With the overindulgence of the festive period a distant memory, many of us will have stopped worrying about damage done to our livers by the couple of extra glasses of wine. Soberingly, we may have to worry about a more pertinent and silent threat to liver health. With incidence of non-alcoholic steatohepatitis (NASH) at epidemic proportions, forecast to affect over 18 million patients in the US alone by 2036, and no currently approved therapies, this disease has been touted as the next untapped pharmaceutical market capable of producing future generations of blockbuster drugs. There has been a huge increase in interest from industry in recent years, driven by the enormous potential for commercial success, but many aspects of NASH drug development are still in their nascent stages. Next week (26-27th February), London is set to host the 2018 Global NASH Congress, with an array of speakers looking to fill in the knowledge gaps of NASH therapy development.
With four emerging therapies in Phase III trials for NASH, we are at an exciting point in the disease’s history; the first approved therapies are within our grasp. However, with no precedent set for regulatory bodies reviewing/approving drugs for the treatment of NASH and recent research by DRG suggesting that major stakeholders (i.e. hepatologists, endocrinologists and MCOs) disagree on the most important primary endpoints for Phase III evaluation of NASH1 guidance is needed to carefully shape trial design to provide relevant data to support the first drug approvals for NASH. On Monday afternoon, the FDA’s Stephanie O. Omokaro will discuss FDA regulatory considerations for clinical trial endpoints in order to facilitate development of pharmacotherapies intended to treat NASH.
Despite the lack of approved drugs for NASH, the recent focus on the disease has spawned a robust and diverse development pipeline across the industry, composed of drugs targeting a plethora of pathways. Later on Monday afternoon sees Novartis’ Eric Hughes discussing the benefits of targeting different pathways of NASH pathogenesis, including its metabolic, inflammatory, and fibrotic components., and how this could shape future development of mono- and combination therapies. On top of this, Hughes will speak to how future collaborations within industry and academia could provide the best treatment options for NASH.
Although liver biopsy is the gold-standard for the diagnosis of NASH, it is an expensive and invasive procedure that is not without its risks. On Tuesday morning, keynote speaker Dr. Quentin Anstee, Professor of Experimental Hepatology & Consultant Hepatologist at the Institute of Cellular Medicine, Newcastle, UK, will discuss the current non-invasive technologies available for the diagnosis of NASH (including “wet” biomarkers and imaging techniques) and will discuss the promising experimental approaches and their impact on clinical trials and clinical practice.
Stakeholders in NASH are sure to learn a lot about the road ahead for drug development from these speakers and we look forward to seeing when how their investments in time, effort, and finances will pay off.
For other highlights of the Global Engage NASH 2018 Congress, take a look at my colleague Tim Blackstock’s thoughts here.
For DRG’s assessment of the NASH market, please click here.
1Non-alcoholic Steatohepatitis | Access and Reimbursement | US: How Could the Positive and Negative Metabolic Effects of NASH Pipeline Therapies Influence U.S. Physicians and Payers in the Future?; scheduled for publication in Q2 2018.