The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to BA-102, NeuroNOS's lead investigational therapy for the treatment of Phelan-McDermid Syndrome (PMS), a rare genetic disorder associated with Autism Spectrum Disorder (ASD).
NeuroNOS, a biopharmaceutical company focused on developing treatments for autism and Alzheimer's disorders and a subsidiary of Beyond Air (NASDAQ: XAIR), announced the designation on April 21, 2025. The company plans to initiate first-in-human clinical trials for ASD in the United States in 2026.
Phelan-McDermid Syndrome is caused by deletions or mutations affecting the SHANK3 gene, resulting in global developmental delay, intellectual disability, severe speech impairments, and features of ASD. Currently, there are no FDA-approved treatments specifically indicated for this condition.
"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."
Orphan Drug Designation Benefits
The FDA's Orphan Drug Designation provides NeuroNOS with several key development incentives, including:
- Seven years of market exclusivity upon approval
- Tax credits for qualified clinical trials
- Waiver of FDA application fees
- Access to FDA protocol assistance
These incentives are designed to encourage the development of treatments for rare diseases affecting fewer than 200,000 people in the United States.
Addressing an Unmet Medical Need
Prof. Haitham Amal, Chief Scientific Officer of NeuroNOS, emphasized the significance of this development: "Phelan-McDermid Syndrome represents a critical unmet medical need. 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."
Novel Approach to Neurodevelopmental Disorders
NeuroNOS specializes in creating therapies based on small molecules that cross the blood-brain barrier to regulate Nitric Oxide (NO) levels in the brain. The company's preclinical studies have demonstrated that NO is present at elevated levels in children with ASD and adults suffering from brain-related diseases such as Alzheimer's and brain cancers.
The company's research suggests that managing NO levels in the brain is crucial for maintaining normal brain function. This approach represents a potentially groundbreaking strategy for addressing neurodevelopmental disorders at their biochemical roots.
Understanding Phelan-McDermid Syndrome
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.
The syndrome results from deletions or mutations in the terminal region of chromosome 22, most often affecting the SHANK3 gene, which plays a critical role in the formation and function of synapses in the brain.
Research Collaboration Network
NeuroNOS's work builds on collaborations with leading research institutions, including The Hebrew University of Jerusalem. Beyond Air, NeuroNOS's parent company, has partnered with the university to advance a pre-clinical program dedicated to the treatment of ASD and other neurological disorders.
The Hebrew University of Jerusalem is Israel's leading academic and research institution, ranked 12th worldwide in biotechnology patent filings and commercial development. Its technology transfer company, Yissum, has registered over 11,500 patents globally and licensed over 1,140 technologies.
Beyond Air's Broader Mission
Beyond Air is a commercial stage medical device and biopharmaceutical company dedicated to harnessing the power of endogenous and exogenous Nitric Oxide to improve the lives of patients suffering from respiratory illnesses, neurological disorders, and solid tumors.
The company has already 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 other LungFit systems in clinical trials for the treatment of severe lung infections, including viral community-acquired pneumonia and nontuberculous mycobacteria.
The development of BA-102 for Phelan-McDermid Syndrome represents an important expansion of Beyond Air's portfolio into targeted neurological therapies, potentially offering new treatment options for patients with limited therapeutic alternatives.
