Tovecimig (CTX-009)

Tovecimig has no approved indication. The indication under development is unresectable advanced, metastatic or recurrent biliary tract cancer, including intrahepatic cholangiocarcinoma, extrahepatic cholangiocarcinoma, gallbladder cancer and ampullary carcinoma, in adults who have progressed after one prior gemcitabine- and platinum-containing chemotherapy regimen. The FDA Fast Track designation covers CTX-009 in combination with paclitaxel for previously treated metastatic or locally advanced biliary tract cancer, and the April 2026 orphan drug designation covers biliary tract cancer. COMPANION-002 excludes participants who are eligible for an approved molecularly targeted therapy on a labeled regimen after first-line chemotherapy, so the target population is the subgroup without an actionable alteration; the sponsor states that all targeted therapies together are appropriate for approximately 10% to 15% of patients with biliary tract cancer and estimates more than 15,000 patients per year in the United States as potentially eligible. No biomarker selection for DLL4 or VEGF-A expression is required for enrollment in COMPANION-002. Tovecimig is also being studied in first-line biliary tract cancer, colorectal cancer, glioblastoma, and a planned basket study of DLL4-positive tumors.