Visirna Announces Breakthrough Therapy Designation Granted for VSA003 for Treatment of Patients with Homozygous Familial Hypercholesterolemia (HoFH)

SHANGHAI, CHINA – January 29th, Visirna announced that the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) granted Breakthrough Therapy Designation for VSA003 for the treatment of patients with Homozygous Familial Hypercholesterolemia (HoFH). Patients with HoFH are exposed to extremely high levels of LDL-C, leading to a significantly increased risk of atherosclerotic cardiovascular disease (ASCVD), angina or myocardial infarction. The extreme elevation of LDL-C in the majority of HoFH patients is due to the functional loss or defect of low-density lipoprotein receptors (LDLR). The mechanisms of existing lipid-lowering drugs are mostly LDLR-dependent, resulting in limited efficacy in HoFH patients. VSA003 is a small interfering RNA (siRNA) drug that targets angiopoietin-like protein 3 (ANGPTL3). Through a dual lipid-lowering mechanism involving both LDLR-independent and LDLR-dependent pathways, it effectively reduces LDL-C levels in HoFH patients. "We are grateful to the CDE for recognizing the potential clinical value of VSA003 and granting it breakthrough therapy designation," said Dr. Zou Xiaoming, CEO of Visirna. "This is the second innovative siRNA drug from Visirna with breakthrough therapy designation, following VSA001. Currently, Visirna is actively preparing for phase 3 clinical trials of VSA003 in HoFH and other dyslipidemia indications. Through close collaboration with research institutions and clinical experts, we aim to expedite the clinical development of VSA003 in China, providing better treatment options for Chinese patients with HoFH and dyslipidemia." The Breakthrough Therapy Designation review policy is designed to facilitate the development and expeditious review of novel medicines that are intended for the prevention or treatment of serious, life-threatening diseases or diseases that severely impact the quality of life for which there is no existing treatment, or where sufficient evidence indicates advantages of the novel drug over currently available treatment options. Drugs granted Breakthrough Therapy Designations receive priority communications and guidance from the CDE to promote and expedite the drug development process. HoFH is a rare autosomal dominant genetic disease. Patients with HoFH are exposed to extremely high LDL-C levels from birth, significantly increasing the risk of ASCVD. The onset of systemic ASCVD is early and rapid in these patients, with angina or myocardial infarction occurring, leading to death before the age of 20 to 30. It poses significant risks of mortality and disability, severely jeopardizing the patients' life and health. The prevalence of HoFH is approximately 1/300,000 to 1/160,000 and has been included in China's "First Catalog of Rare Diseases". When statins fail to achieve LDL-C targets, non-statin lipid-lowering drugs such as cholesterol absorption inhibitors (e.g., ezetimibe) or proprotein convertase subtilisin/kexin type 9 inhibitors (PCSK9 inhibitors) can be used in combination. Research indicates that approximately 85-90% of HoFH patients have extremely high LDL-C levels due to LDLR functional loss or defect. The mechanisms of existing lipid-lowering drugs are mostly LDLR-dependent, resulting in limited efficacy and safety in HoFH patients. VSA003 is a siRNA drug that specifically targets ANGPTL3 mRNA in liver cells, effectively reducing the production of ANGPTL3 protein. This leads to a decrease in ANGPTL3 protein levels in the liver and circulation, thereby reducing blood LDL-C level through multiple mechanisms and achieving LDL-C reduction through LDLR-independent pathways. Visirna was founded in 2022 in a strategic partnership with Arrowhead Pharmaceuticals (NASDAQ: ARWR). Based in China, with a global vision, Visirna aims to be a leader in the research and development of siRNA therapeutics. The current product pipeline includes three siRNA programs in clinical development targeting cardiovascular and metabolic diseases. Through the comprehensive integration of internal and external resources, Visirna has established a full industry chain capabilities covering drug discovery, clinical development, local production, and commercialization. For more information, please visit www.visirna.com.

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