A Phase 2 Clinical Trial to Evaluate the Efficacy and Safety of VSA006 in Chinese NASH Patients

A Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase II Clinical Trial to Evaluate the Efficacy and Safety of VSA006 Injection in Chinese Adult Patients With Nonalcoholic Steatohepatitis (NASH)

Human genetic studies have shown that loss of function (LOF) mutations in HSD17β13 gene have a protective effect on the progression of alcohol-related and non-alcohol-related liver diseases, such as NASH, without significant adverse phenotypes.

VSA006 is a siRNA drug targeting HSD17β13 mRNA in the liver and reduce the protein level of HSD17β13. Based on phase 1 study results in healthy volunteers and NASH/suspected NASH patients, this phase 2 study is designed to evaluate the efficacy, safety, PK profiles and immunogenicity of VSA006 in Chinese NASH patients.

Inclusion Criteria:

• body mass index (BMI) of 24-35 kg/m2 ;
• NASH patients confirmed by liver histopathology: NAS score is ≥ 4 and CRN fibrosis is F2 or F3 ;
• At screening, ALT is > ULN;
• At screening, the liver fat content measured by MRI-PDFF is ≥ 8%;
• Weight change < 5% at least 3 months prior to screening;
• For patient with T2DM, the hypoglycemic agents and HbA1c is stable

Exclusion Criteria:

• Pregnant or lactating women;
• Previous diagnosis of alcoholic liver disease or hepatitis/liver disease due to other causes;
• Previous or current diagnosis of cirrhosis or decompensated cirrhosis;
• Previous or current diagnosis of hyperthyroidism, hypothyroidism, or other diseases that can lead to fatty degeneration of liver;
• Participants diagnosed with type 1 diabetes, or with unstable type 2 diabetes
• Participants who cannot receive an MRI examination;