NEO100-01 Meets Primary End Point, Supporting Further Development in IDH1-Mutant Glioma

Clinical Summary:

• Design/Population: The multicenter, open-label NEO100-01 trial assessed intranasal NEO100 (purified perillyl alcohol) in patients with recurrent or progressive grade III/IV IDH1-mutant high-grade glioma previously treated with radiation and temozolomide. 
• Key Outcomes: NEO100 met the primary end point, with a 6-month progression-free survival rate of 48.9% compared with a prespecified 20% benchmark (P = .0047). Median overall survival was 26.09 months, and the objective response rate was 8.3%. 
• Clinical Relevance: These findings support further evaluation of intranasal NEO100 as a potential treatment for patients with recurrent IDH1-mutant high-grade glioma, a population with limited approved treatment options.

Topline results from the phase 2a NEO100-01 study showed that intranasal NEO100 met its primary end point in patients with recurrent or progressive IDH1-mutant high-grade glioma, achieving a 6-month progression-free survival (PFS) rate of 48.9% compared with a prespecified 20% benchmark for standard of care.

NEO100-01 is a multicenter, open-label, phase 1/2a study evaluating intranasal NEO100, a purified formulation of perillyl alcohol, in patients with radiographically confirmed recurrent or progressive grade III astrocytoma or grade IV glioma harboring an IDH1 mutation. All patients had previously experienced treatment failure following radiation or combined temozolomide and radiation. 

The phase 2a portion of this study enrolled 24 of a planned 28 patients who received 1152 mg per day of NEO100, self-administered intranasally 4 times daily in 28-day cycles until disease progression, death, or withdrawal. The primary end point was PFS at 6 months. Key secondary end points included objective response rate (ORR), overall survival (OS), and safety. Response and progression were assessed according to RANO 2.0 criteria by independent central review.

The Kaplan-Meier–estimated 6-month PFS rate was 48.9%, significantly exceeding the prespecified 20% benchmark (P = .0047). Median OS was 26.09 months, with estimated OS rates of 86.7% at 6 months, 60.9% at 12 months, and 54.1% at 24 months.

Five of the 24 patients remained on active treatment at the time of analysis. One patient remained progression-free for approximately 19 months, while another achieved a partial response that had continued for 114 days through the end of cycle 8 and remained ongoing. The ORR was 8.3%, corresponding to 2 responses among 24 patients. 

NEO100 was reported to be generally well tolerated, with no major toxicities observed across the cohort and adverse events predominantly low grade. Detailed safety findings have not been reported and are expected to be presented with the full phase 2a dataset at a future medical meeting. 

NeOnc plans to request a Type B meeting with the FDA to discuss a potential registrational development pathway for NEO100 in recurrent IDH1-mutant high-grade glioma. Additional prespecified analyses, including outcomes according to grade III vs grade IV disease, pharmacokinetics, and quality-of-life measures are ongoing. 

Source:

NeOnc Technologies. NeOnc Technologies reports positive topline phase 2a results for intranasal NEO100 in recurrent IDH1-mutant high-grade glioma. Accessed on August 12, 2026. https://investors.neonc.com/news-releases/news-release-details/neonc-technologies-reports-positive-topline-phase-2a-results