Hebrew University’s preclinical results recently published in Advanced Science show a reversal of the molecular, synaptic and behavioral autism spectrum disorder (ASD) phenotypes
Plan to initiate a first-in-human clinical trial of nNOS for treatment of ASD in 2025
Conference call at 4:30 PM ET today, June 15, 2023
GARDEN CITY, N.Y., June 15, 2023 — Beyond Air, Inc. (NASDAQ: XAIR) (“Beyond Air” or the “Company”), a commercial stage medical device and biopharmaceutical company focused on 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 (through its affiliate Beyond Cancer, Ltd.), today announced that it has entered into an agreement with Yissum Research Development Company of the Hebrew University of Jerusalem, LTD. to license the commercial rights for multiple neuronal nitric oxide synthase (nNOS) inhibitor candidates, currently being developed for the treatment of autism spectrum disorder (ASD) and other neurological conditions. There are currently no therapies approved by the U.S. Food and Drug Administration (FDA) specifically for the treatment of ASD.
“We are the world’s leading developer of nitric oxide based therapies, as evidenced by FDA approval of our LungFit PH to treat term and near-term neonates with hypoxic respiratory failure (“persistent pulmonary hypertension of the newborn” or “PPHN”), our robust pipeline of revolutionary LungFit systems in clinical trials for the treatment of various severe lung infections using inhaled NO, the development of ultra-high concentration NO to treat solid tumors through Beyond Cancer, and now the development of nNOS inhibitors to treat autism,” stated Steve Lisi, Chairman and Chief Executive Officer of Beyond Air. “The results of groundbreaking preclinical studies conducted by Dr. Haitham Amal of the Hebrew University suggest a direct link between NO and ASD, which may lead to the first therapy to reverse ASD behavioral deficits. We are pleased to have entered into this partnership and look forward to initiating a planned first-in-human study in 2025.”
“This is an exciting novel approach to treating ASD. The partial inhibition of nNOS reduces nitrosative stress biomarkers and reverses the molecular, synaptic and behavioral ASD-associated phenotypes. Confirming these results in humans should be expedited,” commented Dr. Amal.
Dr. Amal is an Assistant Professor at the Institute for Drug Research, Faculty of Medicine at the Hebrew University of Jerusalem. He began his academic journey at the age of seventeen, with a degree in Pharmacy, from the Hebrew University. From there he moved to Tel Aviv University for his master’s degree in neuropharmacology in the Faculty of Medicine. His PhD was conducted at the Technion Institute of Technology in Israel. Dr. Amal started his work on ASD and Alzheimer’s disease at MIT in 2015. At MIT, the first paper of his postdoctoral research was published in the Journal of Molecular Psychiatry – the leading journal in Psychiatry. Dr. Amal’s research has been widely published, with more than 30 publications on cancer, autism, Alzheimer’s disease, and other brain disorders; he has been the recipient of many awards including from the Wolf Foundation. Dr. Amal is a member of many local and international scientific committees and societies.
Under the terms of the agreement, Beyond Air will pay the Hebrew University of Jerusalem, LTD. fees associated with certain pre-clinical, clinical, regulatory and sales milestones. Additionally, the Company will pay a low single digit royalty on net sales.
At normal physiological levels, NO production and degradation are balanced and involved in normal cell signaling and in regulating numerous physiological functions. At higher concentrations, however, NO produced via nNOS becomes toxic and can lead to abnormal cell signaling and cell death. This dysregulation of nNOS may be responsible for various neurodevelopmental, neuropsychiatric, and neurodegenerative disorders including ASD.
Dr. Haitham Amal’s lab at the Hebrew University of Jerusalem conducted multidisciplinary comprehensive experiments using cellular and mouse models, as well as clinical samples and human induced pluripotent stem cells (iPSCs) to investigate the role of NO in ASD. To establish if NO leads to biochemical and cellular ASD-like phenotypes, C57BL/6J wild-type (WT) male mice were treated with an NO donor, S-nitroso-N-acetyl penicillamine (SNAP). The study demonstrated, biochemically and behaviorally, that treating WT mice with a NO donor led to an ASD-like phenotype. To determine the involvement of NO in ASD, the study inhibited NO production pharmacologically in the Shank3 mouse (M1) model. For this purpose, M1 mice were treated with a nNOS inhibitor, 7-nitroindazole (7-NI). The Cntnap2(-/-) mutant mouse (M2) model was also used. Like the M1 model, the M2 mice were treated with 7-NI. The results showed that pharmacological intervention with a neuronal NO synthase inhibitor in both models led to a reversal of the molecular, synaptic, and behavioral ASD-associated phenotypes.
Additionally, the study showed that treating human iPSC-derived cortical neurons from patients with SHANK3 mutations with the nNOS inhibitor showed similar therapeutic effects.
This innovative work suggests that NO may play a significant role in ASD development and suggests a novel strategy for an effective treatment. These important findings have unlocked a new pathway to examine NO in diverse mutations on the spectrum, as well as in other neurodevelopmental disorders and psychiatric diseases.
The pre-clinical research, funded by HUJI, was published in Advance Science on May 22, 2023 and can be found here.
Conference Call & Webcast
Company management will host a conference call and webcast at 4:30 p.m. ET today, June 15, 2023, to discuss today’s announcement. To access the call, please dial:
- US: 1-877-407-0784
- Int’l: 1-201-689-8560
- Israel Toll Free: 1 809 406 247
Conference ID number: 13739448
- Participants can use Guest dial-in #s above and be answered by an operator OR click the Call me™ link for instant telephone access to the event.
- Call me™ link will be made active 15 minutes prior to scheduled start time.
To access the live audio webcast, please click here, or go to the Events section of Beyond Air’s website at https://www.beyondair.net/news-events/events/. A replay will be available on the Company’s website shortly after the conclusion of the event for approximately 90 days.
About the Hebrew University of Jerusalem
The Hebrew University of Jerusalem is Israel’s leading academic and research institution. Serving some 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. In 2022 Hebrew University was ranked at number 77 in the 2022 Academic Ranking of World Universities by Shanghai Ranking, making it the leading Israeli university in the world. 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.
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 11,000 patents globally; licensed over 1,140 technologies and has spun out more than 200 companies. Yissum’s business partners span the globe and include companies such as Boston Scientific, ICL, Intel, Johnson & Johnson, Merck, Novartis and many more.
For further information please visit www.yissum.co.il
About Autism Spectrum Disorder (ASD)
ASD is a serious neurodevelopmental and behavioral disorder, and one of the most disabling conditions and chronic illnesses in children. ASDs include a wide range of developmental disorders that share a core of neurobehavioral deficits manifested by abnormalities in social interactions, deficits in communication, restricted interests, and repetitive behaviors. In 2023, the CDC reported that approximately 1 in 36 children in the U.S. is diagnosed with an ASD. On average, ASD costs an estimated $60,000 a year through childhood, with the bulk of the costs in special services and lost wages related to increased demands on one or both parents. Mothers of children with ASD, who tend to serve as the child’s case manager and advocate, are less likely to work outside the home. On average, they work fewer hours per week and earn 56 percent less than mothers of children with no health limitations and 35 percent less than mothers of children with other disabilities or disorders. The cost of caring for Americans with autism reached $268 billion in 2015 and is expected to rise to $461 billion by 2025 in the absence of more-effective interventions and support across the life span.
About Beyond Air®, Inc.
Beyond Air is a medical device and biopharmaceutical company dedicated to harnessing the power of nitric oxide through its revolutionary NO Generator and Delivery System, LungFit, that uses NO generated from ambient air to deliver precise amounts of NO to the lungs for the potential treatment of a variety of pulmonary diseases. LungFit can generate up to 400 ppm of NO, for delivery either continuously or for a fixed amount of time and has the ability to either titrate dose on demand or maintain a constant dose. The Company has received FDA approval for its first system, LungFit PH, for persistent pulmonary hypertension of the newborn. 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), nontuberculous mycobacteria (NTM) and severe lung infections in other settings. Additionally, Beyond Cancer, 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.
Forward Looking Statements
This press release contains “forward-looking statements” concerning , among other things, the potential safety and efficacy of the Company’s nitric oxide based therapies, including the Company’s newly licensed multiple neuronal nitric oxide synthase (nNOS) inhibitor product candidates currently being developed for the treatment of Autism spectrum disorder (ASD) and other neurological conditions; the Company’s expectations regarding the therapeutic potential of its product candidates in a number of indications; the potential for the Company to develop the first therapy to reverse the negative behavioral effects of ASD; the Company’s clinical development plans, including its plans to initiate a first-in-human clinical trial for ASD in 2025; the Company’s expectations regarding the benefits of the license agreement with Yissum Research Development Company of the Hebrew University of Jerusalem; and the expected costs of caring for Americans with autism in the absence of more-effective interventions and support across the life span. Forward-looking statements include statements about expectations, beliefs, or intentions regarding product offerings, business, results of operations, strategies or prospects. You can identify such forward-looking statements by the words “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 risks related to: uncertainties and delays relating to the design, enrollment, completion, and results of clinical trials; unanticipated costs and expenses; the timing of regulatory filings for the Company’s product candidates; clinical trial results may not support regulatory approval or further development in a specified indication or at all; actions or advice of the FDA or other comparable regulatory authorities may affect the design, initiation, timing, continuation and/or progress of clinical trials or result in the need for additional clinical trials; the size and growth potential of the markets for the Company’s product candidates, and the company’s ability to service those markets; the Company’s expectations, projections and estimates regarding expenses, future revenue, capital requirements, and the availability of and the need for additional financing; the Company’s ability to obtain additional funding to support its clinical development and commercial programs; the approach to discover and develop novel drugs, which is unproven and may never lead to efficacious or marketable products; obtaining, maintaining and protecting intellectual property utilized by the Company’s products and product candidates; 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 Cancer and Beyond Air undertake no obligation to update, and have no policy of updating or revising, these forward-looking statements, except as required by applicable law.
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