NeOnc Technologies Holdings, Inc. Expects Full Enrollment in Promising Phase 2a Trial of NEO100-01 for Malignant Gliomas in September
NeOnc Technologies Holdings (NASDAQ: NTHI) announced expected full enrollment completion for its Phase 2a clinical trial of NEO100-01 in September, with only six patients remaining to reach the 25-patient target. The trial investigates NEO100, a purified perillyl alcohol delivered intranasally for treating malignant gliomas, specifically IDH1 mutant Grade III and IV astrocytomas.
The trial's read-out data is anticipated in early 2026. NEO100-01 represents the first intranasal therapy of its kind for malignant gliomas, designed to bypass the blood-brain barrier through olfactory and trigeminal nerves. Phase 1 study data indicated extended survival post-recurrence with minimal side effects in IDH1 mutation patients. The FDA has granted both Orphan Drug Designation and Fast Track Status to NEO100.
NeOnc Technologies Holdings (NASDAQ: NTHI) ha annunciato il completamento della registrazione totale previsto per il suo trial clinico di fase 2a di NEO100-01 a settembre, con solo sei pazienti rimanenti per raggiungere l'obiettivo di 25 pazienti. Il trial studia NEO100, un alcol perillil purificato somministrato per via intranasale per il trattamento dei gliomi maligni, in particolare gli astrocytomi di grado III e IV con mutazione IDH1.
I dati di lettura del trial sono attesi all'inizio del 2026. NEO100-01 rappresenta la prima terapia intranasale del suo genere per i gliomi maligni, progettata per superare la barriera emato-encefalica attraverso i nervi olfattivi e trigeminali. I dati dello studio di fase 1 hanno indicato un prolungamento della sopravvivenza dopo la recidiva con effetti collaterali minimi nei pazienti con mutazione IDH1. La FDA ha concesso sia la Designazione di Farmaco Orfano che lo Stato di Fast Track a NEO100.
NeOnc Technologies Holdings (NASDAQ: NTHI) anunció la finalización esperada de la inscripción total para su ensayo clínico de fase 2a de NEO100-01 en septiembre, con solo seis pacientes restantes para alcanzar el objetivo de 25 pacientes. El ensayo investiga NEO100, un alcohol perilílico purificado administrado por vía intranasal para el tratamiento de gliomas malignos, específicamente astrocitomas grado III y IV con mutación IDH1.
Se anticipa que los datos de lectura del ensayo se publiquen a principios de 2026. NEO100-01 representa la primera terapia intranasal de este tipo para gliomas malignos, diseñada para eludir la barrera hematoencefálica a través de los nervios olfativos y trigeminales. Los datos del estudio de fase 1 indicaron una mayor supervivencia tras la recidiva con efectos secundarios mínimos en pacientes con mutación IDH1. La FDA ha otorgado tanto la Designación de Medicamento Huérfano como el Estado de Vía Rápida a NEO100.
NeOnc Technologies Holdings (NASDAQ: NTHI)는 NEO100-01의 2a상 임상 시험에 대한 전체 등록 완료가 9월에 예상된다고 발표했으며, 25명 목표에 도달하기 위해 남은 환자는 6명뿐입니다. 이 시험은 악성 신경교종 치료를 위해 비강으로 전달되는 정제된 페릴릴 알코올인 NEO100을 조사합니다. 특히 IDH1 변이 3, 4등급 별아교세포종을 대상으로 합니다.
시험의 결과 데이터는 2026년 초에 발표될 것으로 예상됩니다. NEO100-01은 악성 신경교종을 위한 최초의 비강 치료제로, 후각 및 삼차신경을 통해 혈액-뇌 장벽을 우회하도록 설계되었습니다. 1상 연구 데이터는 IDH1 변이 환자에서 재발 후 생존 기간 연장을 나타내며 부작용은 최소화되었습니다. FDA는 NEO100에 대해 희귀 의약품 지정 및 신속 승인 상태를 부여했습니다.
NeOnc Technologies Holdings (NASDAQ: NTHI) a annoncé que l'achèvement de l'inscription totale pour son essai clinique de phase 2a de NEO100-01 est prévu pour septembre, avec seulement six patients restants pour atteindre l'objectif de 25 patients. L'essai étudie NEO100, un alcool périllylique purifié administré par voie intranasale pour traiter les gliomes malins, en particulier les astrocytomes de grade III et IV avec mutation IDH1.
Les données de lecture de l'essai sont attendues au début de 2026. NEO100-01 représente la première thérapie intranasale de son genre pour les gliomes malins, conçue pour contourner la barrière hémato-encéphalique à travers les nerfs olfactifs et trigéminaux. Les données de l'étude de phase 1 ont indiqué une survie prolongée après la récidive avec des effets secondaires minimes chez les patients présentant une mutation IDH1. La FDA a accordé à NEO100 à la fois la désignation de médicament orphelin et le statut Fast Track.
NeOnc Technologies Holdings (NASDAQ: NTHI) gab bekannt, dass die vollständige Einschreibung für seine Phase 2a-Studie von NEO100-01 im September voraussichtlich abgeschlossen sein wird, wobei nur noch sechs Patienten verbleiben, um das Ziel von 25 Patienten zu erreichen. Die Studie untersucht NEO100, einen gereinigten Perillylalkohol, der intranasal zur Behandlung von malignen Gliomen verabreicht wird, insbesondere IDH1-mutierte Grad III und IV Astrozytome.
Die Auswertung der Studiendaten wird für Anfang 2026 erwartet. NEO100-01 stellt die erste intranasale Therapie ihrer Art für maligne Gliome dar, die darauf ausgelegt ist, die Blut-Hirn-Schranke über die Riech- und Trigeminusnerven zu umgehen. Daten aus der Phase-1-Studie zeigten eine verlängerte Überlebenszeit nach Rezidiv mit minimalen Nebenwirkungen bei Patienten mit IDH1-Mutation. Die FDA hat NEO100 sowohl die Orphan Drug Designation als auch den Fast Track Status verliehen.
- Near completion of Phase 2a trial enrollment (19/25 patients)
- Promising Phase 1 data showing extended survival with minimal side effects
- FDA granted both Orphan Drug Designation and Fast Track Status
- First-in-class intranasal therapy for malignant gliomas
- Novel delivery mechanism bypassing blood-brain barrier
- Final trial results not available until early 2026
- Still in early clinical development phase
- Efficacy endpoints yet to be proven
Insights
NeOnc's NEO100-01 approaching full enrollment in its Phase 2a trial represents a noteworthy development in the challenging field of malignant glioma treatment. The trial's focus on IDH1 mutant Grade III and IV astrocytomas demonstrates a targeted approach to a genetically defined subset of these aggressive tumors.
The intranasal delivery mechanism addressing the blood-brain barrier represents a potentially significant innovation. The blood-brain barrier has historically been one of the most formidable obstacles in treating CNS malignancies, forcing developers to rely on invasive procedures or systemic therapies with poor CNS penetration. A non-invasive delivery method that bypasses this barrier could be transformative if efficacy is confirmed.
Early signals from Phase 1 showing extended survival in IDH1 mutant patients with minimal side effects warrants attention, though specific survival metrics would be needed for comprehensive evaluation. The FDA's grant of both Orphan Drug Designation and Fast Track Status validates the unmet need in this indication and provides regulatory advantages to accelerate development.
For context, Grade IV gliomas (glioblastoma) carry a median survival of approximately 15-18 months with standard treatment. Any therapy demonstrating meaningful survival extension with reduced toxicity would represent a considerable advance in neuro-oncology. However, the 2026 timeline for data readout indicates we're still in early development stages, with efficacy outcomes not yet established.
This clinical trial update for NeOnc Technologies represents a meaningful operational milestone that reduces execution risk for their lead asset. For a
Near-complete enrollment (19 of 25 patients) with full enrollment expected by September indicates the trial is progressing without recruitment challenges that often plague CNS oncology studies. This suggests the company is executing effectively on its development timeline.
The dual regulatory advantages of Orphan Drug Designation and Fast Track Status create potential for accelerated approval pathways and extended market exclusivity if efficacy is demonstrated. For rare disease indications like malignant gliomas, these designations significantly enhance commercial potential by reducing competition.
While the expected data readout in early 2026 confirms a substantial waiting period for efficacy confirmation, each de-risking event potentially rerates small biotechs like NTHI. The targeted approach to IDH1 mutant tumors aligns with precision medicine paradigms that have attracted premium valuations.
Importantly, this update focuses solely on clinical progression without providing information on cash runway. Small CNS-focused biotechs typically require substantial capital to reach pivotal trials and potential commercialization. Investors should evaluate whether NTHI's current funding can sustain operations through the 2026 data readout or if dilutive financing might be required.
-- Trial Read-Out Data Expected in Early 2026 --
CALABASAS, Calif., April 14, 2025 (GLOBE NEWSWIRE) -- NeOnc Technologies Holdings, Inc. (NASDAQ: NTHI), a clinical-stage biopharmaceutical company focused on innovative treatments for central nervous system (CNS) cancers and disorders, today announced that it expects full enrollment in its Phase 2a clinical trial for its lead therapeutic candidate, NEO100-01 in September. Only six patients remain to complete the trial’s 25-patient enrollment target.
This landmark trial, for which read-out data is expected in early 2026, investigates NEO100, a proprietary, highly purified version of perillyl alcohol, delivered intranasally, a non-invasive method designed to bypass the blood-brain barrier through the olfactory and trigeminal nerves. NEO100-01 is the first intranasal therapy of its kind evaluated in malignant gliomas, particularly targeting IDH1 mutant Grade III and IV astrocytomas. Grade IV gliomas are among the most aggressive and deadly forms of brain cancer.
“We believe our intranasal delivery mechanism can present a breakthrough in central nervous system oncology, potentially offering a practical and elegant route to circumvent the blood-brain barrier,” said Dr. Thomas Chen, Chief Executive Officer and Chief Scientific Officer at NeOnc Technologies. “Early data from our Phase 1 study provides support that patients with IDH1 mutations experienced extended survival post-recurrence with minimal side effects. We are optimistic that this approach could transform treatment outcomes for this patient population.”
“Reaching near full enrollment in the NEO100-01 trial, with only six patients remaining, is a positive and exciting development,” said Executive Chairman, Amir Heshmatpour. “Our team believes we can meet the FDA’s efficacy endpoints and deliver a positive outcome for patients battling these devastating tumors.”
NEO100 has been granted both Orphan Drug Designation and Fast Track Status by the U.S. Food and Drug Administration (FDA), reinforcing its potential as a significant advancement in the treatment of malignant gliomas.
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
All statements other than statements of historical facts included in this press release are "forward-looking statements" (as defined in the Private Securities Litigation Reform Act of 1995). Generally, such forward-looking statements include statements regarding expectations, possible or assumed future actions, business strategies, events or results of operations, including statements regarding expectations or predictions or future financial or business performance or conditions and those statements that use forward-looking words such as "projected," "expect," "possibility" and "anticipate," or similar expressions. The achievement or success of the matters covered by such forward-looking statements involve significant risks, uncertainties, and assumptions. Actual results could differ materially from current projections or implied results. The Company cautions that statements and assumptions made in this news release constitute forward-looking statements and make no guarantee of future performance. Forward-looking statements are based on estimates and opinions of management at the time statements are made. The information set forth herein speaks only as of the date hereof. The Company and its management are under no obligation, and expressly disclaim any obligation, to update, alter or otherwise revise any forward-looking statements following the date of this news release, whether because of new information, future events or otherwise, except as required by law.
“NEO100” is a registered trademark of NeOnc Technologies Holdings, Inc.
Company Contact:
Patrick Walters
Chief Operations Officer
NeOnc Technologies Holdings, Inc.
info@neonc.com
Investor Relations:
Roger Pondel / Laurie Berman
PondelWilkinson Inc.
(310) 279-5980
rpondel@pondel.com
lberman@pondel.com
FAQ
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