Stem Cell Medicine has licensed an exosomes based technology for the treatment of neurodegenerative and neuropsychiatric indications, specifically, autism spectrum disorder (ASD). The treatment is based on vesicles, 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 preclinical 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.

"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," Prof. Ditza A. Zachor, Head, The Autism Center/ALUT, Assaf Harofeh Medical Center 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," Ehud Marom, SCM's Chairman, said. "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."

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