NEO100-01

Aug. 12, 2026: NeOnc Technologies Holdings, Inc. (NASDAQ: NTHI) reported positive topline Phase 2a results from NEO100-01, an open-label study of intranasal NEO100 (purified perillyl alcohol) in recurrent or progressive Grade III/IV IDH1-mutant glioma after prior radiation and temozolomide. The primary endpoint was met: six-month progression-free survival was 48.9% (95% CI: 26.3-68.1) versus a pre-specified 20% benchmark for standard of care (p=0.0047).

Secondary results included median overall survival of 26.09 months; overall survival was 86.7% at 6 months, 60.9% at 12 months, and 54.1% at 24 months. Objective response rate was 8.3% (2/24) by RANO 2.0. Five of 24 patients remained on treatment; one patient was progression-free for about 19 months, and one partial response remained ongoing beyond 114 days.

The company said NEO100 was well tolerated, with no major toxicities and predominantly low-grade adverse events. The Phase 2a cohort enrolled 24 of a planned 28 patients at 1,152 mg/day, self-administered intranasally four times daily.

Next steps: NeOnc plans to request a Type B FDA meeting to discuss a registrational path in recurrent IDH1-mutant high-grade glioma. Additional subgroup, pharmacokinetic, and quality-of-life analyses are ongoing, and full Phase 2a data are planned for a future medical meeting.