NeOnc Reports Early Clinical Improvement in First Child Treated with NEO100 Under Compassionate Use in Abu Dhabi

The child is receiving treatment outside the pediatric trial, and the single-patient observations do not establish causation.

Rhea-AI Summary

NeOnc Technologies Holdings (Nasdaq: NTHI) announced early clinical improvement in its first child treated with investigational NEO100 in Abu Dhabi. Treating physicians reported that the critically ill three-year-old boy with an aggressive brain tumor is breathing without ventilator support and beginning to move his legs after treatment began. These observations involve one patient and do not establish that NEO100 caused the improvement or predict other patients’ outcomes.

The child did not qualify for the NEO100-03 pediatric trial and receives intranasal treatment outside it through NeOnc’s Compassionate Care program, coordinated with physicians and the Department of Health – Abu Dhabi. In June 2026, NeOnc announced local IND authorization across three NEO100 protocols, with the pediatric Phase 1 and Phase 1b pathway pending further protocol review. The authorizations do not constitute commercial approval.

Positive

• Minor pointFirst Abu Dhabi pediatric NEO100 patient is breathing without ventilator support and beginning to move his legs.
• Minor pointAbu Dhabi IND authorization announced in June 2026 covers NEO100-01, NEO100-02 and NEO100-03 protocols.
• Minor pointNEO100 received FDA Rare Pediatric Disease Designation for pediatric-type diffuse high-grade gliomas, announced in March 2025.

Negative

• Minor pointSingle-patient observations do not establish that NEO100 caused improvement or predict outcomes in other patients.
• Minor point. Forward-looking: it has not happened yet and may not happen.Pediatric Phase 1 and Phase 1b pathway remains pending further protocol review.

Key Figures

Patients treated: 1 patient

Patients treated

1 patient

First patient receiving NEO100 through the Compassionate Care program

Key Terms

intranasal, ind authorization, rare pediatric disease designation, phase 1, +1 more

5 terms

intranasal medical

"investigational intranasal NEO100"

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.

ind authorization regulatory

"the Department of Health – Abu Dhabi had granted IND authorization"

Authorization of an Investigational New Drug (IND) is a regulator’s formal go-ahead that allows a company to begin testing a new drug or biologic in human clinical trials. It matters to investors because it moves a research program from laboratory and animal testing into human studies, serving as a regulatory milestone that signals progress, reduces one type of development uncertainty, and enables the company to pursue safety and effectiveness data needed for eventual market approval—much like receiving a building permit before construction can start.

rare pediatric disease designation regulatory

"granted Rare Pediatric Disease Designation to NEO100"

A rare pediatric disease designation is an official regulatory status given to a drug or therapy that targets a serious or life‑threatening condition primarily affecting children and is uncommon in the population. It matters to investors because the status often brings financial and development perks — such as tax credits, reduced fees, faster review and periods of market protection — which can lower costs, speed approval and improve the commercial outlook; think of it as a VIP pass that makes bringing a scarce, child‑focused treatment to market easier and potentially more profitable.

phase 1 regulatory

"adult Phase 1 through Phase 2 studies"

Phase 1 is the first stage of testing a new drug or medical treatment in people, focused primarily on safety, how the body handles the product, and finding a tolerated dose. Think of it as a short, tightly controlled experiment with a small group to check for dangerous side effects before wider testing; for investors it is an early milestone that reduces some uncertainty but still carries high risk and potential for both big value changes and setbacks.

diffuse high-grade gliomas medical

"for pediatric-type diffuse high-grade gliomas"

A group of malignant primary brain tumors that grow in an infiltrative (“diffuse”) pattern through surrounding brain tissue and are classified as high-grade because they show aggressive features and rapid growth (historically World Health Organization grade 3 or 4). Their diagnosis relies on tissue examination plus molecular markers under current WHO neuropathology rules, and because the tumors are not well circumscribed they are difficult to remove completely by surgery and are treated with combinations of surgery, radiation and systemic therapy.

AI-generated analysis. How Rhea-AI works. Not financial advice.

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Treating physicians report that a critically ill three-year-old boy is breathing independently and showing renewed movement following initiation of investigational treatment

CALABASAS, Calif., Oct. 16, 2026 (GLOBE NEWSWIRE) -- NeOnc Technologies Holdings, Inc. (Nasdaq: NTHI) (“NeOnc” or the “Company”), a clinical-stage biopharmaceutical company developing therapies for central nervous system (CNS) cancers, today announced early clinical improvement in its first patient treated in Abu Dhabi, United Arab Emirates, with investigational intranasal NEO100 through the Company’s Compassionate Care program. According to his treating physicians, the patient, a critically ill three-year-old boy with an aggressive brain tumor, is now breathing independently without ventilator support and beginning to move his legs following initiation of treatment. These early observations involve one patient receiving compassionate-use treatment and do not establish that NEO100 caused the improvement or predict outcomes in other patients.

“Hearing that this little boy is breathing on his own and beginning to move his legs is profoundly moving. For his family, every sign of progress matters,” said Amir F. Heshmatpour, Executive Chairman, President and Chief Executive Officer of NeOnc. “We remain cautious about these early observations and deeply grateful to his physicians and our partner, the Department of Health – Abu Dhabi, for making access to treatment possible.”

The treatment reflects coordination between NeOnc, the child’s treating physicians and the Department of Health – Abu Dhabi to provide access to investigational NEO100. The patient did not meet the eligibility criteria for the Company’s NEO100-03 pediatric clinical trial and is receiving treatment outside that trial through NeOnc’s Compassionate Care program. The program provides access to NEO100 for patients with serious or life-threatening conditions who have no comparable or satisfactory treatment alternatives.

“The improvements reported by this child’s treating physicians are encouraging, and continued clinical observation is essential to understand his progress,” said Thomas Chen, MD, PhD, Founder, Chief Medical Officer and Chief Scientific Officer of NeOnc. “This individual experience does not establish safety or efficacy. Our pediatric clinical program is designed to evaluate NEO100 systematically in children with serious brain tumors.”

NEO100 is a highly purified form of perillyl alcohol administered intranasally. Its delivery approach is designed to provide a non-invasive route to the brain. NEO100 remains investigational, and its safety and efficacy in pediatric patients have not been established.

The treatment builds on NeOnc’s expanding development activities in Abu Dhabi. In June 2026, the Company announced that the Department of Health – Abu Dhabi had granted IND authorization for NEO100 across its NEO100-01, NEO100-02 and NEO100-03 protocols, encompassing adult Phase 1 through Phase 2 studies and a pediatric Phase 1 and Phase 1b pathway pending further protocol review. In March 2025, NeOnc announced that the U.S. Food and Drug Administration had granted Rare Pediatric Disease Designation to NEO100 for pediatric-type diffuse high-grade gliomas. These authorizations and designations do not constitute approval for commercial use.

About NeOnc Technologies Holdings, Inc.

NeOnc Technologies Holdings, Inc. is a clinical-stage biopharmaceutical company developing therapies for central nervous system cancers. Its investigational programs include intranasal NEO100 and oral NEO212. NEO212 has completed Phase 1 dose escalation and is advancing toward Phase 2 development. NeOnc exclusively licenses a worldwide portfolio of issued patents and pending applications from the University of Southern California related to NEO100, NEO212 and other drug candidates and delivery approaches.

For more information, visit https://neonc.com.

Forward-Looking Statements

This press release contains forward-looking statements, including statements regarding the continued treatment of pediatric patients in Abu Dhabi through the Company's Compassionate Care program, the Company's clinical development activities in the United Arab Emirates, and the clinical potential of NEO100. These statements reflect current expectations and involve risks and uncertainties. Patients may not be treated or respond as expected, and treatment remains subject to applicable clinical, regulatory and institutional requirements; experience with individual patients treated through compassionate use may not be predictive of results in clinical trials; future studies may not demonstrate safety or efficacy; and no regulatory pathway or marketing approval is assured. Actual results may differ materially. Readers should consider the risk factors described in the Company's most recent filings with the Securities and Exchange Commission. These statements speak only as of the date of this release.

The Company undertakes no obligation to update forward-looking statements except as required by law.

“NEO100” and “NEO212” are registered trademarks of NeOnc Technologies Holdings, Inc.

Contacts

Company Contact:
info@neonc.com

Investor Contact:
Jon Nugent
Jon Nugent Communications
jon@jonnugent.com
205-566-3026

This press release was published by a CLEAR® Verified individual.

FAQ

AI-generated questions and answers. How Rhea-AI works. Not financial advice.

What improvement was reported in NeOnc’s first child treated with NEO100 in Abu Dhabi?

Treating physicians reported that the three-year-old boy is breathing independently without ventilator support and beginning to move his legs after treatment began. These early observations concern one compassionate-use patient and do not establish that NEO100 caused the improvement.

Who can access NEO100 through NeOnc’s Compassionate Care program?

The program provides access to NEO100 for patients with serious or life-threatening conditions who have no comparable or satisfactory treatment alternatives.