Biliary Tract Cancer
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Jul 20 – Jul 27, 2026
Domvanalimab plus zimberelimab in unresectable and immunotherapy refractory biliary tract cancers: a phase 2 trial.
NAT COMMUN · Q1 JOURNAL - RANK #10/135TOP-TIER
This phase 2, prospective clinical trial (NCT05724563) investigated the Fc-silent anti-TIGIT antibody domvanalimab combined with the anti-PD-1 antibody zimberelimab in 29 patients with unresectable biliary tract cancers that had progressed after prior PD-1/L1 therapy. Participants received the combination therapy and were assessed for confirmed objective response rate (primary end-point) alongside disease control rate, 6-month progression-free survival, duration of response, overall survival, and safety. The confirmed objective response rate was 10.3 % (95 % CI 2.2–27.4 %), disease control rate 44.8 %, 6-month PFS rate 17.2 % (95 % CI 6.0–35.8 %), and median duration of response 13.6 months; treatment-related adverse events occurred in 36.4 %, mainly low-grade pruritus and rash. Although the prespecified ORR target was not met, the durable responses, favorable safety, and translational biomarker insights support further evaluation of anti-TIGIT-based combinations in refractory biliary tract cancer.
10.1038/s41467-026-75554-z
Jul 06 – Jul 13, 2026
Durvalumab Plus Chemotherapy for Advanced Biliary Tract Cancer: A Post Hoc Analysis of the TOPAZ-1 Randomized Clinical Trial.
JAMA ONCOL · Q1 JOURNAL - RANK #14/326TOP-TIER
This post hoc analysis of the global, double-blind, placebo-controlled, phase 3 TOPAZ-1 randomized clinical trial assessed 4-year overall survival (OS) and safety of durvalumab plus gemcitabine/cisplatin (GemCis) vs placebo+GemCis in advanced biliary tract adenocarcinoma. 685 adults were randomized at 105 sites in 17 countries; treatment was GemCis on days 1 and 8 every 3 weeks for up to 8 cycles, then durvalumab or placebo every 4 weeks; outcomes were assessed ≈48 months after the last randomization (data cutoff Feb 28, 2025). Median OS was 13.0 months (95% CI, 11.6-14.1) with durvalumab+GemCis vs 11.4 months (95% CI, 10.1-12.5) with placebo+GemCis (HR, 0.75; 95% CI, 0.64-0.88); 48-month OS rates were 11.8% vs 4.3%. Serious treatment-related adverse events occurred in 15.4% vs 17.3%, and discontinuations in 6.2% vs 5.3%, supporting clinically manageable safety and durable survival benefit.
10.1001/jamaoncol.2026.2204
Ivonescimab plus gemcitabine and cisplatin as first-line therapy for advanced biliary tract cancer: a multicenter, open-label phase 2 trial.
J HEPATOL · Q1 JOURNAL - RANK #3/147TOP-TIER
This multicenter, open-label phase II trial evaluated the combination of ivonescimab with gemcitabine and cisplatin as a first-line therapy for 30 treatment-naive patients with unresectable locally advanced or metastatic biliary tract cancer (BTC). The primary endpoint was the objective response rate (ORR), which was 66.7% (95% CI: 47.2–82.7%) with 1 complete response and 19 partial responses; the median progression-free survival (mPFS) was 8.5 months and the median overall survival (mOS) was 16.8 months. Treatment-related adverse events occurred in all patients, the most common being anemia (83.3%), neutrophil count decrease (76.7%), and platelet count decrease (73.3%), but no treatment-related deaths were reported. The trial also identified MAP2K7 as a resistance-associated biomarker, suggesting it as a potential therapeutic target for optimizing ivonescimab-based therapy.
10.1016/j.jhep.2026.06.033
Durvalumab with gemcitabine-based chemotherapy regimens in advanced biliary tract cancer: primary results from the phase IIIb TOURMALINE study.
J HEPATOL · Q1 JOURNAL - RANK #3/147TOP-TIER
The phase IIIb TOURMALINE trial (NCT05771480) prospectively evaluated first-line durvalumab (1500 mg IV; 60-minute first infusion, then 30-minute infusions) combined with investigator’s choice of seven gemcitabine-based chemotherapy regimens in advanced biliary tract cancer, with primary focus on Grade 3/4 possibly related adverse events (PRAEs) within 6 months. Among 142 participants (median age 68; 72.5% metastatic; median follow-up 11.63 months), 50.7% (95% CI 42.19–59.19) experienced Grade 3/4 PRAEs and there were no serious AEs of special interest with fatal outcomes. Median PFS was 7.39 months (95% CI 6.74–9.07), median OS 13.50 months (95% CI 11.24–20.53), and ORR 33.1% (95% CI 25.44–41.48). Safety was manageable across regimens, 30-minute infusions did not increase infusion reactions, and efficacy suggests clinical activity, supporting feasibility across diverse chemotherapy backbones.
10.1016/j.jhep.2026.06.025
Jun 29 – Jul 06, 2026
A randomized phase 2 study of combination atezolizumab and varlilumab (CDX-1127) with or without cobimetinib in previously-treated unresectable biliary tract cancer.
CLIN CANCER RES · Q1 JOURNAL - RANK #29/326TOP-TIER
This randomized, open-label phase 2 clinical trial evaluated whether combining the CD27 agonist varlilumab with PD-L1 inhibitor atezolizumab, with or without the MEK inhibitor cobimetinib, improves outcomes in patients with previously treated unresectable biliary tract cancer. Fifty-seven adults were randomized to atezolizumab + varlilumab + cobimetinib (CAV; n=29) or atezolizumab + varlilumab (AV; n=28); treatments were given in 28-day cycles, and co-primary endpoints were overall response rate (ORR) and progression-free survival (PFS), with CD8⁺ T-cell infiltration as a correlative endpoint. At interim analysis the study was stopped early owing to low efficacy: ORR was 0 % for CAV and 3.8 % for AV, median PFS was 2.40 vs 1.84 months respectively (hazard ratio 0.67, 95 % CI 0.38–1.18), and similar safety profiles were observed. The investigators conclude that although CAV increases intratumoral CD8⁺ T-cells, neither regimen provides meaningful clinical benefit in this setting.
10.1158/1078-0432.CCR-25-4893
Jun 22 – Jun 29, 2026
Prognostic markers, quality of life (QoL) and value of health (V-He) in advanced biliary cancers (ABC) treated with second-line active symptom control (ASC) alone or ASC with oxaliplatin-5-FU chemotherapy (ASC + FOLFOX) in the randomised phase III, multicentre, open-label ABC-06 clinical trial.
ESMO OPEN · Q1 JOURNAL - RANK #36/326
The ABC-06 randomised phase III trial evaluated second-line modified FOLFOX plus active symptom control (ASC) versus ASC alone for advanced biliary cancer. Researchers measured baseline tumour marker levels (CA19-9, CEA, CA125) and QoL scores (EORTC QLQ-C30, QLQ-BIL21, EQ-5D) in 162 patients. Elevated tumour markers predicted shorter overall survival, with multivariable-adjusted hazard ratios up to 1.70 (95% CI 1.13-2.56) for CA125, and stable CA19-9 was associated with numerically longer progression-free and overall survival. FOLFOX did not diminish QoL and seemed to preserve some domains, supporting its role as a standard second-line therapy.
10.1016/j.esmoop.2026.107777
Hepatic artery infusion pump chemotherapy for unresectable intrahepatic cholangiocarcinoma: Pooled individual patient-level analysis of four clinical trials.
J HEPATOL · Q1 JOURNAL - RANK #3/147TOP-TIER
This pooled analysis assessed long-term overall survival (OS) in patients with unresectable, liver-confined intrahepatic cholangiocarcinoma (iCCA) treated with hepatic artery infusion pump (HAIP) chemotherapy with floxuridine (FUDR). Individual patient data from four prospective phase II trials were pooled, totaling 142 patients, with or without systemic therapy; the primary outcome was OS. Key results included a partial response rate of 53% (73/139), a disease control rate of 96%, a pooled median OS of 26 months (95% CI: 22-30), and 3-year and 5-year OS rates of 28% and 15%, respectively. The authors conclude that these findings provide the most comprehensive long-term survival benchmarks for HAIP chemotherapy and support further prospective evaluation within modern multimodality treatment strategies.
10.1016/j.jhep.2026.06.022
Jun 08 – Jun 15, 2026
Extracellular matrix biomarkers of T-cell infiltration and tumor fibrosis predict response to nivolumab ± ipilimumab with SBRT in biliary tract cancer: insights from the CheckPAC trial.
BMC MED · Q1 JOURNAL - RANK #19/332
Objective: to evaluate the pharmacodynamic and predictive value of extracellular matrix–derived serum biomarkers in metastatic biliary tract cancer treated with SBRT plus immune checkpoint inhibitors. In the prospective CheckPAC trial (NCT02866383), 61 patients received SBRT with nivolumab (n=19) or nivolumab/ipilimumab (n=42); C4G, PRO-C3, PRO-C6, reC1M, C3M, and C4M were measured at baseline and day 60, and associations with overall survival (OS) and clinical benefit were analyzed using multivariable Cox and landmark methods. Higher baseline PRO-C3 and reC1M correlated with lack of clinical benefit and shorter OS (both p<0.05), PRO-C3 remained independently associated with OS after adjustment, and C4G increased by day 60 in all patients with clinical benefit (p<0.001), with increases linked to clinical benefit (p=0.007) and longer OS (p=0.0045). Patients with low PRO-C3 and increased C4G had the most favorable survival; these biomarkers may enable pharmacodynamic response tracking, warranting independent validation.
10.1186/s12916-026-04989-4
Jun 01 – Jun 08, 2026
Pemigatinib for Unresectable or Metastatic Cholangiocarcinoma With Fibroblast Growth Factor Receptor-2 Rearrangement: Results From the Phase 3 FIGHT-302 Trial.
J CLIN ONCOL · Q1 JOURNAL - RANK #6/326TOP-TIER
This phase 3, randomized, global trial (FIGHT-302) evaluated pemigatinib as first-line therapy in adults with advanced FGFR2-rearranged cholangiocarcinoma. One hundred sixty-seven patients were randomized to pemigatinib (13.5 mg daily) or chemotherapy (gemcitabine/cisplatin), with crossover allowed. Median progression-free survival was 8.3 vs 6.8 months (HR 0.58; p=0.0078), objective response rate 47% vs 15%, and median overall survival 24.4 vs 25.0 months. Pemigatinib demonstrated superior PFS versus chemotherapy with consistent safety, supporting its use as first-line targeted therapy for this population.
10.1200/JCO-26-00788
May 18 – May 25, 2026
First-Line Pembrolizumab Plus Chemotherapy for Advanced Biliary Tract Cancer: China Subgroup Analysis of the Randomized Phase 3 KEYNOTE-966 Study.
ADV THER · Q1 JOURNAL - RANK #82/352
This randomized, double-blind, phase 3 Chinese subgroup analysis of the global KEYNOTE-966 trial assessed first-line pembrolizumab 200 mg every 3 weeks plus gemcitabine/cisplatin versus placebo plus gemcitabine/cisplatin in 158 adults with previously untreated advanced biliary tract cancer. After a median follow-up of 20.5 months, median overall survival was 14.1 months for pembrolizumab and 9.9 months for placebo (HR 0.74, 95% CI 0.51-1.08); median progression-free survival was 5.6 versus 5.7 months (HR 0.83). Objective response rate reached 36.0% versus 28.9%, and median duration of response was 10.2 versus 5.7 months, respectively. Grade 3/4 treatment-related adverse events occurred in 71.6% and 70.7% of patients, with no treatment-related deaths; authors conclude pembrolizumab added to chemotherapy yields clinically meaningful survival improvement and acceptable safety in Chinese patients with advanced biliary tract cancer.
10.1007/s12325-026-03578-4