NeOnc's Nasal Spray Shows Promise in Glioblastoma Trial, Offering New Hope Beyond Blood-Brain Barrier

NEW YORK, NY – November 13, 2025

A New Approach to a Deadly Disease

NeOnc Technologies Holdings, Inc. announced today the completion of patient enrollment in its Phase 2a clinical trial (NCT02704858) evaluating NEO100 for recurrent IDH1-mutant high-grade glioma, a particularly aggressive form of brain cancer. This milestone represents a significant step forward in the company’s pursuit of innovative therapies for central nervous system (CNS) cancers, and offers a glimmer of hope for patients with limited treatment options. The trial, designed to assess both the safety and efficacy of NEO100, is expected to yield preliminary data in the second quarter of 2026.

The challenge of treating brain tumors has long been compounded by the blood-brain barrier (BBB), a protective mechanism that prevents many drugs from reaching the tumor site. NeOnc’s approach centers on bypassing this barrier altogether, utilizing an intranasal delivery method that allows NEO100 to reach the brain via the olfactory and trigeminal nerves. This novel delivery system, if proven effective, could revolutionize the treatment of not only glioblastoma but also other CNS disorders.

The Science Behind NEO100

NEO100 is a highly purified formulation of perillyl alcohol (POH), a naturally occurring compound found in the essential oils of various plants. The company believes its purification process and delivery method maximize the therapeutic potential of POH while minimizing systemic side effects. The current trial focuses on patients with recurrent or progressive IDH1-mutant high-grade glioma—a subset of glioblastoma characterized by a specific genetic mutation. These patients often experience disease progression despite conventional treatments, creating a significant unmet medical need.

“The ability to effectively deliver drugs to the brain is a long-standing challenge in oncology,” said one industry observer. “NeOnc’s intranasal approach is particularly intriguing, as it directly addresses this issue. The fact that they’ve completed enrollment in a Phase 2a trial is a testament to the potential of their technology.”

The trial, conducted at multiple leading cancer centers across the United States, is evaluating a dose regimen designed to maximize therapeutic benefit while minimizing toxicity. Researchers are closely monitoring patients for both clinical response—measured by changes in tumor size and functional status—and safety signals. The initial Phase 1 portion of the study helped establish the maximum tolerated dose of NEO100, paving the way for the current evaluation of efficacy in a larger cohort of patients.

Navigating the Competitive Landscape

The glioblastoma treatment landscape remains fiercely competitive, with established therapies including surgery, radiation, and chemotherapy—often combined with the alkylating agent temozolomide. While these treatments can provide initial benefits, the vast majority of patients experience disease recurrence. Several companies are actively developing new therapies targeting various aspects of glioblastoma biology, including immunotherapies, oncolytic viruses, and targeted agents.

NeOnc distinguishes itself by focusing on drug delivery, addressing a fundamental limitation of many promising therapies. “Even if you have a highly effective drug, it won’t work if it can’t reach the tumor,” explained one researcher familiar with the company’s technology. “NeOnc’s approach has the potential to unlock the full therapeutic potential of drugs that have previously been hampered by the blood-brain barrier.”

The company has secured Orphan Drug Designation, Fast Track Status, and Rare Pediatric Disease Designation from the FDA, recognizing the significant unmet medical need and the potential of NEO100. These designations provide certain regulatory benefits, including potential tax credits and market exclusivity.

Financial Considerations and Future Outlook

As a clinical-stage biotechnology company, NeOnc relies heavily on funding from venture capital, grants, and potential partnerships. The company has been actively raising capital to support its clinical development programs and expand its research pipeline. The completion of patient enrollment in the Phase 2a trial is a crucial de-risking event, potentially attracting further investment and increasing the company’s valuation.

“There’s always financial risk associated with clinical-stage biotech companies,” noted one financial analyst. “But NeOnc has a compelling technology and a clear path forward. If the Phase 2a data is positive, the company could be well-positioned to secure partnerships with larger pharmaceutical companies or even pursue commercialization on its own.”

The company's intellectual property portfolio extends to 2038, providing a significant competitive advantage. Patents covering the composition of matter for NEO100 and the intranasal delivery method protect the company’s core technology. The company is also exploring potential applications of its drug delivery platform in other CNS disorders, including Alzheimer’s disease and Parkinson’s disease. Looking ahead, the company plans to initiate additional clinical trials and expand its research pipeline, building on its innovative approach to drug delivery and its commitment to addressing unmet medical needs in the fight against brain cancer.