NeuroNOS Granted FDA Orphan Drug Designation for Phelan-McDermid Syndrome, a Neurodevelopmental Disorder Linked to Autism

April 21, 2025 16:05 ET | Source: Beyond Air™ 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 This press release contains “forward-looking statements” (as defined in Section 27A of the Securities Act and Section 21E of the Securities Exchange Act of 1934, as amended). You can identify such forward-looking statements by the words “appears,” “expects,” “plans,” “anticipates,” “believes” “expects,” “intends,” “looks,” “projects,” “goal,” “assumes,” “targets” and similar expressions and/or the use of future tense or conditional constructions (such as “will,” “may,” “could,” “should” and the like) and by the fact that these statements do not relate strictly to historical or current matters. Rather, forward-looking statements relate to anticipated or expected events, activities, trends or results as of the date they are made. Because forward-looking statements relate to matters that have not yet occurred, these statements are inherently subject to risks and uncertainties that could cause actual results to differ materially from any future results expressed or implied by the forward-looking statements. These forward-looking statements are only predictions and reflect views as of the date they are made with respect to future events and financial performance. Many factors could cause actual activities or results to differ materially from the activities and results anticipated in forward-looking statements, including those related to the completion of the offering, risks related to the ability to raise additional capital; the timing and results of future pre-clinical studies and clinical trials; the potential that regulatory authorities, including the FDA and comparable non-U.S. regulatory authorities, may not grant or may delay approval for our product candidates; the approach to discover and develop novel drugs, which is unproven and may never lead to efficacious or marketable products; the ability to fund and the results of further pre-clinical studies and clinical trials of our product candidates; obtaining, maintaining and protecting intellectual property utilized by products; obtaining regulatory approval for products; competition from others using similar technology and others developing products for similar uses; dependence on collaborators; and other risks, which may, in part, be identified and described in the “Risk Factors” section of Beyond Air’s most recent Annual Report on Form 10-K and other of its filings with the Securities and Exchange Commission, all of which are available on Beyond Air’s website. Beyond Air undertakes no obligation to update, and have no policy of updating or revising, these forward-looking statements, except as required by applicable law. Investor Relations contactsCorey Davis, Ph.D.LifeSci Advisors, LLCCdavis@lifesciadvisors.com(212) 915-2577

NeuroNOS Granted FDA Orphan Drug Designation for Phelan-McDermid Syndrome, a Neurodevelopmental Disorder Linked to Autism

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