NeOnc Technologies Holdings, Inc. (NASDAQ:NTHI) shares climbed 11.7% after the biotechnology company announced positive topline results from a Phase 2a study evaluating intranasal NEO100 in patients with recurrent IDH1-mutant high-grade glioma.
The trial achieved its primary endpoint, with six-month progression-free survival reaching 48.9%. That compared with a pre-specified standard-of-care benchmark of 20%, with the result achieving statistical significance at p = 0.0047.
Median overall survival reached 26.09 months, while 86.7% of participants were alive six months after starting treatment.
NEO100 trial meets progression-free survival endpoint
The Phase 2a study enrolled 24 patients with Grade III or Grade IV IDH1-mutant tumours that had returned or progressed following treatment with radiation and temozolomide.
Five participants remain on active treatment, including one patient who has remained progression-free for approximately 19 months.
The objective response rate was 8.3%, with two of the 24 patients achieving a response to treatment.
The progression-free survival results provided the main positive catalyst for NeOnc shares, given the limited treatment options currently available for patients with recurrent high-grade disease.
Intranasal treatment shows favourable safety profile
No major toxicities were reported among patients participating in the study, with adverse events predominantly classified as low grade.
NEO100 is administered intranasally, allowing patients to take the treatment at home four times per day in 28-day treatment cycles.
The combination of the efficacy findings and tolerability profile provides NeOnc with additional clinical evidence as it considers the next stage of development for the programme.
NeOnc plans FDA meeting on registrational pathway
Following the positive Phase 2a results, NeOnc intends to request a Type B meeting with the U.S. Food and Drug Administration.
The company plans to use the meeting to seek alignment with the regulator on a potential registrational development pathway for NEO100 in recurrent IDH1-mutant high-grade glioma.
There is currently no approved targeted therapy specifically for this treatment setting. Existing approved IDH-targeted therapies have been developed for lower-grade disease in the front-line setting rather than recurrent high-grade tumours.
Full Phase 2a data planned for future medical meeting
NeOnc expects to present the complete Phase 2a dataset at a future medical conference, including more detailed information about the treatment’s safety profile.
Additional pre-specified analyses are also continuing, including evaluations of patient subgroups, pharmacokinetics and quality-of-life outcomes.
The planned FDA discussions will represent the next significant step for the NEO100 programme as NeOnc explores a potential path towards a registrational study.