NeuroNOS Granted FDA Orphan Drug Designation for Glioblastoma, the Most Common and Deadliest Primary Malignant Brain Cancer in Adults
NeuroNOS, a subsidiary of Beyond Air (NASDAQ: XAIR), has received FDA Orphan Drug Designation (ODD) for its lead therapy BA-101 in treating Glioblastoma (GBM), an aggressive brain cancer. This marks the company's second ODD and its entry into oncology.
GBM currently has limited treatment options with median survival less than 12 months, and survival rates of less than 20% at two years and less than 10% at five years. The ODD grants several benefits, including seven years of market exclusivity, tax credits for clinical trials, and prescription drug user fee waivers.
The company's approach focuses on NO inhibition strategy, supported by research showing nitric oxide as an important modulator in GBM treatment response.
NeuroNOS, controllata di Beyond Air (NASDAQ: XAIR), ha ottenuto la Designazione di Farmaco Orfano (ODD) dalla FDA per il suo principale trattamento BA-101 contro il Glioblastoma (GBM), un tumore cerebrale aggressivo. Si tratta della seconda ODD per la società e del suo ingresso nel campo dell'oncologia.
Il GBM dispone attualmente di opzioni terapeutiche limitate, con una sopravvivenza mediana inferiore a 12 mesi e tassi di sopravvivenza di meno del 20% a due anni e di meno del 10% a cinque anni. La ODD comporta diversi vantaggi, tra cui sette anni di esclusività di mercato, crediti d'imposta per gli studi clinici e l'esenzione dalle tasse per la presentazione di farmaci soggetti a prescrizione.
L'approccio dell'azienda si basa su una strategia di inibizione del NO, sostenuta da ricerche che identificano il monossido di azoto come importante modulatore nella risposta alle terapie per il GBM.
NeuroNOS, filial de Beyond Air (NASDAQ: XAIR), ha recibido la Designación de Fármaco Huérfano (ODD) de la FDA para su terapia principal BA-101 en el tratamiento del Glioblastoma (GBM), un cáncer cerebral agresivo. Esta es la segunda ODD de la compañía y marca su entrada en oncología.
El GBM cuenta actualmente con opciones de tratamiento limitadas, con una supervivencia media inferior a 12 meses y tasas de supervivencia de menos del 20% a los dos años y de menos del 10% a los cinco años. La ODD ofrece varios beneficios, incluyendo siete años de exclusividad en el mercado, créditos fiscales para ensayos clínicos y exención de las tasas de usuario por fármacos con receta.
El enfoque de la compañía se centra en una estrategia de inhibición del NO, respaldada por investigaciones que muestran que el óxido nítrico es un modulador importante en la respuesta al tratamiento del GBM.
NeuroNOS는 Beyond Air (NASDAQ: XAIR)의 자회사로서, 공격적인 뇌종양인 교모세포종(GBM) 치료를 위한 주력 치료제 BA-101에 대해 FDA 희귀의약품 지정(ODD)을 받았습니다. 이는 회사의 두 번째 ODD이자 종양학 분야 진출을 의미합니다.
GBM은 현재 치료 옵션이 제한적이며, 중앙 생존기간이 12개월 미만이고 2년 생존율은 20% 미만, 5년 생존율은 10% 미만입니다. ODD는 7년간의 시장 독점권, 임상시험 세액공제, 처방약 사용자 수수료 면제 등 여러 혜택을 제공합니다.
회사의 접근법은 NO(일산화질소) 억제 전략에 중점을 두고 있으며, 일산화질소가 GBM 치료 반응의 중요한 조절자임을 보여주는 연구들이 이를 뒷받침합니다.
NeuroNOS, filiale de Beyond Air (NASDAQ: XAIR), a obtenu la Désignation de Médicament Orphelin (ODD) de la FDA pour son traitement principal BA-101 dans le glioblastome (GBM), une tumeur cérébrale agressive. Il s'agit de la deuxième ODD de la société et de son entrée en oncologie.
Le GBM dispose actuellement de peu d'options thérapeutiques, avec une survie médiane inférieure à 12 mois et des taux de survie de moins de 20 % à deux ans et de moins de 10 % à cinq ans. L'ODD confère plusieurs avantages, notamment sept ans d'exclusivité commerciale, des crédits d'impôt pour les essais cliniques et une exonération des frais pour les médicaments sur ordonnance.
L'approche de la société privilégie une stratégie d'inhibition du NO, appuyée par des recherches montrant que l'oxyde nitrique est un modulateur important de la réponse aux traitements du GBM.
NeuroNOS, eine Tochtergesellschaft von Beyond Air (NASDAQ: XAIR), hat für ihre führende Therapie BA-101 zur Behandlung von Glioblastom (GBM), einem aggressiven Hirntumor, die Orphan Drug Designation (ODD) der FDA erhalten. Dies ist die zweite ODD des Unternehmens und markiert dessen Einstieg in die Onkologie.
Für GBM gibt es derzeit nur begrenzte Behandlungsoptionen, mit einer medianen Überlebenszeit von unter 12 Monaten und Überlebensraten von unter 20 % nach zwei Jahren bzw. unter 10 % nach fünf Jahren. Die ODD bringt mehrere Vorteile mit sich, darunter sieben Jahre Marktexklusivität, Steuergutschriften für klinische Studien und Befreiung von Gebühren für verschreibungspflichtige Arzneimittel.
Der Ansatz des Unternehmens konzentriert sich auf eine NO-Inhibitionsstrategie, gestützt von Forschungsergebnissen, die Stickstoffmonoxid als wichtigen Modulator der Therapieantwort beim GBM identifizieren.
- Received FDA Orphan Drug Designation for BA-101 in Glioblastoma treatment
- Seven years of market exclusivity upon approval
- Tax credits and fee waivers through ODD benefits
- Second orphan drug designation, expanding therapeutic portfolio
- Entry into oncology market with significant unmet medical need
- Early-stage development - therapy not yet in human trials
- Significant development time and costs ahead before potential commercialization
Insights
FDA's Orphan Drug Designation for NeuroNOS's glioblastoma therapy provides regulatory advantages but remains in early development stages.
The FDA's granting of Orphan Drug Designation (ODD) to NeuroNOS's BA-101 for glioblastoma represents a significant regulatory milestone with several tangible benefits. This designation provides the company with tax credits toward clinical trials, prescription drug user fee waivers, and most importantly, seven years of market exclusivity upon potential approval. This exclusivity period operates independently from patent protection, effectively extending the commercial runway for BA-101.
Glioblastoma represents a compelling target with substantial unmet need - it's the most common and aggressive primary brain malignancy in adults, with median survival less than 12 months and five-year survival below 10% despite current standard-of-care treatments. The market, while smaller than major cancer indications (affecting fewer than 200,000 people in the US), benefits from regulatory incentives designed to encourage development in such underserved areas.
It's critical to note that BA-101 appears to be in pre-clinical development stages, as the CSO mentions "accelerating development toward first-in-human studies." The scientific approach - focusing on nitric oxide (NO) modulation - represents a novel mechanism of action, though clinical validation remains pending. This is NeuroNOS's second ODD designation, suggesting a strategic focus on the rare disease regulatory pathway and marks their expansion into oncology applications.
BOSTON, Sept. 08, 2025 (GLOBE NEWSWIRE) -- NeuroNOS, a biopharmaceutical company focused on developing treatments for neurological disorders and neuro-oncology, and a subsidiary of Beyond Air (NASDAQ: XAIR), today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its lead investigational therapy, BA-101, for the treatment of Glioblastoma (GBM).
GBM is an aggressive primary brain tumor with limited treatment options and poor prognosis under current standard-of-care approaches. While surgery, radiation, and temozolomide are standard of care and can extend survival, they are not considered curative in glioblastoma. Median survival is less than 12 months with two and five-year survival being less than
“We are pleased to receive orphan drug designation from the FDA for the treatment of glioblastoma. This is our second orphan drug designation and further highlights our mission to bring targeted therapies to individuals and families affected by rare neurological conditions, while also marking our entrance into oncology,” said Amir Avniel, CEO of NeuroNOS. “Glioblastoma is one of the most common and deadliest brain cancers in adults, however, patients have seen little improvement in treatment options over the past several decades. Emerging industry research shows that NO is an important modulator of biological therapy response in Glioblastoma. We believe this data and the urgent unmet medical need have highlighted the opportunity for our groundbreaking science to develop small molecule therapies that balance nitric oxide levels in the brain. We believe an NO inhibition strategy has the potential to transform outcomes for patients.”
The FDA grants ODD to drugs and biologics that are intended for safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S. ODD provides certain incentives, such as tax credits toward the cost of clinical trials upon approval and prescription drug user fee waivers. If a product receives Orphan Drug Status from the FDA, that product is entitled to seven years of market exclusivity for the disease in which it has ODD, which is independent from intellectual property protection.
"Glioblastoma represents a profound unmet need," said Prof. Haitham Amal, CSO of NeuroNOS. "Our published papers and unpublished data showed a strong link between NO and GBM”. Prof. Amal continues, “We are committed to working closely with regulators, investigators, patient groups, and foundations to accelerate development of BA-101 toward first-in-human studies."
About Glioblastoma
Glioblastoma (GBM) is the most common and aggressive malignant primary brain tumor in adults. Despite surgery, radiation, and chemotherapy (typically temozolomide), median survival is measured in months. Dysregulated nitric oxide (NO) signaling and aberrant nitric oxide synthase (NOS) activity—including neuronal NOS (nNOS)—have been implicated in GBM biology, supporting tumor proliferation, invasiveness, angiogenesis, and therapy resistance. Preclinical studies report that NOS inhibition, including nNOS-targeting compounds, can reduce GBM cell proliferation and tumor growth and may enhance responses to temozolomide in model systems.
About NeuroNOS
NeuroNOS is at the forefront of developing innovative treatments for neurodevelopmental and neurodegenerative disorders. The company specializes in creating therapies based on small molecules that cross the blood-brain barrier to regulate Nitric Oxide (NO) levels in the brain. Preclinical studies conducted by NeuroNOS have demonstrated that NO is present at elevated levels in children with Autism Spectrum Disorder (ASD) and adults suffering from brain-related diseases such as Alzheimer's and brain cancers. The company's research has shown that managing NO levels in the brain is crucial for maintaining normal brain function. By leveraging this groundbreaking science, NeuroNOS aims to bring transformative therapies to those affected by these challenging conditions, ultimately improving individuals' lives. Through collaborations with leading research institutions and experts in the field, the company is committed to advancing medical innovation and delivering life-changing treatments. For more information, please visit https://www.neuro-nos.com.
About Beyond Air, Inc.
Beyond Air is a commercial stage medical device and biopharmaceutical company dedicated to harnessing the power of endogenous and exogenous Nitric Oxide (NO) to improve the lives of patients suffering from respiratory illnesses, neurological disorders, and solid tumors. The company has received FDA approval for its first system, LungFit® PH, for the treatment of term and near-term neonates with hypoxic respiratory failure. Beyond Air is currently advancing its other revolutionary LungFit systems in clinical trials for the treatment of severe lung infections, such as viral community-acquired pneumonia (including COVID-19), and nontuberculous mycobacteria (NTM) among others. Also, the company has partnered with The Hebrew University of Jerusalem to advance a pre-clinical program dedicated to the treatment of Autism Spectrum Disorder (ASD) and other neurological disorders. In addition, Beyond Cancer, Ltd., an affiliate of Beyond Air, is investigating ultra-high concentrations of NO with a proprietary delivery system to target certain solid tumors in the pre-clinical setting. For more information, visit www.beyondair.net.
About the Hebrew University of Jerusalem (HUJI)
The Hebrew University of Jerusalem is Israel's leading academic and research institution. Serving 24,000 students from 80 countries, it produces a third of Israel's civilian research and is ranked 12th worldwide in biotechnology patent filings and commercial development. Faculty and alumni of the Hebrew University have won eight Nobel Prizes and a Fields Medal. For more information about the Hebrew University, please visit http://new.huji.ac.il/en.
About Yasim
Yissum is the technology transfer company of the Hebrew University of Jerusalem. Founded in 1964, it serves as a bridge between cutting-edge academic research and a global community of entrepreneurs, investors, and industry. Yissum's mission is to benefit society by converting extraordinary innovations and transformational technologies into commercial solutions that address our most urgent global challenges. Yissum has registered over 11,500 patents globally, licensed over 1,140 technologies and has spun out more than 245 companies. Yissum's business partners span the globe and include companies such as Boston Scientific, ICL, Merck and many more. For further information please visit www.yissum.co.il.
Forward Looking Statements
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FAQ
What FDA designation did Beyond Air's subsidiary NeuroNOS (NASDAQ: XAIR) receive for Glioblastoma treatment?
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