NeuroNOS Granted FDA Orphan Drug Designation for Phelan-McDermid Syndrome, a Neurodevelopmental Disorder Linked to Autism
NeuroNOS, a subsidiary of Beyond Air (NASDAQ: XAIR), has received FDA Orphan Drug Designation for BA-102, its lead investigational therapy targeting Phelan-McDermid Syndrome (PMS), a rare genetic disorder associated with Autism Spectrum Disorder (ASD).
PMS is caused by SHANK3 gene deletions or mutations, resulting in developmental delays, intellectual disability, and severe speech impairments. Currently, no FDA-approved treatments exist specifically for PMS. The company plans to initiate first-in-human clinical trials for ASD in the United States in 2026.
The Orphan Drug Designation provides significant benefits including:
- Seven years of market exclusivity upon approval
- Tax credits for qualified clinical trials
- FDA application fee waivers
- Access to FDA protocol assistance
NeuroNOS, una controllata di Beyond Air (NASDAQ: XAIR), ha ottenuto la Designazione di Farmaco Orfano da parte della FDA per BA-102, la sua principale terapia sperimentale rivolta alla Sindrome di Phelan-McDermid (PMS), una rara patologia genetica associata al Disturbo dello Spettro Autistico (ASD).
La PMS è causata da delezioni o mutazioni del gene SHANK3, che provocano ritardi nello sviluppo, disabilità intellettiva e gravi difficoltà nel linguaggio. Attualmente non esistono trattamenti approvati dalla FDA specifici per la PMS. L'azienda prevede di avviare nel 2026 negli Stati Uniti i primi studi clinici sull'uomo per l'ASD.
La Designazione di Farmaco Orfano offre vantaggi significativi, tra cui:
- Sette anni di esclusiva di mercato dopo l'approvazione
- Crediti d'imposta per studi clinici qualificati
- Esenzioni dalle tasse di domanda FDA
- Accesso all'assistenza sui protocolli FDA
NeuroNOS, una subsidiaria de Beyond Air (NASDAQ: XAIR), ha recibido la Designación de Medicamento Huérfano de la FDA para BA-102, su principal terapia en investigación dirigida al Síndrome de Phelan-McDermid (PMS), un trastorno genético raro asociado con el Trastorno del Espectro Autista (TEA).
El PMS es causado por deleciones o mutaciones en el gen SHANK3, lo que resulta en retrasos en el desarrollo, discapacidad intelectual y graves dificultades del habla. Actualmente no existen tratamientos aprobados por la FDA específicamente para PMS. La compañía planea iniciar ensayos clínicos en humanos para TEA en Estados Unidos en 2026.
La Designación de Medicamento Huérfano ofrece beneficios significativos, entre ellos:
- Siete años de exclusividad en el mercado tras la aprobación
- Créditos fiscales para ensayos clínicos calificados
- Exenciones en las tasas de solicitud a la FDA
- Acceso a asistencia en protocolos de la FDA
NeuroNOS는 Beyond Air(NASDAQ: XAIR)의 자회사로, 희귀 유전 질환인 펠란-맥더미드 증후군(PMS)을 표적으로 하는 주요 임상 후보 치료제 BA-102에 대해 FDA 희귀의약품 지정을 받았습니다. PMS는 자폐 스펙트럼 장애(ASD)와 관련이 있습니다.
PMS는 SHANK3 유전자 결실 또는 돌연변이로 인해 발생하며, 발달 지연, 지적 장애 및 심각한 언어 장애를 초래합니다. 현재 PMS에 대해 FDA 승인된 치료법은 없습니다. 회사는 2026년에 미국에서 ASD 대상 최초 임상시험을 시작할 계획입니다.
희귀의약품 지정은 다음과 같은 주요 혜택을 제공합니다:
- 승인 후 7년간 시장 독점권
- 자격 있는 임상시험에 대한 세금 공제
- FDA 신청 수수료 면제
- FDA 프로토콜 지원 접근
NeuroNOS, une filiale de Beyond Air (NASDAQ : XAIR), a reçu la désignation de médicament orphelin par la FDA pour BA-102, sa principale thérapie expérimentale ciblant le Syndrome de Phelan-McDermid (PMS), une maladie génétique rare associée au trouble du spectre autistique (TSA).
Le PMS est causé par des délétions ou mutations du gène SHANK3, entraînant des retards de développement, une déficience intellectuelle et de graves troubles du langage. À ce jour, aucun traitement spécifique au PMS n’a été approuvé par la FDA. La société prévoit de lancer les premiers essais cliniques chez l’humain pour le TSA aux États-Unis en 2026.
La désignation de médicament orphelin offre des avantages importants, notamment :
- Sept ans d’exclusivité sur le marché après approbation
- Crédits d’impôt pour les essais cliniques éligibles
- Exonérations des frais de dossier FDA
- Accès à l’assistance pour les protocoles FDA
NeuroNOS, eine Tochtergesellschaft von Beyond Air (NASDAQ: XAIR), hat die FDA Orphan Drug Designation für BA-102 erhalten, ihre führende experimentelle Therapie zur Behandlung des Phelan-McDermid-Syndroms (PMS), einer seltenen genetischen Erkrankung, die mit Autismus-Spektrum-Störung (ASD) verbunden ist.
PMS wird durch Deletionen oder Mutationen im SHANK3-Gen verursacht und führt zu Entwicklungsverzögerungen, geistiger Behinderung und schweren Sprachstörungen. Derzeit gibt es keine von der FDA zugelassenen spezifischen Behandlungen für PMS. Das Unternehmen plant, 2026 in den USA erste klinische Studien am Menschen für ASD zu starten.
Die Orphan Drug Designation bietet bedeutende Vorteile, darunter:
- Sieben Jahre Marktexklusivität nach Zulassung
- Steuergutschriften für qualifizierte klinische Studien
- Gebührenbefreiung bei FDA-Anträgen
- Zugang zur FDA-Protokollunterstützung
- Received FDA Orphan Drug Designation providing 7-year market exclusivity and development incentives
- Targeting an unmet medical need with no current FDA-approved treatments
- Clear development timeline with clinical trials planned for 2026
- Clinical trials haven't started yet - still years away from potential commercialization
- Early-stage development with no proven efficacy in humans yet
Insights
FDA orphan drug designation brings regulatory benefits to XAIR's subsidiary for a rare disorder with no approved treatments, but clinical trials won't start until 2026.
The FDA's Orphan Drug Designation (ODD) for NeuroNOS's BA-102 represents a significant regulatory milestone for this Beyond Air subsidiary. This designation provides tangible benefits including seven years of market exclusivity upon potential approval, tax credits for qualified clinical trials, waiver of FDA application fees, and access to FDA protocol assistance.
The designation targets Phelan-McDermid Syndrome (PMS), a rare genetic disorder with no FDA-approved treatments currently available. This creates a potential first-mover advantage in an uncontested treatment landscape. However, investors should note the extended development timeline - first-in-human trials aren't planned until 2026, indicating BA-102 is still in preclinical development.
For Beyond Air, this regulatory recognition validates their subsidiary's research direction in the neurodevelopmental disorder space. The ODD benefits could reduce development costs and regulatory hurdles for this program, though it remains an early-stage asset with all typical drug development risks. The designation establishes a clearer regulatory pathway for a treatment addressing a recognized unmet medical need.
Orphan designation for Phelan-McDermid Syndrome addresses a genetic autism subtype with no approved treatments, but development remains in early stages.
Phelan-McDermid Syndrome represents a clinically distinct genetic subtype within the broader autism spectrum. Caused by deletions or mutations affecting the SHANK3 gene, PMS manifests with global developmental delay, intellectual disability, severe speech impairments, and in many cases, features of autism spectrum disorder (ASD).
The current therapeutic landscape for PMS is characterized by a complete absence of FDA-approved treatments specifically targeting the condition. Management approaches have historically been to supportive care and symptom management rather than addressing underlying mechanisms.
NeuroNOS's strategy appears focused on addressing genetic underpinnings of this autism subtype, potentially offering a more targeted approach than broader ASD interventions. The FDA recognition through orphan designation acknowledges both the rarity of the condition and the significant unmet need.
While this regulatory milestone demonstrates progress, the planned initiation of first-in-human trials in 2026 indicates the program remains in early development stages. The company's work with the FDA, patient advocacy groups, and clinical investigators will be critical to advancing a therapy that could potentially address the root causes of this challenging condition.
The FDA designation marks a significant milestone in the development of a novel treatment for a rare neurodevelopmental disorder
Plan to initiate first-in-human U.S. clinical trials of lead drug candidate for treating ASD in 2026
BOSTON, April 21, 2025 (GLOBE NEWSWIRE) -- NeuroNOS, a biopharmaceutical company focused on developing treatments for autism and Alzheimer disorders, 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-102, for the treatment of Phelan-McDermid Syndrome (PMS), a syndrome associated with Autism Spectrum Disorder (ASD). The company is on track to initiate first-in-human clinical trials for ASD in the United States in 2026.
PMS is a rare genetic disorder most commonly caused by deletions or mutations affecting the SHANK3 gene leading to a range of symptoms, including global developmental delay, intellectual disability, severe speech impairments, and in many cases features of ASD. Currently, there are no FDA-approved treatments specifically indicated for PMS.
“Receiving orphan drug designation from the FDA is a significant step forward for this autism program, as well as our broader mission to bring targeted therapies to individuals and families affected by rare neurodevelopmental conditions,” said Amir Avniel, CEO of NeuroNOS. “By focusing on the genetic underpinnings of Phelan-McDermid Syndrome—a syndromic form of autism—we aim to address the root cause of symptoms and offer new hope where few options currently exist.”
ODD provides key development incentives, including seven years of market exclusivity upon approval, tax credits for qualified clinical trials, waiver of FDA application fees, and access to FDA protocol assistance.
“Phelan-McDermid Syndrome represents a critical unmet medical need,” said Prof. Haitham Amal, CSO of NeuroNOS. “We are committed to working closely with the FDA, patient advocacy groups, scientific foundations, and clinical investigators to accelerate development of a therapy that could meaningfully improve quality of life for those living with this challenging genetic condition.”
About Phelan-McDermid Syndrome
Phelan-McDermid Syndrome (PMS) is a rare neurodevelopmental disorder caused by deletions or mutations in the terminal region of chromosome 22, most often affecting the SHANK3 gene. PMS is characterized by intellectual disability, severe speech delays, motor impairments, hypotonia, and autism spectrum behaviors. It is considered a syndromic form of autism, in contrast to idiopathic autism, due to its clear genetic etiology and associated physical and neurological symptoms.
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 Yissum
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 is the significance of FDA Orphan Drug Designation for XAIR's BA-102 therapy?
When will XAIR begin clinical trials for its autism treatment BA-102?
What is Phelan-McDermid Syndrome and how does XAIR's treatment target it?
What is the current treatment landscape for Phelan-McDermid Syndrome?
