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Stem Cell Medicine Ltd Licenses Exosome Technology (Exosomes Derived from Adult Stem Cells) for Intranasal Treatment of Autism Spectrum Disorder (ASD) from Tel Aviv University; Pre-Clinical Testing Shows Improvement in Several Behavioral Phenotypes

On January 2, 2019, Stem Cell Medicine Ltd. (SCM), a biotechnology company developing new therapies for neurological indications, including mesenchymal stem cells (MSCs) and anti-BMP (bone morphogenic protein) molecules for the treatment of multiple sclerosis, and gene therapy for the treatment of neuropathic pain, announced that it has licensed an innovative exosomes-based technology for the treatment of neurodegenerative and neuropsychiatric indications, specifically, autism spectrum disorder (ASD). The treatment is based on exosomes, derived from adult stem cells (MSC-exo), that are administered intranasally. The technology was developed by Professor Dani Offen, Sackler School of Medicine, Sagol School of Neurosciences, Tel Aviv University, and was licensed from Ramot, the Business Engagement Center at Tel Aviv University. The first indication to be developed with the exosome technology is ASD, which is a group of neurodevelopmental disorders characterized by three core symptoms: severe impairment of social interactions and communication skills, increased repetitive behaviors, and cognitive inflexibility. The prevalence of ASD has been steadily increasing in children over the past several years, with no effective treatment, hence, it represents a growing unmet medical need. More than 3.5 million Americans live with ASD. According to the US Department of Health and Human Services (CDC), the prevalence of autism in US children in 2018 is estimated to be 1 in 59 children and has increased from 1 in 110 children in 2010, making it the fastest-growing developmental disability with currently no FDA-approved drug. Given the size of the patient population and lack of treatments, the market opportunity is compelling. SCM projects that a regulatory approved therapeutic based on the exosome technology would have blockbuster potential within five years following market launch.

In efficacy studies conducted in pre-clinical animal models, intranasal treatment with MSC-exo was associated with a significant improvement in the several autistic behavioral phenotypes. Social interaction and ultrasonic vocalizations increased, repetitive behaviors were reduced, and there was a significant improvement in maternal behaviors of pup retrieval. No negative symptoms were observed.

Professor Ditza A. Zachor, Head, The Autism Center/ALUT, Assaf Harofeh Medical Center in Israel, said: "The MSC-derived exosomes are a novel promising technology that presented strong efficacy in the pre-clinical studies conducted by the Company, providing a clinical development direction for the treatment of autism in pediatric and adult populations, a growing major unmet medical need in this field."

Ehud Marom, SCM's Chairman, said, "Intranasal administration is especially suitable for pediatric ASD patients and, based on the encouraging results we have seen to date with the MSC-exo technology, we are committed to investing in this treatment. This is an important part of SCM's focus and this program is consistent with our goal to bring novel treatments for neurological conditions, including autism, to market. SCM is rapidly progressing from pre-clinical activities to the clinical development. We plan to fund the program by raising $30 million through partnerships and investment."

STEM CELL MEDICINE LTD (SCM)

SCM is a biotechnology company that develops second-generation cell therapy products as stand-alone treatments or in combination with pharmaceuticals, with a focus on neurological indications, including MS, pain, and neuromuscular injuries, and manages the production, registration, and marketing of such products. The company, headquartered in Jerusalem, was founded in 2010 by an experienced team of entrepreneurs from the life science & pharmaceutical industries. SCM's facilities include state-of-the-art R&D laboratories and GMP production cleanrooms that enable an optimal environment for the development of products up to and including clinical trials. For more information, please visit: www.stemcell-medicine.com.

[Press release] [Stem Cell Medicine Ltd]