NeOnc's NEO100 hits 48.9% six-month PFS in mutant glioma trial

NeOnc Technologies' intranasal NEO100 met its primary endpoint in a Phase 2a trial, posting 48.9% six-month progression-free survival in recurrent IDH1-mutant high-grade glioma, more than double the 20% benchmark pre-specified for standard of care (p = 0.0047).

"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, said.

Median overall survival reached 26.09 months, with 86.7% of patients alive at six months, 60.9% at 12 months and 54.1% at 24 months. Five of 24 patients remain on active treatment, including one progression-free for about 19 months and another with a partial response sustained beyond 114 days. No major toxicities were reported.

The company plans to request a Type B meeting with the FDA to align on a registrational path in a setting with no approved targeted therapy. NTHI shares rose 4.37% to $4.54 on the readout, with volume running 124 times the average.

A delivery route that bypasses the blood-brain barrier

NEO100 is a patented, ultra-pure formulation of perillyl alcohol, a naturally occurring monoterpene found in citrus and peppermint oils. Delivered intranasally four times daily in 28-day cycles, the small lipophilic molecule travels along olfactory and trigeminal pathways to reach the brain directly, bypassing the blood-brain barrier that blocks most cancer drugs. Preclinical data suggest it may also transiently open the barrier, enabling entry of otherwise impermeable therapeutics.

The approach targets a population existing IDH-targeted therapies do not serve. Approved and late-stage agents such as vorasidenib were developed for front-line, lower-grade disease — typically Grade 2, non-enhancing tumors in patients who have not yet received radiation or chemotherapy. NEO100-01 enrolled the opposite group: patients with Grade III and Grade IV IDH1-mutant tumors that recurred after radiation and temozolomide, a setting with no approved targeted therapy. Roughly 90 percent of high-grade glioma patients recur within six to nine months of maximal therapy.

What the data mean for investors

The readout is a topline analysis, not the full dataset. The study enrolled 24 of 28 planned patients, and pre-specified subgroup, pharmacokinetic and quality-of-life analyses remain ongoing. The company expects to present the complete Phase 2a dataset at a future medical meeting.

Analysts have set price targets well above the current level. BTIG's Thomas Shrader holds a $15 target, Alliance Global Partners' Matthew Venezia $13 and Maxim Group's Jason McCarthy $20. Insider buying has also drawn attention: Heshmatpour has invested more than $500,000 through open-market purchases, with insider buying approaching $1 million over the past year. Institutions including Bank of America, State Street, Barclays and BlackRock appear among shareholders.

NeOnc holds Orphan Drug, Fast Track and Rare Pediatric Disease designations for NEO100 and has licensed a worldwide patent portfolio from the University of Southern California, with protections extending to 2038. The company's cash runway was not disclosed in the release. NEO212, its second candidate, is also in Phase II trials.

The FDA's response to the proposed registrational path will determine whether NEO100 can reach the market in a disease where patients currently face grim odds — only a quarter of newly diagnosed glioblastoma patients survive 24 months, and fewer than 10 percent survive five years.

This article is for informational purposes only and does not constitute investment advice.