NeOnc Technologies announced that it has finished enrolling all patients in its NEO100‑1 Phase 2a study of the intranasal therapy NEO100 for recurrent IDH1‑mutant high‑grade glioma. The company confirmed the milestone during an investor conference call on November 12, 2025, and issued a formal announcement the following day.
The enrollment completion means the trial can now move to the planned preliminary data readout in the second quarter of 2026. The readout will provide a full‑cohort analysis of efficacy and safety, a critical step toward regulatory submission and potential approval of NEO100 for this aggressive brain cancer.
NEO100’s intranasal delivery platform is designed to bypass the blood‑brain barrier, a major hurdle for CNS therapeutics. The company’s lead compound has already earned Orphan Drug Designation, Fast Track Status, and Rare Pediatric Disease Designation from the FDA, which can accelerate development and grant market exclusivity.
The announcement follows exceptionally positive interim data from a 24‑patient cohort reported on November 12. Five patients (21%) achieved a radiographic response, far exceeding the <8% response rate seen with salvage therapies. Forty‑four percent of patients reached six‑month progression‑free survival, surpassing historical benchmarks of 21‑31% for this disease. Eight patients (33%) remained alive 18 months after starting NEO100, and no significant toxicity was observed with prolonged intranasal use.
Management highlighted the milestone as a “defining inflection point.” Executive Chairman and CEO Amir Heshmatpour said the enrollment success, coupled with the strong interim data, “propels us into the next stage of value creation” and underscores the potential for durable survival benefits with a non‑invasive delivery method.
Financially, NeOnc remains pre‑commercial with negative margins and a high cash burn. A $50 million partnership with Quazar Investment was expected to close by October 23, 2025, providing critical funding for continued development. The company’s cash position and ability to sustain operations will depend on securing additional financing or achieving regulatory milestones that unlock revenue streams.
On the day of the investor call, the company’s stock gained 5.48%, reflecting investor optimism around the clinical data and the upcoming data readout. The market reaction was driven by the 21% radiographic response rate, the 44% PFS‑6 figure, and the absence of significant toxicity, all of which exceeded historical benchmarks for recurrent IDH1‑mutant glioma.
NeOnc’s progress in the NEO100‑1 trial positions it as a potential leader in the niche of intranasal CNS therapies, but the company’s financial challenges and reliance on external funding remain key risks to its long‑term viability.
The content on BeyondSPX is for informational purposes only and should not be construed as financial or investment advice. We are not financial advisors. Consult with a qualified professional before making any investment decisions. Any actions you take based on information from this site are solely at your own risk.