NeOnc Technologies Holdings, Inc. Receives Rare Pediatric Disease Designation for NEO100 in Treatment of Pediatric-Type Diffuse High-Grade Gliomas
NeOnc Technologies Holdings (NASDAQ: NTHI) has received Rare Pediatric Disease Designation (RPDD) from the FDA for their drug NEO100™ (perillyl alcohol) in treating pediatric-type diffuse high-grade gliomas. This aggressive form of brain cancer primarily affects children from birth to 18 years.
The designation qualifies NTHI for a potential Rare Pediatric Disease Priority Review Voucher (PRV) upon marketing approval of NEO100. These vouchers, which can be sold or transferred, hold significant strategic and financial value in the current market.
The FDA's decision was based on the disease meeting the statutory definition of a 'rare disease or condition.' The company is advancing NEO100 through clinical trials while collaborating with healthcare providers and patient advocacy groups to accelerate pediatric brain cancer treatment development.
NeOnc Technologies Holdings (NASDAQ: NTHI) ha ricevuto la Designazione per Malattie Pediatriche Rare (RPDD) dalla FDA per il loro farmaco NEO100™ (alcol perillilico) nel trattamento dei gliomi diffusi ad alto grado di tipo pediatrico. Questa forma aggressiva di cancro al cervello colpisce principalmente i bambini dalla nascita fino ai 18 anni.
La designazione qualifica NTHI per un potenziale Voucher di Revisione Prioritaria per Malattie Pediatriche Rare (PRV) al momento dell'approvazione per la commercializzazione di NEO100. Questi voucher, che possono essere venduti o trasferiti, hanno un notevole valore strategico e finanziario nel mercato attuale.
La decisione della FDA si basa sul fatto che la malattia soddisfa la definizione legale di 'malattia o condizione rara.' L'azienda sta portando avanti NEO100 attraverso studi clinici mentre collabora con fornitori di assistenza sanitaria e gruppi di advocacy per pazienti per accelerare lo sviluppo del trattamento del cancro al cervello pediatrico.
NeOnc Technologies Holdings (NASDAQ: NTHI) ha recibido la Designación de Enfermedad Pediátrica Rara (RPDD) de la FDA para su medicamento NEO100™ (alcohol perilílico) en el tratamiento de gliomas difusos de alto grado de tipo pediátrico. Esta forma agresiva de cáncer cerebral afecta principalmente a niños desde el nacimiento hasta los 18 años.
La designación califica a NTHI para un potencial Vale de Revisión Prioritaria para Enfermedades Pediátricas Raras (PRV) al momento de la aprobación de comercialización de NEO100. Estos vales, que pueden ser vendidos o transferidos, tienen un valor estratégico y financiero significativo en el mercado actual.
La decisión de la FDA se basó en que la enfermedad cumple con la definición legal de 'enfermedad o condición rara.' La empresa está avanzando NEO100 a través de ensayos clínicos mientras colabora con proveedores de atención médica y grupos de defensa de pacientes para acelerar el desarrollo del tratamiento del cáncer cerebral pediátrico.
NeOnc Technologies Holdings (NASDAQ: NTHI)는 소아형 확산 고등급 신경교종 치료를 위한 약물 NEO100™(페릴릴 알코올)에 대해 FDA로부터 희귀 소아 질환 지정(RPDD)을 받았습니다. 이 공격적인 형태의 뇌암은 주로 출생부터 18세까지의 어린이에 영향을 미칩니다.
이 지정은 NTHI가 NEO100의 마케팅 승인을 받을 경우 희귀 소아 질환 우선 검토 바우처(PRV)를 받을 수 있는 자격을 부여합니다. 이 바우처는 판매되거나 양도될 수 있으며 현재 시장에서 상당한 전략적 및 재정적 가치를 지닙니다.
FDA의 결정은 이 질병이 '희귀 질환 또는 상태'의 법적 정의를 충족한다는 데 기반을 두고 있습니다. 이 회사는 임상 시험을 통해 NEO100을 진행하고 있으며, 소아 뇌암 치료 개발을 가속화하기 위해 의료 제공자 및 환자 옹호 그룹과 협력하고 있습니다.
NeOnc Technologies Holdings (NASDAQ: NTHI) a reçu la désignation de Maladie Pédiatrique Rare (RPDD) de la FDA pour leur médicament NEO100™ (alcool périllylique) dans le traitement des gliomes diffus à haut grade de type pédiatrique. Cette forme agressive de cancer du cerveau touche principalement les enfants de la naissance jusqu'à 18 ans.
Cette désignation qualifie NTHI pour un potentiel Bon de Révision Prioritaire pour Maladies Pédiatriques Rares (PRV) lors de l'approbation de mise sur le marché de NEO100. Ces bons, qui peuvent être vendus ou transférés, ont une valeur stratégique et financière significative sur le marché actuel.
La décision de la FDA était basée sur le fait que la maladie répond à la définition légale d'une 'maladie ou condition rare.' L'entreprise fait progresser NEO100 à travers des essais cliniques tout en collaborant avec des prestataires de soins de santé et des groupes de défense des patients pour accélérer le développement du traitement du cancer du cerveau pédiatrique.
NeOnc Technologies Holdings (NASDAQ: NTHI) hat von der FDA die Auszeichnung für seltene pädiatrische Krankheiten (RPDD) für ihr Medikament NEO100™ (Perillylalkohol) zur Behandlung von diffusen hochgradigen Gliomen bei Kindern erhalten. Diese aggressive Form von Gehirnkrebs betrifft hauptsächlich Kinder von der Geburt bis zum 18. Lebensjahr.
Die Auszeichnung qualifiziert NTHI für einen potenziellen Prioritätsbewertungs-Gutschein für seltene pädiatrische Krankheiten (PRV) nach der Zulassung von NEO100. Diese Gutscheine, die verkauft oder übertragen werden können, haben einen erheblichen strategischen und finanziellen Wert auf dem aktuellen Markt.
Die Entscheidung der FDA basierte darauf, dass die Krankheit die gesetzliche Definition einer 'seltenen Krankheit oder Bedingung' erfüllt. Das Unternehmen entwickelt NEO100 weiter durch klinische Studien und arbeitet mit Gesundheitsdienstleistern und Patientenvertretungsgruppen zusammen, um die Entwicklung von Behandlungen für pädiatrischen Gehirnkrebs zu beschleunigen.
- Received FDA Rare Pediatric Disease Designation for NEO100
- Eligible for valuable Priority Review Voucher upon marketing approval
- PRV can be sold or transferred, representing potential financial asset
- Product still in clinical trials with no guaranteed approval
- No immediate revenue impact from designation
Insights
The FDA's Rare Pediatric Disease Designation (RPDD) for NEO100 represents a meaningful regulatory milestone for NeOnc Technologies. This designation serves three critical functions:
First, it formally recognizes pediatric-type diffuse high-grade gliomas as a "rare disease" affecting children from birth to 18 years, confirming the unmet medical need in this population. Second, it positions NEO100 (perillyl alcohol) in a specialized regulatory pathway designed to accelerate development of pediatric treatments.
Most significantly, the RPDD makes NeOnc eligible for a Priority Review Voucher (PRV) upon eventual marketing approval. PRVs are transferable assets that typically command
However, investors should understand that this designation does not evaluate NEO100's efficacy or safety profile. The path to commercialization still requires successful completion of clinical trials and regulatory review. The PRV value is only realized upon eventual approval, which remains several steps away in the development pipeline.
What makes this particularly notable is that PRVs provide both strategic flexibility (faster review of future products) and potential monetization options (selling to larger pharmaceutical companies) - either outcome would benefit NeOnc significantly if NEO100 ultimately secures approval.
Pediatric-type diffuse high-grade gliomas represent one of the most challenging cancers affecting children, with dismal survival rates and treatment options. These aggressive brain tumors have historically been resistant to conventional therapies, creating an urgent need for novel approaches.
NEO100 (perillyl alcohol) is particularly interesting because of its mechanism - while the article doesn't detail the specific approach, perillyl alcohol has shown potential anti-cancer properties through multiple pathways in preclinical research. The intranasal delivery method (suggested by the company's previous communications) could potentially improve drug delivery across the blood-brain barrier, a significant challenge in treating brain tumors.
The FDA's designation validates the serious and life-threatening nature of these pediatric brain tumors and acknowledges the therapeutic landscape. However, this designation only recognizes the target disease's severity and rarity, not the specific promise of NEO100.
From a clinical perspective, this regulatory step should accelerate NEO100's development timeline if the therapy demonstrates efficacy in ongoing trials. The aggressive nature of pediatric high-grade gliomas means that even incremental improvements in outcomes would represent meaningful progress for affected families.
WESTLAKE VILLAGE, Calif., March 31, 2025 (GLOBE NEWSWIRE) -- NeOnc Technologies Holdings, Inc. (NASDAQ Global Market: NTHI), a clinical-stage medical biotechnology company, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to perillyl alcohol (NEO100™) for the treatment of pediatric-type diffuse high-grade gliomas, a serious and life-threatening condition affecting children and adolescents.
The designation, issued under Section 529(a)(3) of the Federal Food, Drug, and Cosmetic Act (FD&C Act), recognizes the urgent need for treatment options for pediatric-type diffuse high-grade gliomas. The FDA’s decision is based on findings that the disease primarily affects individuals from birth to 18 years and meets the statutory definition of a “rare disease or condition.”
“This designation marks a significant milestone in our efforts to develop innovative therapies for children battling this aggressive form of brain cancer,” said Amir Heshmatpour, Executive Chairman, NeOnc Technologies Holdings, Inc. “We remain committed to advancing NEO100 through clinical development to bring new hope to patients and families facing this devastating disease.”
“Receiving the Rare Pediatric Disease Designation for NEO100 is a crucial step forward in our mission to develop effective treatments for children facing diffuse high-grade gliomas,” said Dr. Thomas Chen, CEO and Chief Science Officer of NeOnc Technologies Holdings, Inc. “This designation not only validates the potential of our research, but also strengthens our commitment to delivering innovative therapies that can make a real difference in children’s lives.”
The Rare Pediatric Disease Designation makes NeOnc Technologies eligible to receive a Rare Pediatric Disease Priority Review Voucher (PRV) upon approval of NEO100’s marketing application. The PRV program is designed to encourage the development of new drugs and biologics for rare pediatric diseases by providing companies with an expedited regulatory review process. These vouchers, which are transferable and have been sold in recent years, have significant strategic and financial value, depending on market conditions and demand.
“Currently, there is a dynamic market for PRV’s, which are quite valuable,” added Heshmatpour. “This potential asset further underscores the value of our progress, especially for the patients and families we aim to serve.”
As NeOnc Technologies continues to progress NEO100 through clinical trials, the company remains dedicated to working closely with the FDA and the broader medical community to bring this promising therapy to children in need. The company encourages collaboration with researchers, healthcare providers, and patient advocacy groups to further accelerate advancements in pediatric brain cancer treatment.
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 neonctech.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
What is the significance of NTHI's Rare Pediatric Disease Designation for NEO100?
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