The prevalence of liver disease has increased significantly in recent years. Nonalcoholic fatty liver disease (NAFLD) and nonalcoholic steatohepatitis (NASH) are the leading cause of liver disease.¹ It is estimated that 3 to 12 percent of adult Americans have NASH.
Theratechnologies intends to submit its Phase 3 study protocol to the United States Food and Drug Administration (FDA) and the European regulatory agencies in the fourth quarter of 2020. Subject to approval from the regulatory agencies, the enrollment of patients is planned for the first quarter of 2021. Patients will be treated for a period of 18 months. As per published regulatory guidelines, the primary endpoints will assess NAS score normalisation and absence of worsening of fibrosis stage, or fibrosis improvement ≥ 1 stage and no worsening of NAS.
Theratechnologies intends to use a new investigational formulation of tesamorelin, known as “F8”, for the Phase 3 trial in NASH. In addition, a supplemental Biologics License Application (sBLA) is expected to be filed with the FDA in early 2022 in HIV-associated lipodystrophy using a convenient, multi-dose pen injector currently being developed for this new formulation.
The effect of tesamorelin has recently been evaluated in patients with HIV and NAFLD following one-year treatment. 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.
¹ LaBrecque et al., Nonsalcoholic fatty liver disease and nonalcoholic steatohepatitis, World Gastroenterology Organization Global Guidelines, June 2012