Agios Pharmaceuticals Inc. reported positive data from its dose-escalation and expansion cohorts of the phase 1 trial for AG-120 in isocitrate dehydrogenase-1 mutant positive cholangiocarcinoma, a rare cancer of the bile ducts within and outside of the liver.
The ongoing phase 1 trial is assessing the safety and tolerability of the drug in advanced solid tumors, including glioma, cholangiocarcinoma and chondrosarcomas with an IDH1 mutation.
The drug was administered at 100 milligrams twice daily, and 300mg, 400mg, 500mg, 800mg and 1200mg once a day over a 28-day cycle. The median number of prior systemic therapies was two and 97% of patients received a prior gemcitabine-based chemotherapy regimen.
A confirmed partial response at 300mg and 500mg was experienced by four patients, while 41 patients experienced stable disease.
Landmark analyses of progression-free survival at six and 12 months were 38.5% and 20.7%, respectively. The median progression-free survival was 3.8 months.
The drug also inhibited plasma 2-hydroxyglutarate to within levels found in healthy volunteers, and also reduced 2-hydroxyglutarate in tumor biopsies, with levels in plasma and tumor biopsies showing a positive correlation.