NeOnc reports positive Phase 2a data for NEO100 in recurrent IDH1-mutant glioma

NeOnc Technologies Holdings, Inc. (Nasdaq: NTHI) announced on August 12, 2026 that the study met its primary endpoint.¹ In the Phase 2a portion of NEO100-01, an open-label study of intranasal NEO100 in patients with recurrent or progressive Grade III and Grade IV IDH1-mutant glioma, progression-free survival at six months came in at 48.9 percent, with a 95 percent confidence interval of 26.3 to 68.1, using RANO 2.0 criteria and Kaplan-Meier estimation, versus a 20 percent benchmark built into the study as the expected rate for standard of care, yielding a p-value of 0.0047.¹

On survival, median overall survival was 26.09 months, with 12 deaths among the 24 patients, and overall survival stood at 86.7 percent at six months, 60.9 percent at 12 months, and 54.1 percent at 24 months.¹

Regarding disease control, five of the 24 patients remain on active treatment, including one patient progression-free for about 19 months and a second who reached a partial response lasting 114 days through Cycle 8 and remains on therapy; the objective response rate was 8.3 percent, or two of 24 patients, by RANO 2.0.¹

On safety, no major toxicities were reported across the cohort, and adverse events so far have mostly been low-grade.¹

The trial enrolled patients whose disease had progressed after prior treatment: all patients had previously failed radiation or combined temozolomide and radiation, and the Phase 2a portion enrolled 24 of a planned 28 patients at the recommended Phase 2 dose of 1,152 mg per day, self-administered intranasally four times daily in 28-day cycles until progression, death, or withdrawal.¹

Looking ahead, the company said it will pursue a "Type B" meeting with regulators to discuss next steps toward a registrational study design, and noted that further analyses covering the Grade III versus Grade IV subgroup, pharmacokinetics, and quality of life are still underway and will be released later, per the filing.