Hepatology Patient Need
The prevalence of liver disease has increased significantly in recent years. According to the National Institute of Diabetes and Digestive and Kidney Diseases of the National Institutes of Health, it is estimated that 20 percent of Americans with Non-Alcoholic Fatty Liver Disease (NAFLD) have Non-Alcoholic Steatohepatitis (NASH). It is believed that 3 to 12 percent of adult Americans have NASH.1
NAFLD is an umbrella term for a spectrum of liver conditions that begin with a build-up of hepatic fat, which can set the stage for inflammation that may promote scarring known as fibrosis. Over time, fibrosis can progress to potentially fatal cirrhosis and even a form of liver cancer called hepatocellular carcinoma.
Usually, NAFLD and NASH are silent diseases with few or no symptoms. A patient may not show symptoms even if they develop cirrhosis due to NASH.
There is currently no approved treatment for NAFLD and NASH in North America and Europe.
The initiation of a Phase 3 clinical trial evaluating tesamorelin for the treatment of NASH is on track to begin in the third quarter of calendar year 2021. In respect of recommendation made by the FDA, Theratechnologies has confirmed a date to meet with the agency and discuss the proposed trial design and protocol. The Company received a “Study May Proceed” letter for the Phase 3 trial from the FDA in January 2021 and we have retained the services of a global, large-scale CRO with experience in implementing large and late-stage clinical trials to assist with the execution of the trial.
A study on the effect of tesamorelin in patients with HIV and NAFLD following one-year of treatment was published in The Lancet HIV in 2019. This study demonstrated that the daily use of tesamorelin causes a significant decrease in hepatic fat (37%) compared to the control group. In addition, 35% of patients treated with tesamorelin demonstrated hepatic fat normalization, compared to only 4% in the control group. This study also suggests potential benefits on the prevention of fibrosis.
After 10 years of real-life experience, the safety profile of tesamorelin in HIV patients with lipodystrophy is well established. Based on current scientific evidence showing a reduction in liver fat and delayed progression of liver fibrosis in patients with HIV infection and NAFLD or NASH, combined with robust intellectual property, a new investigational formulation and the development of a multi-dose pen injector, tesamorelin is believed to be a potential strong candidate for the treatment of NASH in the general population.