NEO100 intranasal glioma therapy

NeOnc Technologies did not announce a new approval or late-stage readout on September 1. The item circulating on social media points back to Phase 2a data the company released on August 12 for intranasal NEO100 in recurrent IDH1-mutant high-grade glioma. NeOnc said the study met its primary endpoint, and the company described the drug as a purified perillyl alcohol formulation delivered through the nose rather than by intravenous infusion. (neonc.com) ClinicalTrials.gov lists the study as NCT02704858, a Phase 1/2a trial in recurrent or progressive grade III or IV IDH1-mutated glioma. ### What exactly is NEO100, and who is testing it? NeOnc Technologies, a California-based clinical-stage biotechnology company, is developing NEO100 as an intranasal formulation of purified perillyl alcohol for central nervous system cancers. (neonc.com) The company’s August 12 release said the Phase 2a portion of NEO100-01 enrolled patients with recurrent or progressive grade III and grade IV IDH1-mutant glioma. ClinicalTrials.gov identifies NEO100-01 as an open-label, multi-site Phase 1/2a study for patients with radiographically confirmed progression of grade IV glioma or recurrent primary or secondary grade IV glioma, as well as grade III disease with IDH1 mutation. The trial record says the study is designed to assess safety, pharmacokinetics and efficacy of repeated-dose NEO100. (neonc.com) ### Why are people talking about the intranasal route? NeOnc said the drug was administered intranasally by patients at home, and Chief Executive Amir F. Heshmatpour said the findings support the company’s intranasal delivery platform in brain cancer. In the same statement, he said the platform is aimed at addressing the blood-brain barrier, which can limit how much drug reaches tumor tissue when therapies are given systemically. (neonc.com) The company’s trial materials also frame intranasal delivery as the core feature of the program. NeOnc’s study page says the approach was investigated for recurrent glioblastoma because recurrent disease responds poorly to repeat surgery, re-irradiation and additional chemotherapy, while prognosis remains poor. (clinicaltrials.gov) ### What did the Phase 2a study show? The August 12 company release said six-month progression-free survival was 48.9% by RANO 2.0 criteria, compared with a pre-specified 20% benchmark for standard of care, with a reported p-value of 0.0047. NeOnc also reported median overall survival of 26.09 months and said 86.7% of patients were alive at six months. OncLive, citing the same topline release, reported that the single-arm Phase 2a study included 24 patients and that five remained on treatment, including one patient with a partial response ongoing beyond 114 days. (neonc.com) The company said no major toxicities were reported and that adverse events were predominantly low grade. (neonc.com) ### Does this mean NEO100 is an approved treatment now? The answer is no. ClinicalTrials.gov still lists NEO100-01 as a clinical study, and NeOnc’s own language describes the August 12 disclosure as positive topline Phase 2a results rather than a regulatory clearance. The current data also apply to a narrower population than the broader phrase “recurrent high-grade glioma” can imply. (neonc.com) NeOnc specified that the Phase 2a results were in recurrent or progressive grade III and grade IV glioma with IDH1 mutation, not all glioma patients. ### What happens next for the program? NeOnc said on August 12 that it plans to request a Type B meeting with the U.S. Food and Drug Administration to discuss a registrational path for NEO100 in recurrent IDH1-mutant high-grade glioma. (onclive.com) The company repeated that point in investor-relations materials tied to the Phase 2a readout. (clinicaltrials.gov) The next concrete milestone to watch is that FDA meeting request and any subsequent company disclosure on trial design, timing or a registrational study. ClinicalTrials.gov continues to list NCT02704858 as the underlying study record for the program. (clinicaltrials.gov) (neonc.com)