Phase 1b NEO100 intranasal perillyl alcohol evaluates safety and drug exposure in selected pediatric brain tumors

NeOnc Technologies’ Phase 1b study evaluates intranasal NEO100, a highly purified formulation of perillyl alcohol, in patients aged 5-18 with protocol-defined pediatric high-grade gliomas and selected other malignant CNS tumors. The early-phase program examines tolerability, dose selection, and pharmacokinetics, including drug exposure in available tumor tissue when tissue can be obtained during clinically indicated procedures. Intranasal administration is intended to investigate a delivery route that may help address CNS exposure limitations.

Published preclinical work describes endoplasmic reticulum stress and apoptosis at higher concentrations and effects on calpain-1/RhoA signaling, tumor-cell migration, and invasion at lower concentrations. These are preclinical mechanistic observations, not a single clinically validated target or proof of efficacy in children. The immediate question is whether intranasal delivery produces acceptable exposure and tolerability in the enrolled pediatric population, supporting further evaluation of this CNS-directed route.