Neonc Technologies Holdings, Inc. Q2 FY2026 earnings call

• Pipeline and Technology Overview

  • The company has two lead product candidates, NEO100 and NEO212, with four ongoing clinical trials, all using intranasal delivery that bypasses the blood-brain barrier non-invasively via the first and fifth cranial nerves.
  • NEO100 is an ultra-pure para-alcohol that acts through multiple anti-cancer mechanisms: inducing endoplasmic reticulum stress to shut down tumor protein synthesis, inhibiting sodium potassium ATPase, inhibiting RAS activity, and blocking cancer cell cycle proliferation, which has demonstrated increased survival in in vivo mouse models.
  • NEO212 is a new chemical entity formed by conjugating NEO100 to standard-of-care temozolomide via a carbamide bond, creating a more stable compound.
  • NEO100 indications in active trials include: (1) NEO100-01: recurrent IDH1-mutant grade 3/4 astrocytoma (Phase 2A completed); (2) NEO100-02: refractory/ malignant meningioma (Phase 2); (3) NEO100-03: pediatric brain cancer (Phase 2); (4) NEO100 plus temozolomide for all brain tumors (Phase 2, Phase 1 completed).

• NEO100-01 Phase 2A Clinical Results

  • The trial met its primary endpoint of 6-month progression-free survival (PFS6), hitting 48.9% against a pre-specified 20% historical benchmark (p=0.0047, statistically significant).
  • Median overall survival (OS) from recurrence was 26.09 months, compared to the 6-9 month historical benchmark for current salvage therapy in this patient population. 86.7% of patients were alive at 6 months, 60.9% at 12 months, and over half of patients remained alive at 24 months.
  • 66.7% of trial patients achieved disease control (combining partial response and stable disease). Across combined Phase 1 and Phase 2 data of 29 patients, 55% were long-term survivors (OS > 1 year post-recurrence), with a median OS of 2.2 years (range 1.2 to over 8 years post-recurrence). The drug demonstrated a favorable safety and tolerability profile.
  • Results fill an unmet need that competing trials have not addressed: the INDIGO trial only demonstrated efficacy in lower-grade IDH1-mutant tumors, while the STELR trial failed to show benefit in grade 4 recurrent IDH1-mutant disease, the population that made up the majority of NEO100's trial.

• Recent Operational Progress

  • The company has obtained approval to run all four of its trials in Abu Dhabi, and expects approval to run trials in Israel shortly. NEO100 is already offered via compassionate use for patients that do not qualify for trials, with the highest volume of compassionate use in the pediatric brain cancer population.

Neon Technologies is a clinical-stage biotech company focused on developing novel brain cancer treatments. It does not have any approved commercial products, so no revenue or financial segment performance is reported in this transcript.

• The company plans to hold an upcoming Type B meeting with the FDA to seek alignment on a randomized Phase 3 registrational trial designed to support accelerated approval of NEO100 for recurrent IDH1-mutant high-grade glioma, against the active comparator lomustine.
  • The proposed Phase 3 trial will use median overall survival as the primary endpoint, with a pre-specified interim analysis based on progression-free survival that may support accelerated approval.
  • No financial guidance is provided, as the company remains in clinical development with no commercial products.

• Forward-looking statements related to trial results and regulatory approval are subject to risks: results from preclinical and early-phase clinical trials are not predictive of future late-phase trial results, and interim data may change after full data collection, auditing, and verification.
  • All of the company's product candidates are in clinical development, have not received regulatory approval for commercial sale, and may never gain approval or become commercially viable.
  • The Phase 2A NEO100 trial was a single-arm trial that compared results to a historical benchmark rather than a contemporary control group, which may affect regulatory evaluation of the efficacy signal.
  • IDH1-mutant recurrent high-grade glioma is an extremely aggressive, hard-to-treat indication with no proven effective approved therapies, and there is no guarantee that the positive Phase 2A signal will replicate in a larger Phase 3 trial.

Q: How do you interpret the 48.9% PFS6 result (double the 20% benchmark) for NEO100, and what does it mean for this patient population? / A: Two independent neuro-oncology key opinion leaders noted that this is one of the most promising signals for recurrent IDH1-mutant high-grade glioma seen in the last decade. Current approved IDH1 inhibitors do not work for higher-risk enhancing recurrent disease, so a positive result would represent a paradigm shift in treatment for this underserved patient group.

Q: Why do some patients without a radiographic response still see extended overall survival? / A: NEO100 acts initially as a cytostatic agent that slows tumor growth, before transitioning to cytotoxic activity that kills cancer cells, so survival benefit can emerge before a visible tumor shrinkage. KOLs added that this is not unprecedented (seen with immunotherapies), and turning this cancer into a chronic controllable disease (similar to insulin for diabetes) would still be a major win for patients.

Q: Is the rapid death after recurrence of this disease recognized by regulators, and what endpoints will be used for the Phase 3 trial? / A: The FDA is already aware of the clinical context of this disease, and accepts that response rate is not the most critical endpoint for this indication. For the registrational Phase 3, median overall survival will be the required primary endpoint, and progression-free survival can be used for an interim analysis to support accelerated approval.

Q: Is NEO100's RAS inhibition activity specific to certain RAS mutation types? / A: Preclinical testing confirms NEO100 has inhibitory activity against all tested forms of RAS, including both KRAS and HRAS, making it a pan-RAS inhibitor.