Visirna's siRNA Therapy VSA006 Receives CDE Approval for Phase 2 Clinical Trial in NASH

September 26, 2023DayThe Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) recently announced on its official website that the small nucleic acid Class 1 innovative drug VSA006 Injection (hereinafter referred to as VSA006), developed by Weierzhen Biotechnology (Shanghai) Co., Ltd. ("Weierzhen" or "Visirna"), has received tacit approval for clinical trials. It will be used to conduct a Phase 2 clinical trial in China targeting adult patients with nonalcoholic steatohepatitis (NASH). VSA006 is the third small nucleic acid drug developed by Visirna to be approved for clinical trials.

VSA006 is a small interfering RNA (siRNA) drug targeting hydroxysteroid 17-beta dehydrogenase 13 (HSD17β13) in hepatocytes. This product has completed safety and pharmacokinetic evaluations in Phase 1/2 clinical trials involving healthy subjects and patients diagnosed with or clinically suspected of having NASH (including patients recruited from Hong Kong). The upcoming randomized, double-blind, placebo-controlled Phase 2 clinical trial in China will assess the safety and efficacy of VSA006 in Chinese patients with NASH.

"Since its establishment, Visirna has successfully launched three"The first-in-class small nucleic acid drug has advanced to the clinical trial stage in China, establishing a leading and highly competitive product pipeline in the cardiovascular and metabolic fields," said Dr. Xiaoming Zou, CEO of Visirna. "We are supporting this IND and subsequent clinical trials with locally produced VSA006 injections, an important milestone since Visirna established its local small nucleic acid drug production capabilities. Visirna looks forward to collaborating closely with investigators to bring more treatment options to patients with NASH as soon as possible."

About Nonalcoholic Steatohepatitis (NASH)

NASH is a disease characterized by hepatocyte injury, hepatic steatosis, inflammatory cell infiltration, and even fibrosis, excluding alcohol-related factors. 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 globally, no drugs for NASH indications have been approved.

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 diagnosed with or clinically suspected of having NASH. Results indicate 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 lowering patient alanine transaminase (ALT) levels.

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 in research and development, production, and commercialization. The company was founded in 2022 and has established a long-term strategic partnership with Arrowhead Pharmaceuticals (NASDAQ: ARWR), a leading international small nucleic acid drug company. Currently, the company's product pipeline includes three small nucleic acid drugs in clinical development targeting cardiovascular and metabolic diseases.

The company's existing pipeline is in a leading position among similar competing products. Its research targets are supported by clear genomic and biological evidence and utilize Arrowhead Pharmaceuticals' proven chemical modification and delivery technology platform. The company adopts a China-US collaborative clinical development and registration strategy, which helps accelerate the registration and market launch of its research products. At the same time, through the comprehensive integration of internal and external resources, it forms an entire industry chain layout from early-stage R&D, clinical R&D, localized production to commercialization.