The Race to Rescue Livers: Finding Treatments for NASH

By Kathleen Hoffman, PhD, MSPH

There is a race to develop drugs to treat non-alcoholic steatohepatitis, commonly called NASH. NASH, an aggressive form of liver disease causing inflammation and scarring, already affects 3-12% of the entire US population.1,2 What’s the incentive? There’s currently no approved treatment, and it can be fatal.3 The current standard of care is lifestyle changes.

NASH is a more serious form of NAFLD (non-alcoholic fatty liver disease) and can result in cirrhosis.4 Related to obesity and diabetes, but not to alcohol consumption, NAFLD already affects 31% of Americans.5,6 NASH is also more prevalent and more severe in Latinx populations, a demographic that is also expected to increase.7 One research paper estimates that NASH will affect 27 million US residents by 2030.8 There is an ever increasing need for treatments due to the expansion of the population with this disease.

Inspire members want people to know that their liver disease is not due to alcoholism.

I need and want to get this out of my system. We are all not alcoholics you don’t need a drink for your liver to pack up.

A “silent” disease, NASH progresses over years for decades, presenting few or no symptoms until the liver is significantly scarred and damaged.9 It’s also unclear how to identify which subset of patients with NAFLD might progress into NASH.10 NASH is the fastest-growing reason for a liver transplant in the US.11

90+ Treatments in the Pipeline

According to a recent article reviewing the status of the NASH treatment pipeline, there are over 90 treatments in development. Some are showing promise despite the disappointing 2020 FDA rejection of obeticholic acid (OCA, Intercept Pharmaceuticals). Using a variety of mechanisms of action, six drugs are in Phase 3 trials, with 37 more in Phase 2.12,13

FDA makes NASH treatments a priority

The FDA’s Center for Drug Evaluation and Research (CDER) reaffirmed that drugs for NASH are a national priority with a webcast on January 29, 2021. The webcast clarified the eligibility criteria for Phase 2 and 3 NASH treatment trials. While the traditional drug approval pathway requires clinical outcome trials, NASH is different. The FDA views it as infeasible and unethical to wait for clinical outcome evidence because the progress of the disease is so slow and results in irreversible damage.

NASH treatments that focus on stages where inflammation (steatohepatitis) is present but not cirrhosis are eligible for “accelerated approval,” which accepts surrogate endpoints in Phase 3, such as tissue samples from participant liver biopsies. In fact, for early Phase 2 (proof of concept) trials, potential participants don’t even have to undergo biopsies; they can be enrolled based on their known NASH risk factors or common test evidence of NAFLD or NASH, including biomarkers like aminotransferase or total bilirubin.14

Inspire members and NASH

So many treatments in competitive development means there’s competition for enrolling trial participants, too. Steatohepatitis, the condition that serves as the gating factor for participant eligibility for Phase 2 NASH clinical trials, is being discussed in over 20 Inspire communities, reflecting the prevalence of common comorbidities including obesity, diabetes, metabolic syndrome, and cardiovascular disease.15

I am new to the site and after a biopsy have been diagnosed with Stage 2 NASH and having problems with high blood sugar (300-400’s) for diabetes even though I’m on an insulin pump. Any ideas on the direction I should take with my diet to lose weight and improve the NASH and diabetes?

It’s not just found in common conditions; NASH is also represented in niche communities, rare diseases, and sometimes among family members.

My daughter now 22 yo dx at 20 with stage 3 NASH now wondering about pregnancy with this disease.

With over 26,000 posts to date and estimated audience size of over 12,000 members, NASH, NAFLD, and its consequences are widely discussed on Inspire. There are opportunities of different kinds available to interact with the Inspire community, including educating an interested population about an insidious disease, learning about the health journey of people with liver disease, obtaining real world voice impacts of this disease on daily life, and a ready population for participating in relevant clinical trials. Finding treatments for NASH is not just a priority for 90+ companies and the FDA — it’s a health concern for more and more people every day.

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10“Regulatory Perspectives for Development of Drugs for Treatment of NASH.”FDA CDER Presentation. January 29, 2021.