Second Genome Expands Development of CNS Platform to Validate Microbial Products as Therapeutics for Autism Spectrum Disorder

March 26, 2019, South San Francisco

SOUTH SAN FRANCISCO, Calif., March 26, 2019 /PRNewswire/ -- Second Genome, Inc., a clinical-stage company focused on the development of novel therapeutics identified through microbiome science, the Stanford University School of Medicine and Oregon State University today announced the receipt of a two-year, $1.94 million Phase II Small Business Innovation Research (SBIR) grant to develop a platform for discovery and validation of key metabolites produced by microbes in CNS disorders. In particular, the teams will study the relationship between the human microbiome and autism spectrum disorders (ASDs).

This grant builds upon Phase I of the grant, which identified specific microbial strains that are either lacking or severely decreased in children with ASD. To date, 111 pairs of siblings were enrolled in the study: one neuro-typic (non-ASD) child and one ASD child born within 2 years of each other. All pairs lived in same house, provided longitudinal stool and saliva samples, and their parents charted each sibling's diet and behavior for three weeks and many submitted video documentation of behavior for participants.

"Recent studies have implicated the microbiome in several central nervous system (CNS) disorders, including autism, Parkinson's and Alzheimer's disease and even addiction," said Matt W. McClure, M.D., Chief Medical officer of Second Genome. "By combining the expertise of Second Genome and leading institutions, we believe we will be able to move from the identification of differentiating factors within the microbiome to potential therapeutics for ASD, which would be a game-changer for families worldwide."

During Phase II of the SBIR grant, Second Genome will conduct clinical, genetic and metabolomic analyses of the collected samples and data to explore the identified strains and their functions in depth. From this in-depth profiling, in addition to metagenomics and metatranscriptomics profiling, Second Genome will then use its exceptionally sensitive, PhyloChip™ technology platform to differentiate bacterial strains depleted in the ASD gut to ultimately discover novel bacterial strains and metabolites found in neuro-typic children that are greatly reduced in their sibling with ASD. Such metabolites could pass the blood-brain barrier and may affect the central nervous system and modulate behavior. Stanford University School of Medicine will then conduct in vivo experiments in animals to test the effect of these identified metabolites in CNS disease. Teams under Dennis P. Wall, Ph.D., associate professor of pediatrics, psychiatry and biomedical data sciences and Theo Palmer, PhD, professor of neurosurgery at Stanford University School of Medicine as well as Maude David, assistant professor of microbiology and pharmaceutical science at Oregon State University will also conduct exome sequencing to determine the child's genotype and build potential diagnostic tools.

"This study is a true demonstration of how Second Genome is redefining disease through the lens of the microbiome in ASD that complements our work in inflammatory bowel disease, metabolic disease, inflammation, and cancer," said Karim Dabbagh, Ph.D., CEO of Second Genome. "In parallel with our efforts in autism, we remain on track to complete our Phase 2a clinical trial in NASH in early 2020, and will soon initiate IND enabling studies for our mucosal healing program, as well as discover leads in our immuno-oncology program."

About Second Genome
Second Genome is a clinical-stage company focused on discovering and developing therapeutics identified using microbiome science. The company is evaluating SGM-1019 in a Phase 2 clinical trial in patients with NASH, moving into IND-enabling studies for a second clinical candidate involved in mucosal healing, and has a pipeline of discovery candidates in the area of immuno-oncology. The deep pipeline results from Second Genome's robust drug discovery platform, which elucidates and interrogates important microbial (microbiome) functions that strongly influence human health and disease, and serves as the basis for therapeutic discovery. Central to this platform is the world's largest, dynamic, curated database and a suite of software, hardware and data science capabilities that allows Second Genome to accurately identify microbial biology in health and disease. For more information, please visit www.secondgenome.com.