Phase 3 D07001 oral gemcitabine (deoxycytidine antimetabolite) + capecitabine in advanced BTC

In this Phase 3, randomized, double-blind, placebo-controlled, multicenter study (Inno-GO-07), InnoPharmax is testing whether adding D07001-softgel capsules—an oral gemcitabine formulation (a deoxycytidine analog antimetabolite that blocks DNA synthesis via DNA chain termination and ribonucleotide reductase inhibition)—to capecitabine improves overall survival versus placebo + capecitabine in unresectable or metastatic biliary tract cancer (cholangiocarcinoma, gallbladder cancer, or ampullary carcinoma). Patients are randomized 1:1:1 to D07001 60 mg + capecitabine, D07001 100 mg + capecitabine, or placebo + capecitabine, continuing until progression, death, withdrawal, or completion of 12 cycles. The scientific/commercial angle is a registrational attempt to “recycle” gemcitabine in a metronomic oral formulation after prior IV gemcitabine/platinum and subsequent fluoropyrimidine-based regimens, potentially offering a convenient all-oral regimen. Eligible patients must have progressed on gemcitabine + cisplatin (or substituted platinum) and also progressed on/refused FOLFOX or progressed on irinotecan + fluorouracil-based therapy, with flexibility for patients who received appropriate targeted therapies first when actionable alterations are present (eg, FGFR2 fusions are a minority in intrahepatic cholangiocarcinoma, while MSI-high is rare in BTC). After gemcitabine/platinum ± durvalumab, today’s care is molecularly targeted therapy when eligible (eg, FGFR/IDH1/HER2/BRAF/NTRK/RET/KRAS G12C subsets), otherwise later-line systemic therapy options often include FOLFOX and/or irinotecan/5-FU regimens, with modest outcomes—so this trial is trying to establish an OS-improving, broadly applicable add-on.