In the last 15 years, there has been a growing understanding that the composition of the large populations of bacteria and other microorganisms (collectively referred to as the microbiota or microbiome) resident in the human gastrointestinal (GI) tract can have a significant impact on health. This major role of the human microbiome in disease has opened the possibility of transformational therapies for many disorders that are poorly treated by conventional approaches. Public awareness of this fast-moving field is high, and the commercial interest is evidenced by increasing pharmaceutical company activity and high-profile investments in the field. Indeed, during 2014 and 2015, there have been investments (equity + debt) of close to US$ 0.7bn (see here).
However, this new field of biology and therapeutic paradigm are still in their infancy and there are no approved microbiome drugs. Fecal microbiota transplant (FMT) is the only available therapy, and it is only an investigational treatment. Nevertheless, the development pipeline of microbiome therapeutics has several promising candidates that are likely to result in commercial success stories in the foreseen future.
At Calixor Pharma Consulting, we believe that the microbiome field is particularly interesting for three main reasons:
The recent advances in high-throughput technologies have enabled characterisation of complex microbial communities in terms of taxonomy, gene expression, and production of metabolites within the human host. These advances in technologies have made possible the analysis of the role of specific bacterial strains in immune cells in the intestine. New therapies therefore emerged using these identified “good” bacteria.
There is an increase number of evidences linking the imbalance of the intestinal microbiota, often referred to as dysbiosis, with several large markets diseases, such as Inflammatory Bowel Diseases (IBD), metabolic diseases and CNS diseases. Therefore, not only a successful microbiome-based therapy has an enormous potential in terms of treating some illnesses that are currently not efficiently dealt with, but the potential commercial market is considerable.
There is no microbiome-based therapy approved, however, several positive early clinical results have emerged recently. Hence, it appears timely for pharmaceutical companies and investors to get involved in this field. A recent microbiome therapeutics market analysis has identified more than 100 microbiome products, in clinical and preclinical stages, which are being developed as therapeutic interventions for various disease areas. A healthy 27% of this pipeline accounts for molecules in clinical development; of these, majority are in PhII (see here).
Recommended literature references include:
Gut microbiota: a potential new territory for drug targeting (Nature Reviews Drug Discovery, 2008, see here)
The human microbiome: at the interface of health and disease (Nature Reviews Genetics, 2012, see here)
Drugging the gut microbiome (Nature Biotechnology, 2016, see here – Good paper for an outline of the current competitor landscape in the field).