Item 1. Business
We are a microbiome therapeutics platform company developing a novel class of biological drugs, which we refer to as Ecobiotic® microbiome therapeutics. The human microbiome is an ecosystem of microorganisms, including bacteria, fungi and viruses, that, when unhealthy, or dysbiotic, can leave the body more susceptible to infections, metabolic disorders, allergies, autoimmune disease, inflammation and other serious conditions. Our drug candidates are designed to restore health by repairing the function of a dysbiotic microbiome. We are initially focused on implementing our microbiome therapeutics platform to develop Ecobiotic microbiome therapeutics that treat dysbiosis in the colonic microbiome, one of the most diverse microbial ecologies in the human body.
The human microbiome is one of the richest and most diverse ecosystems on earth, with a population of approximately 30 – 50 trillion microorganisms that live in our intestines, mouth, skin and elsewhere in or on the body. In a healthy, symbiotic state the colonic microbiome plays an important role in human health, helping the body digest food, resist pathogens, regulate the metabolic systems, develop and regulate the immune system and synthesize essential nutrients and vitamins. However, the colonic microbiome may change in composition for a variety of reasons, including in response to antibiotics or following gastrointestinal infection. These changes in composition may result in the loss of key commensal microbes and/or the gain of pathogenic microbes, resulting in a state of dysbiosis, and associated loss or gain of metabolic and/or immune function. While the study of the human microbiome is not new, the scientific community’s understanding of the microbiome, and the colonic microbiome in particular, has been significantly advanced through metagenomics, which has enabled the broader understanding of the human microbiome at the organismal, functional and community level. Scientific research has correlated dysbiosis in the colonic microbiome with various conditions, including: infectious diseases, metabolic diseases, and inflammatory and immune diseases and cancer.
We are developing a new approach to treating disease by modulation of the dysbiotic colonic microbiome by using our Ecobiotic microbiome therapeutics to improve patient outcomes. Our approach is premised on the hypothesis that the proximal cause of many diseases is a dysbiosis in the natural state of the colonic microbiome. We believe that the restoration of a dysbiotic colonic microbiome using Ecobiotic microbiome therapeutics represents a paradigm shift in the approach to treating underlying disease. There are currently no therapeutics approved by the U.S. Food and Drug Administration, or the FDA, that are designed to restore the microbiome to a healthy state.
Our approach to discovery and design is based on an iterative bedside-to-bench-to-bedside drug discovery strategy, i.e. reverse translation that begins with data on the human microbiome that we gather from clinical studies. From these clinical data, we identify the microbiological and functional differences between a healthy and a diseased microbiome, which we then use to design potential Ecobiotic microbiome therapeutics. After further in-lab preclinical testing, selected Ecobiotic microbiome therapeutic candidates are then studied in clinical trials. We apply a comparative genomic systems biology framework that leverages proprietary computational, microbiological and screening capabilities to design lead candidates that target the microbiological and functional deficiencies identified in the setting of human disease. We are able to apply this framework and experience to clinical data sets from published studies and those generated with our collaborators, as well as to the proprietary clinical data set we have generated through our clinical trials. We believe we can utilize our knowledge and data to design Ecobiotic microbiome therapeutics to treat various medical conditions. We also have advanced capabilities in pharmacokinetic and pharmacodynamic analytics, and the production and formulation of colonic bacteria as well as spore forms of bacteria into therapeutics. We believe that the combination of experience, proprietary data and proprietary know-how that comprise our microbiome therapeutics platform provides us with a competitive advantage in the design and development of microbiome therapeutics. Further, our approach and platform, which enable the design, testing, optimization, manufacturing and formulation of Ecobiotic microbiome therapeutic candidates, provide a framework that we believe can significantly reduce the time typically required to advance therapeutics to the clinic.
Using our microbiome therapeutics platform, we are focusing our resources on obtaining clinical results from our highest-priority, clinical programs in ulcerative colitis, or UC, a form of inflammatory bowel disease, or IBD, with SER-287, Clostridium difficile infection, or CDI, with SER-109, and our recently initiated Phase 1b study with SER-401 in patients with metastatic melanoma, as well as our preclinical SER-301 program in UC.
The clinical development of SER-287 to treat UC, is supported by successful clinical and preclinical studies. Preclinical colitis animal models and in vitro screens provide evidence that SER-287 administration has the potential to result in reduced pathology and modulation of inflammatory and immunological functional pathways. Published clinical reports also suggest that modulation of the microbiome through repetitive fecal microbiota transplantation, or FMT may lead to meaningful clinical response in UC patients.