORR was 55.6% (95% CI: 30.8-78.5), the disease control rate (DCR) was 88.9% (95% CI: 65.3-98.6), and the median duration of response (mDOR) was not reached (95% CI: 40.14-NR). In the 18 patients evaluable for response treated with 120 mg/m 2 Q3W, complete responses were observed in 4 patients (2 confirmed; 2 unconfirmed) and 8 patients experienced confirmed partial responses (44.4%). Most responses with Rina-S 120 mg/m 2 were observed early (at week 6). Only one patient in the 120 mg/m 2 treatment arm was not evaluable. In the Rina-S 100 mg/m 2 Q3W treatment arm (N=22), at a median on study follow-up of 46 weeks, the confirmed ORR was 22.7% (95% CI: 7.8-45.4), the DCR was 86.4% (95% CI: 65.1-97.1), and the mDOR was not reached (95% CI, 16.3-NR). Partial responses were observed in 4 patients (18.2%) and 1 patient (4.5%) experienced a complete response. Rina-S 120 mg/m 2 has been selected for further evaluation in the RAINFOL-01 and Phase 3 RAINFOL-02 trials for patients with platinum resistant ovarian cancer (PROC). In this Phase 1/2 study, common treatment-emergent adverse events (TEAEs) included anemia, nausea, neutropenia, leukopenia, fatigue, thrombocytopenia, vomiting, diarrhea, alopecia, and hypokalemia. Dose reductions and treatment discontinuations were infrequent and no new safety signals were observed. “The updated results reinforce the potential of Rina-S and further validate our development approach in advanced ovarian cancer,” said Judith Klimovsky, M.D., Executive Vice President and Chief Development Officer of Genmab. “We are excited to keep moving forward with the ongoing Phase 3 trial, to evaluate the potential of Rina-S as a treatment option for patients facing this challenging disease.” The safety and efficacy of rinatabart sesutecan has not been established for these investigational uses. About the RAINFOL TM -01 Trial RAINFOL-01 ( NCT05579366 ) is an open-label, multicenter Phase 1/2 study, designed to evaluate the safety and efficacy of rinatabart sesutecan (Rina-S) as a single agent Q3W at various doses in solid tumors that are known to express FRα. The study consists of multiple parts including Part A dose-escalation cohorts; Part B tumor-specific monotherapy dose-expansion cohorts; Part C platinum-resistant ovarian cancer (PROC) cohort; and Part D combination therapy cohorts. Part B of the trial includes the B1 cohort, a dose expansion study in patients with histologically or cytologically confirmed advanced OC (epithelial ovarian cancer, primary peritoneal cancer, or fallopian tube cancer). Patients were randomized 1:1 to 100 mg/m 2 and 120 mg/m 2 dose cohorts. Median age was 62.5 and 64.5 years in the 100 mg/m 2 and 120 mg/m 2 cohorts, respectively. Study participants were previously treated with a median of 3 prior lines of therapy (range 1-4). Patients received prior treatment with bevacizumab (90.9% in the 100 mg/m 2 group and 90.0% in the 120 mg/m 2 group respectively), PARP inhibitors (68.2%; 65%), and mirvetuximab soravtansin (18.2%; 19%). Initial results from Part B of this trial were presented during a mini-oral session at the European Society of Medical Oncology Congress 2024 (ESMO). About Ovarian Cancer Ovarian cancer is a major global health issue, with over 320,000 new cases diagnosed annually worldwide. i It ranks as the eighth most common cancer and the eighth leading cause of cancer-related deaths among women globally. ii The disease is often diagnosed at an advanced stage due to its subtle and non-specific symptoms, such as abdominal bloating, pelvic pain and difficulty eating. iii Standard of care for platinum resistant ovarian cancer typically involves single agent chemotherapy (pegylated liposomal doxorubicin (PLD), topotecan, gemcitabine or paclitaxel). iv Approximately 70-90% of women with advanced-stage ovarian cancer worldwide experience a recurrence after initial treatment. v Ovarian cancer has a low five-year survival rate, which varies significantly by region, but generally hovers around 30-50%. vi,vii About Rinatabart Sesutecan (Rina-S ® ; GEN1184) Rinatabart sesutecan (Rina-S; GEN1184) is a FRα-targeted, TOPO1 ADC, currently being evaluated for the potential treatment of ovarian cancer and other FRα-expressing cancers. A Phase 3 trial (RAINFOL-02, NCT06619236 ) evaluating Rina-S in patients with platinum resistant ovarian cancer compared to treatment of investigator’s choice is ongoing. In January 2024, the U.S. Food and Drug Administration granted Fast Track designation to Rina-S for the treatment of patients with FRα-expressing high-grade serous or endometrioid platinum-resistant ovarian cancer. Please visit www.clinicaltrials.gov for more information. About Genmab Genmab is an international biotechnology company with a core purpose of guiding its unstoppable team to strive toward improving the lives of patients with innovative and differentiated antibody therapeutics. For more than 25 years, its passionate, innovative and collaborative team has invented next-generation antibody technology platforms and leveraged translational, quantitative and data sciences, resulting in a proprietary pipeline including bispecific T-cell engagers, antibody-drug conjugates, next-generation immune checkpoint modulators and effector function-enhanced antibodies. By 2030, Genmab’s vision is to transform the lives of people with cancer and other serious diseases with knock-your-socks-off (KYSO) antibody medicines ® . Established in 1999, Genmab is headquartered in Copenhagen, Denmark, with international presence across North America, Europe and Asia Pacific. For more information, please visit Genmab.com and follow us on LinkedIn and X . i World Cancer Research Fund International. https://www.wcrf.org/cancer-trends/ovarian-cancer-statistics/ . Accessed August 2024. ii World Ovarian Cancer Coalition. https://worldovariancancercoalition.org/about-ovarian-cancer/key-stats/ . Accessed August 2024. iii Dilley, James et al. Ovarian cancer symptoms, routes to diagnosis and survival – Population cohort study in the ‘no screen’ arm of the UK Collaborative Trial of Ovarian Cancer Screening (UKCTOCS). Gynecologic oncology vol. 158,2 (2020): 316-322. doi:10.1016/j.ygyno.2020.05.002. iv Eskander RN, Moore KN, Monk BJ, Herzog TJ, Annunziata CM, O’Malley DM and Coleman RL (2023) Overcoming the challenges of drug development in platinum-resistant ovarian cancer. Front. Oncol. 13:1258228 Ovarian Cancer Research Alliance. https://ocrahope.org/patients/diagnosis-and-treatment/recurrence/ . v Ovarian Cancer Research Alliance. https://ocrahope.org/patients/diagnosis-and-treatment/recurrence/ . vi European Institute of Women’s Health. https://eurohealth.ie/policy‐brief‐women‐and‐ovarian‐cancer‐in‐the‐eu‐2018/ . Accessed August 2024. vii American Cancer Society. Stages of Ovarian Cancer. https://www.cancer.org/cancer/types/ovarian-cancer/detection-diagnosis-staging/survival-rates.html. Accessed August 2024 . SOURCE: Genmab

Clinical ResultPhase 3ASCOFast Track

17 Mar 2025

·pipelinereview.com

Imfinzi approved in the EU as first and only immunotherapy for limited-stage small cell lung cancer

Approval based on ADRIATIC Phase III trial results which showed a 27% reduction in the risk of death versus placebo LONDON, UK I March 17, 2025 I AstraZeneca’s Imfinzi (durvalumab) has been approved in the European Union (EU) as monotherapy for the treatment of adults with limited-stage small cell lung cancer (LS-SCLC) whose disease has not progressed following platinum-based chemoradiation therapy (CRT). The approval by the European Commission follows the positive opinion of the Committee for Medicinal Products for Human Use and is based on results from the ADRIATIC Phase III trial, which were published in The New England Journal of Medicine . SCLC is a highly aggressive form of lung cancer, with an estimated 8,000 people treated for LS-SCLC across the five major European countries each year. 1-2 LS-SCLC typically recurs and progresses rapidly, despite initial response to standard-of-care chemotherapy and radiotherapy. 3-4 The prognosis for LS-SCLC is particularly poor, as only 15-30% of patients survive for five years after diagnosis. 5 Suresh Senan, PhD, radiation oncologist at the Amsterdam University Medical Centers, The Netherlands, and principal investigator in the trial, said: “This approval marks a turning point for patients with limited-stage small cell lung cancer in Europe, bringing them an immunotherapy option for the first time. An unprecedented 57% of patients treated with durvalumab were still alive at three years in the ADRIATIC trial. This significant advance establishes a new benchmark in a setting where the standard of care has remained unchanged for decades.” Dave Fredrickson, Executive Vice President, Oncology Haematology Business Unit, AstraZeneca, said: “Imfinzi has the potential to transform how limited-stage small cell lung cancer is treated as the first immunotherapy approved in Europe in this setting. As the only immunotherapy approved for both early and late-stage disease, Imfinzi is poised to become the foundation for transforming outcomes for people with small cell lung cancer.” In the trial, results showed Imfinzi reduced the risk of death by 27% versus placebo (based on an overall survival [OS] hazard ratio [HR] of 0.73; 95% confidence interval [CI] 0.57-0.93; p=0.0104). Estimated median OS was 55.9 months for Imfinzi versus 33.4 months for placebo. An estimated 57% of patients treated with Imfinzi were alive at three years compared to 48% for placebo. Imfinzi also reduced the risk of disease progression or death by 24% (based on a progression-free survival [PFS] HR of 0.76; 95% CI 0.61-0.95; p=0.0161) versus placebo. Median PFS was 16.6 months for Imfinzi versus 9.2 months for placebo. An estimated 46% of patients treated with Imfinzi had not experienced disease progression at two years compared to 34% for placebo. The safety profile for Imfinzi was generally manageable and consistent with the known profile of this medicine. No new safety signals were observed. Imfinzi is approved in the US and several other countries in this setting based on the ADRIATIC results. Regulatory applications are currently under review in Japan and several other countries for this indication. Imfinzi is also approved in combination with chemotherapy for the treatment of extensive-stage SCLC based on the CASPIAN Phase III trial. Notes Small cell lung cancer Lung cancer is the leading cause of cancer death among both men and women, accounting for about one-fifth of all cancer deaths. 6-7 Lung cancer is broadly split into non-small cell lung cancer (NSCLC) and SCLC, with about 15% of cases classified as SCLC. 8 LS-SCLC (Stage I-III) accounts for approximately 30% of SCLC diagnoses and is classified when the disease is generally only in one lung or one side of the chest. 9 The prognosis for patients with LS-SCLC remains poor despite curative-intent treatment with standard-of-care concurrent CRT (cCRT). 10 ADRIATIC The ADRIATIC trial is a randomised, double-blind, placebo-controlled, multi-centre global Phase III trial evaluating Imfinzi monotherapy and Imfinzi plus Imjudo (tremelimumab) versus placebo in the treatment of 730 patients with LS-SCLC who had not progressed following cCRT. In the experimental arms, patients were randomised to receive a 1500mg fixed dose of Imfinzi with or without Imjudo 75mg every four weeks for up to four doses/cycles each, followed by Imfinzi every four weeks for up to 24 months. The dual primary endpoints were PFS and OS for Imfinzi monotherapy versus placebo. Key secondary endpoints included OS and PFS for Imfinzi plus Imjudo versus placebo, safety and quality of life measures. The trial included 164 centres in 19 countries across North and South America, Europe and Asia. Imfinzi Imfinzi (durvalumab) is a human monoclonal antibody that binds to the PD-L1 protein and blocks the interaction of PD-L1 with the PD-1 and CD80 proteins, countering the tumour’s immune-evading tactics and releasing the inhibition of immune response s. In addition to its indications in SCLC, Imfinzi is also the global standard of care based on OS in the curative-intent setting of unresectable, Stage III NSCLC in patients whose disease has not progressed after CRT. Additionally, Imfinzi is approved as a perioperative treatment in combination with neoadjuvant chemotherapy in resectable NSCLC; and in combination with a short course of Imjudo and chemotherapy for the treatment of metastatic NSCLC. Imfinzi is also approved in combination with chemotherapy (gemcitabine plus cisplatin) in locally advanced or metastatic biliary tract cancer and in combination with Imjudo in unresectable hepatocellular carcinoma (HCC). Imfinzi is also approved as a monotherapy in unresectable HCC in Japan and the EU. In March 2025, perioperative Imfinzi added to standard-of-care chemotherapy met the primary endpoint of event-free survival in the MATTERHORN Phase III trial in resectable gastric and gastroesophageal junction cancers. Imfinzi in combination with chemotherapy followed by Imfinzi monotherapy is approved as a 1 st -line treatment for primary advanced or recurrent endometrial cancer (mismatch repair deficient disease only in US and EU). Imfinzi in combination with chemotherapy followed by Lynparza (olaparib) and Imfinzi is approved for patients with mismatch repair proficient advanced or recurrent endometrial cancer in EU and Japan. Since the first approval in May 2017, more than 374,000 patients have been treated with Imfinzi . As part of a broad development programme, Imfinzi is being tested as a single treatment and in combinations with other anti-cancer treatments for patients with SCLC, NSCLC, bladder cancer, breast cancer, several gastrointestinal and gynaecologic cancers, and other solid tumours. AstraZeneca in lung cancer AstraZeneca is working to bring patients with lung cancer closer to cure through the detection and treatment of early-stage disease, while also pushing the boundaries of science to improve outcomes in the resistant and advanced settings. By defining new therapeutic targets and investigating innovative approaches, the Company aims to match medicines to the patients who can benefit most. The Company’s comprehensive portfolio includes leading lung cancer medicines and the next wave of innovations, including Tagrisso (osimertinib) and Iressa (gefitinib); Imfinzi and Imjudo ; Enhertu (trastuzumab deruxtecan) and Datroway (datopotamab deruxtecan) in collaboration with Daiichi Sankyo; Orpathys (savolitinib) in collaboration with HUTCHMED; as well as a pipeline of potential new medicines and combinations across diverse mechanisms of action. AstraZeneca is a founding member of the Lung Ambition Alliance, a global coalition working to accelerate innovation and deliver meaningful improvements for people with lung cancer, including and beyond treatment. AstraZeneca in immuno-oncology (IO) AstraZeneca is a pioneer in introducing the concept of immunotherapy into dedicated clinical areas of high unmet medical need. The Company has a comprehensive and diverse IO portfolio and pipeline anchored in immunotherapies designed to overcome evasion of the anti-tumour immune response and stimulate the body’s immune system to attack tumours. AstraZeneca strives to redefine cancer care and help transform outcomes for patients with Imfinzi as a monotherapy and in combination with Imjudo as well as other novel immunotherapies and modalities. The Company is also investigating next-generation immunotherapies like bispecific antibodies and therapeutics that harness different aspects of immunity to target cancer, including cell therapy and T-cell engagers. AstraZeneca is pursuing an innovative clinical strategy to bring IO-based therapies that deliver long-term survival to new settings across a wide range of cancer types. The Company is focused on exploring novel combination approaches to help prevent treatment resistance and drive longer immune responses. With an extensive clinical programme, the Company also champions the use of IO treatment in earlier disease stages, where there is the greatest potential for cure. AstraZeneca in oncology AstraZeneca is leading a revolution in oncology with the ambition to provide cures for cancer in every form, following the science to understand cancer and all its complexities to discover, develop and deliver life-changing medicines to patients. The Company’s focus is on some of the most challenging cancers. It is through persistent innovation that AstraZeneca has built one of the most diverse portfolios and pipelines in the industry, with the potential to catalyse changes in the practice of medicine and transform the patient experience. AstraZeneca has the vision to redefine cancer care and, one day, eliminate cancer as a cause of death. AstraZeneca AstraZeneca (LSE/STO/Nasdaq: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development, and commercialisation of prescription medicines in Oncology, Rare Diseases, and BioPharmaceuticals, including Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Based in Cambridge, UK, AstraZeneca’s innovative medicines are sold in more than 125 countries and used by millions of patients worldwide. Please visit astrazeneca.com and follow the Company on social media @AstraZeneca . References 1. National Cancer Institute. NCI Dictionary – Small Cell Lung Cancer. Available at: https://www.cancer.gov/publications/dictionaries/cancer-terms/def/small-cell-lung-cancer . Accessed March 2025. 2. AstraZeneca PLC. Investor Relations Epidemiology Spreadsheet. Available at: https://www.astrazeneca.com/investor-relations.html . Accessed March 2025. 3. Qin A, Kalemkerian GP. Treatment Options for Relapsed Small-Cell Lung Cancer: What Progress Have We Made? J Oncol Pract . 2018;14(6):369-370. 4. Cheng Y, et al. Durvalumab after Chemoradiotherapy in Limited-Stage Small-Cell Lung Cancer. N Engl J Med . 2024;391(14):1313-1327. 5. Bebb DG, et al. Symptoms and Experiences with Small Cell Lung Cancer: A Mixed Methods Study of Patients and Caregivers. Pulm Ther . 2023;9:435-450. 6. World Health Organization. International Agency for Research on Cancer. Lung Fact Sheet. Available at: https://gco-iarc-who-int.libproxy1.nus.edu.sg/media/globocan/factsheets/cancers/15-trachea-bronchus-and-lung-fact-sheet.pdf . Accessed March 2025. 7. World Health Organization. International Agency for Research on Cancer. World Fact Sheet. Available at: https://gco-iarc-who-int.libproxy1.nus.edu.sg/media/globocan/factsheets/populations/900-world-fact-sheet.pdf . Accessed March 2025. 8. LUNGevity Foundation. Types of Lung Cancer. Available at: https://www.lungevity.org/for-patients-caregivers/lung-cancer-101/types-of-lung-cancer . Accessed March 2025. 9. American Cancer Society. Small Cell Lung Cancer Stages. Available at: https://www.cancer.org/cancer/types/lung-cancer/detection-diagnosis-staging/staging-sclc.html . Accessed March 2025. 10. Senan S, et al. ADRIATIC: A phase III trial of durvalumab ± tremelimumab after concurrent chemoradiation for patients with limited stage small cell lung cancer. Ann Oncol . 2019;30(suppl. 2):ii25. SOURCE: AstraZeneca

ImmunotherapyPhase 3Clinical ResultDrug Approval

100 Deals associated with Gemcitabine

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KEGG	Wiki	ATC	Drug Bank
D02368	Gemcitabine	L01BC05	DB00441

R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Pancreatic Cancer	Phase 3	-	RenovoRx, Inc.	01 Jun 2023
Metastatic Pancreatic Ductal Adenocarcinoma	Phase 3	Australia	Panbela Therapeutics, Inc.	04 Aug 2022
Locally Advanced Pancreatic Adenocarcinoma	Phase 3	United States	RenovoRx, Inc.	12 Mar 2018
Locally Advanced Pancreatic Adenocarcinoma	Phase 3	Belgium	RenovoRx, Inc.	12 Mar 2018
Advanced Lung Squamous Cell Carcinoma	Phase 3	United States	Sanofi	01 Mar 2010
Advanced Lung Squamous Cell Carcinoma	Phase 3	Belgium	Sanofi	01 Mar 2010
Advanced Lung Squamous Cell Carcinoma	Phase 3	Canada	Sanofi	01 Mar 2010
Advanced Lung Squamous Cell Carcinoma	Phase 3	France	Sanofi	01 Mar 2010
Advanced Lung Squamous Cell Carcinoma	Phase 3	Germany	Sanofi	01 Mar 2010
Advanced Lung Squamous Cell Carcinoma	Phase 3	Hungary	Sanofi	01 Mar 2010

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


ASCO_GU2025	Not Applicable	Non-Muscle Invasive Bladder Neoplasms	30	Intravesical Gemcitabine and Docetaxel	elzqlcdxyr(uwzcpajjci) = ingqewjmtj krejjwsrus (zvwsnpnnzf ) View more	Positive	13 Feb 2025
ASCO_GU2025	Not Applicable	Non-Muscle Invasive Bladder Neoplasms Consensus subtyping models	143	Intravesical Bacillus Calmette-Guerin (BCG)	gehzdnqegn(lhdhroqqpf) = atriytkfxw qpnummhksg (lzjzuimkzy )	Positive	13 Feb 2025
				Sequential intravesical gemcitabine and docetaxel (Gem/Doce)	gehzdnqegn(lhdhroqqpf) = jduihgjlwe qpnummhksg (lzjzuimkzy )
NCT01972919 (CTgov)	Phase 2	Pancreatic carcinoma non-resectable	23	Radiation Therapy+Gemcitabine+Capecitabine	qjpuyyqhcy = haaadpodde gruulazvxd (fuzyksbllo, usbhiezeje - auwbelvntf) View more	-	10 Feb 2025
NCT03257033 (TIGeR-PaC, ASCO_GI2025)	Phase 3	Locally Advanced Pancreatic Adenocarcinoma	19	Intra-arterial gemcitabine	eyutzfnazj(mhfrmehtqs) = zevolmcbtl hixwpufmse (rhpgpzdalk )	Positive	23 Jan 2025
				Intravenous gemcitabine	eyutzfnazj(mhfrmehtqs) = kcxepomyqt hixwpufmse (rhpgpzdalk )
NCT04229004 (Precision Promise (PrP), ASCO_GI2025)	Phase 2/3	Metastatic Pancreatic Cancer Second line	317	Pamrevlumab plus nab-paclitaxel/gemcitabine (Pam + GA)	tqcmarxcvn(sqorasmkia): HR = 1.18 (95% CI, 0.88 - 1.56), P-Value = 0.14 View more	Negative	23 Jan 2025
				Gemcitabine/Abraxane (GA)
NCT05042128 (AGITG ASCEND, ASCO_GI2025)	Phase 2	Metastatic Pancreatic Ductal Adenocarcinoma First line	95	Certepetide + Gemcitabine/Nab-paclitaxel	akukgwcaxs(wlbspbxlgm) = ontsogcswr tjobjevizs (kenttbubfk, 36.4% - 60.5%) View more	Positive	23 Jan 2025
				Placebo + Gemcitabine/Nab-paclitaxel	akukgwcaxs(wlbspbxlgm) = obkwrmjchv tjobjevizs (kenttbubfk, 22.6% - 58.3%) View more
ACTRN12623000223639 (ASCO_GI2025)	Not Applicable	Advanced Pancreatic Ductal Adenocarcinoma	30	Gemcitabine + Nab-paclitaxel + LSTA-1 + Durvalumab	abbrpydsbr(giqqyczibh) = dwvqehmgda moibbjailh (coblzwxpve )	Positive	23 Jan 2025
ASCO_GI2025	Not Applicable	Pancreatic Ductal Adenocarcinoma DNA methylation changes	184	Gemcitabine	ltiaefkwfa(jxvznhewbv) = txjfvmjpmm djodxxnisw (zxhsqgxbwb ) View more	-	23 Jan 2025
				Fluorouracil (5-FU)	ltiaefkwfa(jxvznhewbv) = hefhkqfqnv djodxxnisw (zxhsqgxbwb )
ASH2024	Not Applicable	Diffuse Large B-Cell Lymphoma	-	Glofitamab with Gemcitabine and Oxaliplatin	xpfbkiamyx(qhikgwzfnd) = ktbdufjcbn twswudmnwt (oekknwvuzl, NA) View more	-	09 Dec 2024
				Rituximab with Gemcitabine and Oxaliplatin	xpfbkiamyx(qhikgwzfnd) = uaiqjapvsy twswudmnwt (oekknwvuzl, NA) View more
ASH2024	Not Applicable	Classical Hodgkin's Lymphoma	-	Pembrolizumab Gemcitabine Vinorelbine and Liposomal Doxorubicin (P-GVD)	gyixcbczcf(guqonqznzj) = zwrpjagkhz jwlpuhyhla (kgimbbtfpo ) View more	-	09 Dec 2024

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