Topline phase 2a data showed a 48.9% six-month progression-free survival rate with intranasal NEO100 in previously treated IDH1-mutant glioma.
Intranasal NEO100 (purified perillyl alcohol) produced a 6-month progression-free survival (PFS) rate of 48.9% (95% CI, 26.3%-68.1%) in patients with recurrent or progressive grade III/IV IDH1-mutant glioma, meeting the primary end point of the phase 2a NEO100-01 trial (NCT02704858).1
The median overall survival (OS) was 26.09 months, with 12 deaths reported among 24 patients. OS rates were 86.7% (95% CI, 64.3%-95.5%) at 6 months and 60.9% (95% CI, 36.4%-78.4%) at 12 months.
“These results represent an important milestone for NeOnc and, more importantly, a source of hope for patients with recurrent high-grade glioma who currently have very limited treatment options,” Amir F. Heshmatpour, executive chairman, president, and chief executive officer of NeOnc Technologies, stated in a news release. “NEO100 met the study’s primary end point, demonstrated encouraging survival outcomes, and was administered intranasally by patients at home with no major toxicities reported. We believe these findings support the potential of our intranasal delivery platform to address one of the greatest challenges in treating brain cancer - the blood-brain barrier. Our priority now is to engage with the FDA and align on the most efficient path toward a registrational study.”
NEO100-01 was an open-label, single-arm phase 2a study of intranasal NEO100 in patients with recurrent or progressive grade III and grade IV IDH1-mutant glioma whose disease has recurred or progressed after radiation and temozolomide.1,2
NEO100 was self-administered intranasally by patients at home 4 times daily in 28-day cycles, a delivery route designed to reach the brain along the olfactory pathway while avoiding systemic cytotoxic exposure.¹ The study's primary end point was 6-month PFS rate per RANO 2.0 criteria, benchmarked against a pre-specified 20% rate representing the expected outcome with standard of care in this recurrent setting.
Evidence of durable disease control was observed across the cohort. Five of 24 patients remain on active treatment. One patient has remained progression-free for approximately 19 months, and a second patient achieved a partial response sustained for 114 days through the end of cycle 8. Investigators characterized the survival duration observed in this twice-treated, high-grade population as notable given the therapy's at-home administration and reported tolerability.
No major toxicities were reported across the cohort, and adverse effects (AEs) were predominantly low-grade. The tolerability profile was consistent with the phase 1 part of the study, which suggested that NEO100 was well tolerated at all dose levels, with no severe or dose-limiting toxicities.
“We believe that these results are a powerful validation of the science behind NEO100,” Thomas C. Chen, MD, PhD, founder, chief medical officer and chief scientific officer of NeOnc, added in the news release. “We set out to reach the brain directly, delivering therapy through the nose and along the olfactory pathway rather than forcing a drug past the blood-brain barrier, and what we are seeing in Phase 2a is what our biology predicted and what we saw in Phase 1, where every long-term survivor carried an IDH1 mutation. We believe that survival of this duration in twice-treated, high-grade disease, achieved with a therapy patients take at home and with no major toxicity, is the kind of result that justifies a much larger study.”
