NeOnc Technologies Reports Updated Clinical Results

Rhea-AI Summary

NeOnc Technologies (Nasdaq: NTHI) reported updated clinical results for intranasal NEO100 in recurrent WHO Grade III/IV IDH1-mutant astrocytoma on Dec 15, 2025. The expanded cohort totals 25 patients. Key outcomes: radiographic remission in 24% (6/25), PFS-6 of 44%, and 36% (9/25) alive ≥18 months after treatment start. The company reports no significant toxicity with chronic intranasal dosing. Responses were assessed by RANO criteria and include 1 compassionate-use case, 5 Phase 1, and 18 Phase 2a patients. Management describes the dataset as an important value-inflection point and highlights reproducibility and durability of responses.

Positive

• Radiographic response 24% (6 of 25 patients)
• PFS-6 44% of patients
• Long-term survival 36% (9 of 25) ≥18 months
• No significant toxicity with prolonged intranasal dosing

Negative

• Small cohort of 25 patients limits statistical power
• Heterogeneous dataset combining compassionate-use, Phase 1, and Phase 2a patients

Key Figures

Radiographic remission rate 24% (6 of 25 patients) Recurrent Grade III/IV IDH1-mutant astrocytoma treated with intranasal NEO100

Historical salvage response <8% response rate Typical outcome with salvage therapies in recurrent high-grade gliomas

PFS-6 rate 44% of patients Six-month progression-free survival (PFS-6) with NEO100

Historical PFS-6 benchmark 21–31% IDH1-mutant recurrent high-grade gliomas historical PFS‑6 range

Long-term survival 36% (9 of 25 patients) Patients alive ≥18 months post NEO100 initiation

Cohort size 25 patients Combined Phase 1/2a and compassionate-use NEO100 analysis set

Compassionate-use cases 1 compassionate-use case Included alongside Phase 1 (5) and Phase 2a (18) patients

Toxicity profile No significant toxicity observed Prolonged, chronic intranasal NEO100 administration

Market Reality Check

$7.83 Last Close

Volume Volume 43,939 vs 20-day average 67,988, indicating activity below recent norms. low

Technical Shares at $10.21 are trading above the 200-day MA of $7.70 before this update.

Peers on Argus 2 Up

Pre-news, NTHI was up 8.96% while close peers showed mixed moves: ELTX -2.09%, IPHA -1.59%, CCCC -3.14%, KYTX +2.57%, TRDA +2.31%. Momentum scanner flagged KYTX and CCCC with upside moves, but overall action does not indicate a broad sector-wide move.

Common Catalyst Both NTHI and KYTX had clinical trial-related headlines, suggesting trial data was a key driver for select names rather than the entire biotech group.

Historical Context

Date	Event	Sentiment	Move	Catalyst
Dec 01	Preclinical AI study	Positive	-4.7%	AI-driven preclinical data showed ultrasound enhances NEO100 antitumor potency.
Nov 14	Earnings & pipeline	Positive	-12.3%	Q3 update with $50M partnership, NIH grants, and FDA Phase II go-ahead for NEO212.
Nov 13	Trial enrollment	Positive	-24.1%	Completion of enrollment in pivotal NEO100 Phase 2a with data expected in Q2 2026.
Nov 12	Clinical data update	Positive	+3.6%	Updated NEO100 data showed 21% response, 44% PFS‑6, and 33% ≥18‑month survival.
Nov 07	Investor call notice	Neutral	+2.7%	Announcement of investor call to review NEO100 clinical and compassionate-use data.

Pattern Detected

Clinical and strategic updates have often produced sharp but inconsistent reactions, with several positive clinical milestones met by negative or muted next-day price moves.

Recent Company History

Over the last eight months, NeOnc has repeatedly advanced its CNS oncology pipeline. On Apr 8 and Apr 14, it highlighted progress in NEO212 and NEO100 trials, followed by a $50M regional expansion term sheet on Jul 8. Subsequent FDA Phase II authorization for NEO212 on Sep 10 and strong NEO100 data on Nov 12 reinforced a clinically driven story. The current NEO100 update extends those efficacy and durability signals in the same recurrent IDH1‑mutant glioma setting.

Market Pulse Summary

This announcement extends the NEO100 dataset to 25 patients with recurrent IDH1‑mutant high‑grade gliomas, showing 24% radiographic remission, 44% PFS‑6, and 36% survival beyond 18 months, without significant toxicity. Compared with prior November results, the added responder strengthens the durability signal in this difficult setting. Investors may weigh these outcomes against the company’s disclosed losses and going‑concern language, watching upcoming NEO100 readouts and funding steps closely.

Key Terms

idh1-mutant astrocytoma medical

"recurrent Grade III/IV IDH1-mutant astrocytoma treated with intranasal NEO100"

A brain tumor that arises from supportive glial cells and is defined by a specific change in the IDH1 gene; that genetic change alters the tumor’s behavior, prognosis, and how it responds to treatments. Investors pay attention because this clear biological subgroup can make clinical trials, drug approvals and market opportunities more predictable—like knowing a building’s blueprint before designing a renovation—so therapies targeted to this mutation can command distinct regulatory paths and commercial value.

intranasal medical

"patients received intranasal NEO100, and results were assessed"

Administration or delivery of a drug or vaccine through the nose, typically via a spray or drops that are absorbed by the nasal tissues. For investors, intranasal products can matter because they often offer easier, needle-free use, faster onset of effect, and potential cost or compliance advantages that can widen a product’s market and affect regulatory review, manufacturing complexity, and commercial prospects — similar to how a convenient app can outsell a clunky desktop program.

progression-free survival (pfs-6) medical

"44% of patients achieved six-month progression-free survival (PFS-6)"

Progression-free survival at six months (PFS-6) is the share of patients in a clinical trial who are alive and whose disease has not worsened six months after starting treatment. For investors, PFS-6 is a simple, early snapshot of a drug’s ability to keep a condition stable—like checking whether a boat stays afloat after a rough month—and can signal whether a therapy is likely to move forward in development, attract regulatory attention, or gain market interest.

response assessment in neuro-oncology (rano) medical

"results were assessed using Response Assessment in Neuro-Oncology (RANO) criteria"

Response Assessment in Neuro-Oncology (RANO) is a standardized set of rules researchers and doctors use to judge whether brain tumors are shrinking, stable, or growing after treatment, based on scans and clinical signs. It matters to investors because RANO outcomes are a common benchmark in clinical trials that influence regulatory decisions, perceived drug effectiveness, and commercial prospects — think of it as the scorecard investors use to compare and value cancer therapies.

t2-flair medical

"confirmed by contrast-enhanced, T2-FLAIR, and perfusion MRI"

T2‑FLAIR is a specific MRI imaging setting that dims the bright signal from normal fluid so areas of swelling, scarring or lesions in the brain stand out more clearly. Investors should care because clinical trial results, diagnostic claims and regulatory approvals for drugs or devices often rely on T2‑FLAIR images to show whether a treatment changes disease markers — like using a photo filter that reveals problems otherwise hidden — which can affect commercial prospects and valuation.

perfusion mri medical

"confirmed by contrast-enhanced, T2-FLAIR, and perfusion MRI"

A perfusion MRI is a medical imaging test that maps how blood moves through tissues by taking a series of rapid pictures, similar to watching traffic flow through a neighborhood to see where congestion or gaps occur. For investors, perfusion MRI matters because it can drive demand for imaging equipment, influence the approval and success of drugs and medical devices that affect blood flow, and affect reimbursement and diagnostic market size.

compassionate-use regulatory

"Phase 1/2a and compassionate-use experience evaluating intranasal NEO100"

Compassionate-use is a regulatory pathway that lets seriously ill patients access an experimental drug or medical device before it receives full approval. For investors, it matters because such access can provide real-world safety and effectiveness information, generate early demand or publicity, and change expectations for a product’s commercial prospects, while not guaranteeing eventual regulatory approval or routine market sales.

cns-penetrant medical

"first-in-class, CNS-penetrant metabolic therapy capable of inducing sustained"

A CNS-penetrant drug or molecule can reach and act inside the brain and spinal cord by crossing the blood–brain barrier, the body’s protective filter around the central nervous system. For investors, CNS penetration signals whether a therapy might treat neurological or psychiatric conditions, which affects potential market size, development difficulty, safety risks and regulatory scrutiny — think of it as whether a delivery can pass a guarded gate to reach a secure building.

AI-generated analysis. Not financial advice.

Expanded Clinical Experience Demonstrates Additional Long-Term Survival and Radiographic Remission in Recurrent Grade III/IV IDH1-Mutant Astrocytoma Treated with Intranasal NEO100

CALABASAS, Calif., Dec. 15, 2025 (GLOBE NEWSWIRE) -- NeOnc Technologies Holdings, Inc. (Nasdaq: NTHI) (“NeOnc” or the “Company”), a multi-Phase 2 clinical-stage biopharmaceutical company developing novel therapies for central nervous system (CNS) cancers, today announced updated clinical results from its ongoing Phase 1/2a and compassionate-use experience evaluating intranasal NEO100 in patients with recurrent WHO Grade III/IV IDH1-mutant astrocytoma.

Since the Company’s prior announcement and 8K event on November 12, 2025, an additional patient has achieved both durable long-term survival and radiographic remission, further strengthening the clinical signal observed with intranasal NEO100. With this update, the expanded clinical cohort now includes 25 patients, reinforcing the reproducibility and durability of treatment benefit.

Updated results demonstrate that treatment with intranasally delivered NEO100 has resulted in significant radiographic remission in 6 of 25 patients (24%), representing 3X increase over the 8% response rates typically reported with salvage therapies in recurrent high-grade gliomas.

Additionally, 44% of patients achieved six-month progression-free survival (PFS-6), exceeding historical benchmarks of 21–31% for IDH1-mutant recurrent high-grade gliomas. Importantly, 9 of 25 patients (36%) remain alive ≥18 months following initiation of NEO100, providing further evidence of meaningful long-term survival in this heavily pretreated population.

No significant toxicity has been observed with intranasal administration of NEO100, even with prolonged and chronic dosing.

Amir F. Heshmatpour, Executive Chairman, President & CEO of NeOnc Technologies Holdings, Inc., stated: “The addition of another patient achieving both long-term survival and radiographic remission further validates what we believe is a highly compelling and differentiated therapeutic signal. With radiographic responses now 300% higher than the rate historically reported with salvage therapies, these updated data reinforce our conviction that NEO100 may represent a meaningful advance for patients with recurrent IDH1-mutant high-grade gliomas—patients who have historically had few options. As we move toward a complete data readout, the consistency and durability of these responses continue to exceed expectations.”

Mr. Heshmatpour added: “We view this expanding dataset as a critical value-inflection point for NeOnc and for the neuro-oncology field, suggesting a potential shift away from purely palliative approaches toward durable disease control.”

Founder, Vice-Chairman, and Chief Medical Officer Dr. Thomas Chen commented: “With each additional durable responder, the data increasingly support the hypothesis that intranasal NEO100 may be a first-in-class, CNS-penetrant metabolic therapy capable of inducing sustained radiographic remission and multi-year survival in recurrent IDH1-mutant gliomas.”

Dr. Henry Friedman of Duke University emphasized the implications of these findings, stating, “The results from NEO100 signify a potential paradigm shift in the treatment of recurrent IDH1-mutant gliomas.”

Key Data Highlights:

Radiographic Response: 24% (6 of 25 patients) achieved significant radiographic remission confirmed by contrast-enhanced, T2-FLAIR, and perfusion MRI. This response rate exceeds the <8% typically observed with salvage therapies for recurrent gliomas.

Progression-Free Survival (PFS-6): 44% of patients achieved 6-month progression-free survival (PFS-6), outperforming historical benchmarks of 21–31% for IDH1-mutant recurrent high-grade gliomas.

Long Term Survival: 36% (9 of 25) demonstrated durable survival ≥18 months post-initiation of NEO100.

Tolerability: No significant toxicity observed even with prolonged, chronic intranasal administration.


Study Context: All patients in the combined analysis (1 compassionate-use case, 5 from Phase 1, and 18 from Phase 2a) had confirmed WHO recurrent Grade III/IV IDH1-Mutant Astrocytoma and were included in the analysis if they had been enrolled in the study for at least six months prior to data cutoff. Patients received intranasal NEO100, and results were assessed using Response Assessment in Neuro-Oncology (RANO) criteria.

Collectively, these findings indicate that NEO100 could represent a first-in-class, CNS-penetrant metabolic therapy potentially inducing significant radiographic response and extending survival in patients with recurrent Grade III/IV IDH1-Mutant Astrocytoma, thereby potentially offering a major advancement over past approaches predominantly limited to palliative care.

ABOUT NEONC TECHNOLOGIES HOLDINGS, INC.
NeOnc Technologies Holdings, Inc. is a clinical-stage life sciences company focused on the development and commercialization of central nervous system therapeutics that are designed to address the persistent challenges in overcoming the blood-brain barrier. The company’s NEO™ drug development platform has produced a portfolio of novel drug candidates and delivery methods with patent protections extending to 2038. These proprietary chemotherapy agents have demonstrated positive effects in laboratory tests on various types of cancers and in clinical trials treating malignant gliomas. NeOnc’s NEO100™ and NEO212™ therapeutics are in Phase II human clinical trials and are advancing under FDA Fast-Track and Investigational New Drug (IND) status. The company has exclusively licensed an extensive worldwide patent portfolio from the University of Southern California consisting of issued patents and pending applications related to NEO100, NEO212, and other products from the NeOnc patent family for multiple uses, including oncological and neurological conditions.

For more about NeOnc and its pioneering technology, visit neonc.com.

Important Cautions Regarding Forward Looking Statements
This press release contains “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These forward-looking statements can be identified by terminology such as “may,” “will,” “should,” “intend,” “expect,” “plan,” “budget,” “forecast,” “anticipate,” “believe,” “estimate,” “predict,” “potential,” “continue,” “evaluating,” or similar words. Statements that contain these words should be read carefully, as they discuss our future expectations, projections of future results of operations or financial condition, or other forward-looking information.

Examples of forward-looking statements include, among others, statements regarding whether a definitive agreement will be reached with Quazar. These statements reflect our current expectations based on information available at this time, but future events may differ materially from those anticipated.

The “Risk Factors” section of our most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission, along with other cautionary language in that report or in our subsequent filings, outlines important risks and uncertainties. These may cause our actual results to differ materially from the forward-looking statements herein, including but not limited to the failure to finalize the agreement with Quazar, modifications to its terms, or alternative uses of proceeds.

We assume no obligation to revise or update any forward-looking statements, whether as a result of new information, future developments, or otherwise, except as required by applicable securities laws and regulations.

“NEO100” and NEO “212” are registered trademarks of NeOnc Technologies Holdings, Inc.

Company Contact:
info@neonc.com

Investor Contact:
James Carbonara
Hayden IR
(646)-755-7412
James@haydenir.com

FAQ

What were NeOnc (NTHI) updated response rates for NEO100 in recurrent IDH1-mutant astrocytoma on Dec 15, 2025?

The update reported radiographic remission in 24% (6 of 25 patients).

How did NEO100 perform on 6-month progression-free survival (PFS-6) in the Dec 15, 2025 update for NTHI?

The cohort achieved a PFS-6 of 44%, exceeding historical benchmarks of 21–31%.

How many patients treated with NEO100 remained alive ≥18 months in NeOnc's Dec 15, 2025 dataset (NTHI)?

9 of 25 patients (36%) remained alive at or beyond 18 months after starting NEO100.

What safety findings did NeOnc (NTHI) report for intranasal NEO100 on Dec 15, 2025?

The company reported no significant toxicity observed even with prolonged, chronic intranasal administration.

What patient populations were included in NeOnc's Dec 15, 2025 NEO100 analysis for NTHI?

The combined analysis included 1 compassionate-use case, 5 Phase 1, and 18 Phase 2a patients with recurrent WHO Grade III/IV IDH1-mutant astrocytoma.

How large is the expanded NEO100 clinical cohort reported by NeOnc (NTHI) on Dec 15, 2025?

The expanded clinical cohort includes 25 patients.