Visirna Completes First Patient Dosing in China Phase II Trial of VSA006, a Novel siRNA Therapy for NASH

April 26, 2024Weierzhen Biotechnology (Shanghai) Co., Ltd. ("Visirna") announced that the first patient dosing has been successfully completed in the Phase II clinical trial of its investigational new drug VSA006 Injection (hereinafter referred to as VSA006) at the First Affiliated Hospital of Wenzhou Medical University. This trial is a randomized, double-blind, placebo-controlled, multi-center Phase II clinical trial (CTR20233245), aimed at evaluating the efficacy and safety of VSA006 in Chinese adult patients with nonalcoholic steatohepatitis (NASH).

Professor Wei Lai (Beijing Tsinghua Changgung Hospital), the principal investigator of the leading institution in this clinical trial, stated: "NASH is a serious metabolic-associated fatty liver disease that can lead to end-stage liver diseases such as cirrhosis and hepatocellular carcinoma, posing a severe threat to patients' health and increasing the global burden of liver diseases. However, there are currently very few drug options for NASH treatment. Therefore, we are very pleased to see that Visirna's innovative siRNA drug VSA006 has successfully completed the enrollment of its first patient in the Phase II clinical trial. VSA006 is an innovative siRNA drug targeting HSD17β13. The target is supported by substantial evidence from genome-wide association studies, features a novel mechanism of action, and benefits from the unique therapeutic advantages of small nucleic acid drugs, such as long-lasting efficacy and extended dosing intervals, making this product a potential next-generation drug treatment option for NASH. As the principal investigator of VSA006, I am honored to receive recognition from industry experts."Support and jointly advance the clinical research of this product in China. Thanks to Professor Zheng Minghua and the team for successfully completing the first dosing of a NASH patient in the VSA006 China Phase II clinical trial at the First Affiliated Hospital of Wenzhou Medical University. We also highly anticipate that, with the joint efforts of all clinical research participants and the Visirna team, the clinical trial will be rapidly advanced to bring the first small nucleic acid drug targeting NASH to Chinese patients.

Dr. Zou Xiaoming, CEO of Visirna, stated: "With changes in lifestyle and dietary structure, the prevalence of NASH in China continues to rise, and the demand for disease treatment is growing rapidly. Currently, there are no drugs specifically approved for NASH indications in China. VSA006 is the first small nucleic acid drug in China to enter Phase II clinical trials for NASH indications, and it also leads the domestic clinical pipeline targeting HSD17β13. Thanks to the strong support from various research institutions and clinical experts, the successful dosing of the first patient in the VSA006 Phase II clinical trial marks another significant milestone in Visirna's clinical research efforts, as well as a milestone for small nucleic acid drugs in China’s NASH drug development field. We look forward to the smooth progress of subsequent clinical trials, bringing better therapeutic options to Chinese NASH patients as soon as possible."

About Nonalcoholic Steatohepatitis (NASH)

NASH is a disease characterized by hepatocyte injury, hepatic steatosis, inflammatory cell infiltration, and even fibrosis, which is caused by factors other than alcohol. The incidence of the disease is rapidly increasing worldwide. Without appropriate intervention, NASH can easily progress to liver-related conditions such as cirrhosis and liver cancer, and may even lead to an increase in mortality from extrahepatic diseases. The pathogenesis of NASH is complex, posing significant challenges to new drug development, and there are currently no drugs approved globally for the treatment of NASH.

About VSA006

VSA006 is a small interfering RNA (siRNA) drug targeting hydroxysteroid 17-beta dehydrogenase 13 (HSD17β13) in hepatocytes. Genome-wide association studies have shown that loss-of-function mutations in HSD17β13 can reduce the risk of NASH patients progressing from hepatitis to liver fibrosis or liver cancer. The completed Phase I/II clinical trial evaluated the effects of VSA006 on normal healthy volunteers and patients with confirmed or clinically suspected NASH. Results indicated that VSA006 treatment was well-tolerated, with no treatment-related serious adverse events or discontinuations. VSA006 significantly reduced liver HSD17β13 mRNA and protein levels while effectively decreasing alanine aminotransferase (ALT) levels in patients.

About Visirna

Visirna is a small nucleic acid drug therapy company based in China with a global outlook, aiming to build a biopharmaceutical enterprise with comprehensive capabilities from research and development, production to commercialization. Established in 2022, Visirna has formed a long-term strategic partnership with Arrowhead Pharmaceuticals (NASDAQ: ARWR), an internationally leading small nucleic acid drug company. The company’s current pipeline includes three small interfering nucleic acid drugs targeting cardiovascular and metabolic diseases, all at the clinical development stage. The pipeline holds a leading position among similar competing products and adopts a US-China collaborative clinical development and registration strategy to accelerate the registration and market launch of its products under development. Through the comprehensive integration of internal and external resources, the company has established an entire industrial chain layout from early-stage research, clinical development, localized siRNA drug production to commercialization.