Phase 1b NLG802 (indoximod prodrug; IDO/TDO-pathway immunometabolic modulator) + temozolomide in pediatric CNS tumors

This open-label Phase 1b, dose-escalation study evaluates escalating oral NLG802 (a prodrug of indoximod) given with oral temozolomide in children with relapsed/refractory primary brain and/or spinal tumors, aiming to define safety/tolerability and characterize pharmacokinetics alongside early anti-tumor signals. NLG802 is an oral prodrug of indoximod, an immunometabolic modulator intended to counter IDO/TDO-driven tryptophan-starvation signaling and restore antitumor T-cell activity; the program is sponsored by Lumos Pharma. Temozolomide is an oral DNA-alkylating chemotherapy used here as the combination backbone. The design is commercially interesting because it is a small-molecule “immunometabolic” add-on meant to make a widely used oral chemo backbone work better in a setting where treatment choices after progression are limited. For progressive pediatric CNS tumors, today’s care is typically maximal safe surgery when feasible plus radiation and multi-agent chemotherapy; at relapse, options often become palliative regimens, re-irradiation in select cases, and trial enrollment—so this tests a tolerable, all-oral combination intended to shift that balance.