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Gallbladder cancer is rare but aggressive gastrointestinal malignancy which represents a significant unmet clinical need due to late-stage diagnosis and limited treatment options. Globally, gallbladder cancer accounted for approximately 122,491 new cases and over 89,055 deaths in 2022, with higher prevalence in Asia and parts of South America. The gallbladder cancer pipeline analysis by Expert Market Research highlights increasing research activity focused on targeted therapies, immuno-oncology agents, and biomarker-driven approaches aimed at improving outcomes in this high-mortality cancer.
Major companies involved in the gallbladder cancer pipeline analysis include AccSalus Biosciences, Inc., Tango Therapeutics, Inc., and others.
Leading drugs currently in the pipeline include ACC-1898, CTX-009 and others.
Rising identification of actionable alterations such as HER2 overexpression, FGFR mutations, and DNA damage repair defects in gallbladder cancer is uniquely driving pipeline growth. These molecular insights are enabling targeted and biomarker-driven drug development, accelerating investment in precision therapies for a historically neglected cancer with limited standard treatment options.
The Gallbladder Cancer Pipeline Analysis Report by Expert Market Research gives comprehensive insights into gallbladder cancer therapeutics currently undergoing clinical trials. It covers various aspects related to the details of each of these drugs under development for gallbladder cancer. The gallbladder cancer report assessment includes the analysis of over 100 pipeline drugs and 50+ companies. The gallbladder cancer pipeline landscape will include an analysis based on efficacy and safety measure outcomes published for the trials, including their adverse effects on patients suffering from the condition, and alignment with gallbladder cancer treatment guidelines to ensure optimal care practices.
The assessment part will include a detailed analysis of each drug, drug class, clinical studies, phase type, drug type, route of administration, and ongoing product development activities related to gallbladder cancer.

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The gallbladder cancer pipeline outlook highlights growing efforts to address a disease with limited effective systemic options. Standard treatment typically involves surgical resection for early-stage disease, followed by chemotherapy regimens such as gemcitabine-based combinations in advanced settings, where outcomes remain poor. Pipeline development is increasingly focused on targeted and novel therapies to improve survival.
Gallbladder cancer treatment is witnessing renewed momentum as regulatory advancements support innovation within this underserved oncology space. In October 2025, the US FDA granted Orphan Drug Designation to MDX-124, a first-in-class therapeutic under evaluation for biliary tract cancers, including gallbladder cancer. Early Phase 1b data demonstrated encouraging tumor control and tolerability, supporting continued clinical development and reinforcing innovation within this underserved oncology pipeline.
Gallbladder cancer is a relatively rare yet highly fatal malignancy, ranking 22nd in global cancer incidence with an estimated 122,491 new cases and 89,055 deaths worldwide in 2022 according to GLOBOCAN. Incidence and mortality rates vary significantly by region, with the highest cases reported in China, India, and Japan, and age-standardized rates elevated in parts of South America. The disease disproportionately affects adults aged 50–74, and mortality remains high due to late diagnosis and limited screening programs.
This section of the report covers the analysis of gallbladder cancer drug candidates based on several segmentations, including:
By Phase
The pipeline assessment report covers 50+ drug analyses based on phase:
By Drug Class
The gallbladder cancer pipeline analysis report covers 50+ drug analyses based on drug classes:
By Route of Administration
The pipeline assessment report covers 50+ drug analyses based on the route of administration:
The report covers phase I, phase II, phase III, phase IV, and early-phase drugs. The coverage includes an in-depth analysis of each drug across these phases. According to EMR analysis, phase II covers a major share of the total gallbladder cancer clinical trials. Phase II dominates with 60%, followed by phase III 24%, and phase I with 15%, highlighting mid-stage development focus. Overall, this distribution reflects a strong emphasis on mid-stage clinical validation, with a growing pipeline advancing toward late-stage development to address unmet therapeutic needs in gallbladder cancer.
The drug molecule categories covered under the gallbladder cancer pipeline analysis include small molecules, monoclonal antibodies, peptides, and Polymers. The gallbladder cancer report provides a comparative analysis of the drug classes for each drug in various phases of clinical trials for gallbladder cancer. In November 2024,
the U.S. FDA granted accelerated approval to zanidatamab-hrii (Ziihera®) for adults with previously treated, unresectable or metastatic HER2-positive biliary tract cancer, which includes gallbladder cancer. This bispecific HER2-targeting antibody showed a ~52 % objective response rate in the HERIZON-BTC-01 trial, representing a significant advance where few targeted options existed. The approval also included a companion diagnostic to identify eligible patients.
The EMR report for the gallbladder cancer pipeline covers the profile of key companies involved in clinical trials and their drugs under development. It provides a detailed gallbladder cancer therapeutic assessment, analyzing the competitive dynamics of the clinical trial landscape. Below is the list of a few players involved in gallbladder cancer clinical trials:
This section covers the detailed analysis of each drug under multiple phases, including phase I, phase II, phase III, phase IV, and emerging drugs for gallbladder cancer. It includes product description, trial ID, study type, drug class, mode of administration, and recruitment status of gallbladder cancer drug candidates.
ACC‑1898 is an investigational small‑molecule tyrosine kinase inhibitor (TKI) designed to block multiple aberrant receptor tyrosine kinases that drive tumor growth and spread. It works by inhibiting signaling pathways critical to cancer cell proliferation and survival in advanced solid tumors, including gallbladder cancer, within a Phase I dose‑escalation study. The candidate is being developed by AccSalus Biosciences, a clinical‑stage biopharmaceutical company focused on novel oncology therapies, exploring safety, pharmacokinetics, and early antitumor activity.
CTX-009 (tovecimig) is an investigational bispecific antibody sponsored by Compass Therapeutics and is being evaluated in the Phase II/III COMPANION-002 clinical trial (NCT05506943) for the treatment of unresectable, advanced, metastatic, or recurrent biliary tract cancers, including gallbladder cancer. The drug simultaneously targets delta-like ligand 4 (DLL4) and vascular endothelial growth factor-A (VEGF-A), two key regulators of tumor angiogenesis, thereby disrupting abnormal tumor blood vessel formation and potentially enhancing the efficacy of chemotherapy. In this randomized, open-label study, CTX-009 is administered in combination with paclitaxel and compared against paclitaxel monotherapy, with overall response rate as the primary endpoint and progression-free survival and overall survival as key secondary endpoints.
*Please note that this is only a partial list; the complete list of drugs will be available in the full report.*
The Gallbladder Cancer Pipeline Analysis Report provides a strategic overview of the latest and future landscape of treatments for gallbladder cancer. It provides necessary information for making informed investment decisions along with research, development, and strategic planning efforts. The stakeholders will benefit from the essential insights into gallbladder cancer collaborations, regulatory environments, and potential growth opportunities.
*While we strive to always give you current and accurate information, the numbers depicted on the website are indicative and may differ from the actual numbers in the main report. At Expert Market Research, we aim to bring you the latest insights and trends in the market. Using our analyses and forecasts, stakeholders can understand the market dynamics, navigate challenges, and capitalize on opportunities to make data-driven strategic decisions.*
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