Visirna Therapeutics Advances VSA003, a Potential First-in-Class siRNA Therapy for HoFH, into CDE Breakthrough Therapy Designation

On January 18, the CDE proposed to include VSA003 Injection from Weierzhen Biotechnology (Shanghai) Co., Ltd. ("Visirna") in the breakthrough therapy designation category for the treatment of homozygous familial hypercholesterolemia.

Source of the image: CDE official website

Potential Global First
Positive Clinical Data

Homozygous Familial Hypercholesterolemia (HoFH) is an autosomal recessive genetic disorder caused by homozygous or compound heterozygous mutations in key genes responsible for the catabolism of low-density lipoprotein cholesterol (LDL-C). This disease represents the most severe and rare form of familial hypercholesterolemia, with a clinical predisposition to early-onset atherosclerotic cardiovascular disease.

VSA003 is a liver-targeted siRNA drug for the treatment of lipid metabolism disorders, which mainly works by degrading angiopoietin-like protein 3 (ANGPTL3) mRNA, thereby enhancing the clearance of LDL-C and triglycerides (TG). According to Visirna, VSA003 is expected to become the world's first innovative small nucleic acid drug targeting the ANGPTL3.

In 2023, Visirna officially launched the Phase I clinical trial of VSA003, aiming to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of the drug in healthy adult volunteers in China. In addition, Visirna also mentioned that the clinical trial data of VSA003 overseas was positive, demonstrating good safety and therapeutic potential.

Source of the image: Drug Clinical Trial Registration and Information Disclosure Platform

The inclusion of VSA003 injection in the CDE's breakthrough therapy designation is expected to further accelerate the clinical research process.

Deep Cultivation in the Small Nucleic Acid Field
Three Heavyweight siRNA

Visirna is a biopharmaceutical company focusing on the development of small nucleic acid therapies. It was jointly founded by Arrowhead Pharmaceuticals ("Arrowhead") and Vivo Capital in April 2022. Meanwhile, Visirna has established a long-term cooperative relationship with Arrowhead.

Currently, including VSA003, Visirna has three siRNA drugs in clinical stages.

Image Source: Visirna Official Website

VSA001

VSA001 is a liver-targeted siRNA drug for the treatment of lipid metabolism disorders. It primarily works by significantly and persistently silencing apolipoprotein C3 (APOC3) mRNA to reduce APOC3 protein expression levels. Through both lipoprotein lipase (LPL)-dependent and -independent pathways, it achieves a significant reduction in serum triglycerides (TG), TG-rich lipoproteins (TRL), and remnant lipoprotein levels after lipolysis. VSA001 is currently being studied for the treatment of familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia. In September 2023, VSA001 was designated as a breakthrough therapy by the CDE for the treatment of FCS.

Source of the image: CDE official website

VSA006

VSA006 is an siRNA drug targeting hydroxysteroid 17-beta dehydrogenase 13 (HSD17β13) in hepatocytes. Studies have shown that this drug can significantly reduce liver HSD17β13 mRNA and protein levels while effectively decreasing alanine aminotransferase (ALT) levels in patients. In September 2023, the VSA006 injection successfully obtained the CDE's implied permission for clinical trials to conduct Phase 2 clinical trials in China for adult patients with NASH. Currently, Visirna has completed the registration on the Drug Clinical Trial Registration and Information Disclosure Platform.

Source of the image: Drug Clinical Trial Registration and Information Disclosure Platform

Conclusion

According to the official website, while continuously accelerating the clinical development process of three first-in-class products, Visirna will further enrich its clinical R&D pipeline, including the cardiovascular and metabolic fields. In the future, Visirna is expected to launch more重磅 first-in-class products.

Editor: Muyan

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