The Company plans to seek FDA feedback on its NEO212 clinical development program, including an update to its proposed patient population, trial design, endpoints, dose selection and evidence needed to support a future marketing application. The meeting provides an opportunity for discussion; its scheduling does not establish FDA agreement on a trial design, an accelerated approval pathway or eventual approval of NEO212.
In September 2025, FDA authorized the Company to proceed with the Phase 2a and Phase 2b portions of NEO212-01.
This meeting follows completion of the Phase 1 dose-escalation portion of the NEO212-01 Phase 1/2 clinical trial. NeOnc previously reported that dose escalation was discontinued following a dose-limiting toxicity at 810 mg, administered once daily on Days 1 through 5 of a 28-day cycle, and that 610 mg was selected as the recommended Phase 2 dose.
NeOnc also reported encouraging clinical activity observed during the Phase 1 portion of the trial. In one patient with recurrent IDH1 wild-type, MGMT-methylated glioblastoma (GBM), treatment with NEO212 resulted in a partial response, including an approximately 60% reduction in tumor size, followed by prolonged disease control lasting 21 treatment months. This duration of disease control is notable in the setting of recurrent GBM, where outcomes following recurrence have historically been limited.
In a second patient with lung cancer metastatic to the brain who had previously received and progressed following multiple lines of therapy, including immune checkpoint inhibitors, treatment with NEO212 was associated with stable disease lasting approximately 16 months. Patients with brain metastases from lung cancer who have progressed following multiple prior systemic therapies represent a population with substantial unmet medical need and historically limited treatment options.
While these individual patient outcomes do not establish efficacy and require confirmation in larger studies, the durability of responses observed during Phase 1, together with the established recommended Phase 2 dose, provides the clinical rationale for further evaluation of NEO212 in the Phase 2 portions of the NEO212-01 trial. Building on these findings, NeOnc intends to discuss its potential registrational development strategy and to seek FDA feedback on whether a proposed trial design could support a potential accelerated approval pathway.