NeOnc Technologies announced that the Phase 2a NEO100-01 study of NEO100 intranasal perillyl alcohol in patients with Grade 3 and Grade 4 IDH1-mutant gliomas met its primary endpoint, achieving a 48.9% 6-month progression-free survival rate. That rate exceeded the rate expected for standard of care, which was 20%. The company says that it now plans to request a Type B meeting with the FDA.
The company announced the initiation of the open-label NEO100-01 trial in 2016. The study initially enrolled patients with grade 4 gliomas only; in 2024, the FDA okayed the addition of patients with recurrent grade 3 astrocytoma to the trial. In total, 24 patients were enrolled and used NEO100 via nasal delivery at home 4 times a day. Of those, 5 patients are still receiving treatment, NeOnc says.
According to the company, in addition to meeting the primary endpoint, “secondary survival endpoints were supportive,” including an overall survival rate of 86.7% at 6 months; almost 61% at 12 months and about 54% at 24 months. Median overall survival was just over 26 months. NeOnc notes that NEO100 was well tolerated; that “adverse events to date have been predominantly low-grade”; and that some patients have been using NEO100 on a daily basis for more than a year with no major toxicity.
NeOnc Technologies Executive Chairman, President, and CEO Amir F. Heshmatpour commented, “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. NEO100 met the study’s primary endpoint, 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.”
Chief Medical Officer and Chief Scientific Officer Thomas C. Chen said, “We believe that these results are a powerful validation of the science behind NEO100. 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.”
Chief Clinical Officer Josh Neman added, “In recurrent high-grade glioma, the outcome that matters is how long a patient can hold the disease at bay and still live their life. Five of our 24 patients remain on therapy, one progression-free approaching 19 months and another in an ongoing response approaching four months, and they are taking this treatment at home rather than in an infusion chair. Durability and tolerability together are rare at recurrence, and we believe that combination is what these data point to. We look forward to discussing the findings with the FDA.”
Read the NeOnc Technologies press release
