CALABASAS, Calif., Nov. 12, 2025 (GLOBE NEWSWIRE) — NeOnc Technologies Holdings, Inc. (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 current Phase 1/2a and compassionate care studies involving a cohort of 24 patients with recurrent WHO Grade III/IV isocitrate dehydrogenase 1 (IDH1)–mutant astrocytoma treated with its investigational intranasal agent, NEO100.
Clinical results provided support that treatment with intranasally delivered NEO100 resulted in a significant radiographic response, confirmed by contrast-enhanced and perfusion MRI, in 5 of 24 patients (21%)—a rate that markedly exceeds the <8% response typically observed with salvage therapies for recurrent gliomas. Additionally, results indicated that 44% of patients in this Phase 2a study achieved six-month progression-free survival (PFS-6), surpassing the 21–31% benchmark reported in historical datasets for IDH1-mutant recurrent high-grade gliomas. Furthermore, 8 of 24 patients (33%) remained alive ?18 months after initiation of NEO100, demonstrating long term survival.
In addition, no significant toxicity was reported with intranasal administration of NEO100, even with prolonged, chronic intranasal administration, underscoring its potential utility as a therapeutic option for this patient population.
Amir F. Heshmatpour, Executive Chairman, President & CEO of NeOnc Technologies Holdings, Inc., stated: “With these data, we believe NeOnc stands at the threshold of a true game-changer for one of medicine’s greatest unmet needs—recurrent IDH1-mutant hi grade gliomas. For the first time, we’re witnessing evidence of meaningful radiographic responses and support for durable survival in patients who previously had few, if any, options. As we approach full enrollment of the NEO100 trial—and our full data readout six months post-enrollment—we believe this may represent a potential paradigm shift in the treatment of brain cancer. The data will speak for itself—and for the patients whose lives we are determined to transform.”
Mr. Heshmatpour added: “We view this trial as a pivotal value inflection point for NeOnc and for the broader neuro-oncology field signaling a potential move beyond palliation toward measurable, durable disease control and a possible new standard of care in malignant gliomas.”
Founder, Dr. Thomas Chen, Vice-Chairman and Chief Medical Officer noted that, “The data suggest that intranasal NEO100 may be the first central nervous system (CNS)–penetrant metabolic therapy capable of inducing durable response 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: 21% (5 of 24 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: 33% (8 of 24) 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.
