New Phase II Trial Opens for Tesamorelin in NAFLD

A new Phase II clinical trial investigating tesamorelin for the treatment of non-alcoholic fatty liver disease (NAFLD) in HIV-negative patients has begun recruiting participants. The study represents a significant expansion of the clinical evidence base for tesamorelin beyond its currently approved indication.

Tesamorelin is a synthetic growth hormone-releasing hormone (GHRH) analogue currently approved by the FDA under the brand name Egrifta for the reduction of excess abdominal fat in HIV-infected patients with lipodystrophy. Previous studies in HIV-positive populations demonstrated that tesamorelin significantly reduces liver fat content and hepatic fibrosis markers, prompting interest in its potential application to the broader NAFLD population.

The new trial aims to enrol 180 participants across 12 US clinical sites. Eligible participants must have confirmed NAFLD with hepatic steatosis of at least 10% on MRI-PDFF (proton density fat fraction), a BMI between 28 and 40, and no HIV infection. Participants will be randomised to tesamorelin 2 mg daily or placebo via subcutaneous injection for 48 weeks.

The primary endpoint is the change in liver fat content as measured by MRI-PDFF at 48 weeks. Secondary endpoints include changes in liver stiffness (measured by MR elastography), ALT levels, components of metabolic syndrome, and visceral adipose tissue volume. The study is also collecting liver biopsy data in a subset of participants for histological assessment.

If positive, the results could support a supplemental NDA for an expanded indication, potentially making tesamorelin one of the few pharmacological treatments available for NAFLD in the general population. Current treatment for NAFLD is limited primarily to lifestyle modification, with resmetirom (Rezdiffra) being the only recently approved pharmacological option targeting a related liver condition.