Patients with pre-treated, advanced biliary tract cancer (BTC) may benefit from treatment with nanvuranlat, efficacy data from a phase 2 study indicated. According to the authors, the safety profile was clean.

The current phase 2 study randomized 104 patients with advanced, refractory BTC with a NAT-2 non-rapid phenotype 2:1 to nanvuranlat or placebo.1 The primary endpoint was the progression-free survival (PFS) by blinded independent central review. Dr. Junji Furuse (Kanagawa Cancer Center, Japan) presented the first results of this primary outcome at ASCO-GI 2023.

Patients treated with nanvuranlat had a significantly improved PFS compared with patients treated with placebo (HR, 0.557; P=0.016) and this result was consistent across pre-defined subgroups. Furthermore, the disease-control rate appeared to be higher in the experimental arm than in the control arm (24.6% vs 11.4%).

As for safety, grade 3 or higher adverse event rates were generally low in both arms, not surpassing the 3.0% for a specific event in either study group. However, it seemed that grade 3 or higher cholangitis occurred more frequently in the nanvuranlat arm than in the placebo arm (12.9% vs 0%).

Data from the current phase 2 study support further investigation of nanvuranlat in patients with heavily pre-treated advanced BTC.

Copyright ©2023 Medicom Medical Publishers