What is NASH and why has it become such a huge issue in the United States?
NASH is a chronic disorder, characterized by inflammation and excess fat in the liver. Although asymptomatic in the early stages, patients are at risk for progression to serious complications such as cirrhosis, liver failure, and hepatocellular carcinoma. Current clinical management relies on diet and exercise but is associated with poor compliance. The absence of approved therapies is a major incentive for research into novel therapeutic approaches for this multifaceted condition. Moreover, there is a close link between NASH and obesity, and alongside the surge in obesity due to Western diets, the incidence of NASH has exploded in the last two decades and continues to grow. NASH currently affects 10-15 million Americans and is projected to become the leading cause of liver transplant by 2020. With high unmet need in a very large and growing population, the disorder is a major health burden.
How is the treatment landscape evolving for NASH?
Current therapy for NASH is predicated on dietary changes and control of co-morbidities such as T2D, hyperlipidemia, hypertension, etc. While diet and exercise may be effective in a subset of patients, they are difficult to achieve and maintain, and not all patients are candidates for bariatric surgery. Therapies to slow down or prevent disease progression are needed. Currently, there are numerous clinical trials testing potential therapeutics with several drugs undergoing pivotal trials and anticipated to become available by 2020. However, NASH is a disease with complex underlying biology and multiple therapeutic approaches will likely be required to modify the disease course. Likely, initial approvals of NASH drugs will be followed by trials of combination therapies to ultimately tackle this disorder.
Why is NASH of such high interest to the pharma/biotech space?
Biopharma is investing in the space motivated by the huge unmet medical need and a highly prevalent patient population. Currently there are no approved therapies for NASH and due to the obesity epidemic in the Western world, patient numbers are on the rise. In fact, analysts estimate the market opportunity in NASH to reach $15B in the US by 2025. Furthermore, NASH is just one facet of a multisystem disease and thus development in NASH may have the potential to be translated to other therapeutic areas or vice versa. For example, the metabolic component of NASH has overlap with diabetes and existing diabetes treatments may prove effective in controlling NASH – some companies are currently looking to reposition their diabetes drugs as patents expire. Similarly, the fibrotic component makes NASH an attractive direction for companies already developing in other fibrotic disorders.
To learn more about NASH, register for MassBio's Forum on February 1st, 2018