Table of Contents

• Trial overview
• Impaired renal function and suspected MASH
• Alport syndrome study
• What was measured in the studies
• Who the studies focused on

Trial overview

Two Phase 2 ^[1][2] interventional studies were listed for Vonafexor, and both were marked completed.^[1][2] These studies looked at different patient groups: one with impaired renal function and suspected MASH, and one with Alport syndrome at risk of progression.^[1][2]

Impaired renal function and suspected MASH

The study with NCT ID 2023-509192-16-00 enrolled 50 people and was completed.^[1] It studied people with impaired renal function and suspected MASH, which means a liver condition with fat and inflammation, along with reduced kidney function.^[1] The brief summary said the goal was to determine the effect of Vonafexor on renal function in people with suspected MASH and mild to moderately reduced GFR.^[1]

The primary outcome was the change from baseline in mGFRiohexol and eGFRcreat at week 16.^[1] In simple terms, the study checked whether kidney filtering changed after treatment compared with the start of the study.^[1] The listed interventions included Vonafexor by mouth at 25 mg and 100 mg, along with rosuvastatin and iohexol used for measurement.^[1]

Alport syndrome study

The study with NCT ID 2023-509638-20-00 enrolled 24 patients and was also completed.^[2] It focused on patients with Alport syndrome who were at risk of progression.^[2] The brief summary said the purpose was to assess the safety and tolerability of Vonafexor both during treatment and after treatment stopped.^[2]

The primary outcome tracked the number of treatment-emergent adverse events from the first dose until 2 weeks after the last dose.^[2] It also measured changes in physical examinations, vital signs, laboratory variables, and lipid profile compared with baseline, which is the starting point before treatment begins.^[2] The intervention listed for this study was oral Vonafexor 100 mg.^[2]

What was measured in the studies

The kidney-focused study measured GFR, which is a way to see how well the kidneys filter blood.^[1] It used both a measured method with iohexol and an estimated method based on creatinine.^[1] The Alport syndrome study focused more on safety checks, including adverse events, physical exams, vital signs, laboratory values, and blood fats.^[2]

• Baseline means the starting point before treatment or study procedures begin.^[2]
• Vital signs are basic body measurements such as blood pressure and pulse.^[2]
• Laboratory variables are blood or urine test results that help show how the body is working.^[2]
• Lipid profile is a blood test that measures fats such as cholesterol.^[2]

Who the studies focused on

These trials were not broad studies of all patients with kidney or liver disease.^[1][2] They focused on specific groups: people with suspected MASH and reduced kidney function, and people with Alport syndrome at risk of getting worse.^[1][2] This makes the results more relevant to those exact patient groups, but not necessarily to everyone with similar symptoms.^[1][2]