Visirna Announces Completion of Patient Enrollment in Phase III Trial of siRNA Therapy VSA001 for Familial Chylomicronemia Syndrome in China

January 24, 2024DayWeierzhen Biotechnology (Shanghai) Co., Ltd. ("Visirna" or "Weierzhen") recently announced that the investigational new drug VSA001 Injection (hereinafter referred to as VSA001) has successfully completed the enrollment of over 36 patients in the Phase III clinical trial for familial chylomicronemia syndrome (FCS) in China. The company plans to complete all endpoint evaluations of the clinical trial by the first quarter of 2025, supporting subsequent new drug registration and marketing applications. VSA001 is expected to become the first approved drug for FCS in China.

This trial is a randomized, double-blind, placebo-controlled, multicenter Phase III clinical trial (CTR20231418/NCT05902598), aimed at evaluating the efficacy and safety of VSA001 in Chinese adults with FCS. Thirty-six patients with FCS will be randomly assigned to receive subcutaneous injections once every three months of either 25mg VSA001, 50mg VSA001, or a placebo. The primary endpoint of the trial is the percentage change in fasting serum triglycerides (TG) from baseline to the 10th month of treatment. After the randomized, double-blind phase of the study concludes, patients may opt to continue participating in the open-label phase for long-term evaluation.Observation and Follow-up on the Efficacy and Safety of Medication.

The principal investigator of the leading unit of this clinical trial, Professor Li Yong (Department of Cardiovascular Medicine, Huashan Hospital Affiliated to Fudan University), stated: "FCS is a rare disease that seriously affects the health status of patients and may be life-threatening. From adolescence, patients often suffer from recurrent episodes of...Acute PancreatitisThe suffering, accompanied by a significant increase in the risk of cardiovascular disease and diabetes. Even with strict dietary management and medication, it is not possible to effectively control the patient's triglyceride levels, and there is an urgent clinical need for safer and more effective TG-lowering drugs. The preliminary research results of VSA001 show a sustained, stable, and significant TG-lowering effect, as well as good safety, demonstrating great therapeutic potential and value for FCS. On the other hand, the unique therapeutic advantages of small nucleic acid drugs, such as long-lasting efficacy and longer dosing intervals, are also expected to improve the convenience and compliance of medication for patients with chronic diseases, making VSA001 highly suitable for including FCS among others.HypertriglyceridemiaThe treatment and management of chronic diseases are time-consuming. With the joint efforts of all researchers and the Visirna team, the enrollment of all patients in the VSA001 China FCS Phase III clinical trial was completed within half a year. Thanks to all research centers and researchers for their strong support of this clinical trial."

"FCS currently has no approved drugs, and patients' quality of life and even their health are severely affected. VSA001 is expected to become China's first approved drug for FCS. VSA001 has previously been granted Breakthrough Therapy designation by the CDE, fully demonstrating the regulatory authority's recognition of the drug's potential and value in treating FCS," said Dr. Xiaoming Zou, CEO of Visirna. "The successful and efficient completion of patient enrollment for the VSA001 Phase III clinical trial in China for FCS in such a short time is not only a significant milestone in Visirna's clinical research and development, but also gives us more confidence in bringing the first approved treatment to FCS patients as soon as possible. Going forward, Visirna will continue to work closely with various research institutions and clinical experts to complete the drug treatment, observation, and follow-up for this clinical trial to support the drug’s new drug application for marketing approval in China. Here, we sincerely thank all the patients and researchers involved in this study. In addition to the FCS indication, we are also simultaneously expanding to other indications, including severe hypertriglyceridemia, so that more Chinese patients with dyslipidemia can benefit from the novel and unique therapeutic advantages of VSA001."

AboutVSA001

VSA001 is a liver-targetedSmall interfering RNA(siRNA) drugs effectively and persistently silence the mRNA levels of apolipoprotein C3 (APOC3) to reduce the expression of APOC3 protein. This is followed by dual mechanisms, both dependent and independent of lipoprotein lipase (LPL), to effectively lower serum triglycerides (TG) and TG-rich lipoproteins (TRL) as well as their degradation remnants.

About Familial Chylomicronemia Syndrome (FCS)

FCS is a severe rare genetic disorder, with an estimated prevalence of approximately 1 in 1,000,000 according to incomplete statistics. It is typically caused by loss-of-function mutations or compound/double heterozygous mutations in various single genes (such as LPL, GPIHBP1, APOC2, APOA5, or LMF1). FCS often leads to extremely elevated fasting TG levels (above 880 mg/dL), and severe TG elevation can result in various clinical conditions and serious complications, including acute pancreatitis.Atherosclerosis, type 2 diabetes, obesity, hepatic steatosis, and chronic kidney disease, etc. Currently, there are no approved drugs specifically targeting FCS.

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 collaborates with internationally leading small nucleic acid drug companies.Arrowhead Pharmaceuticals(NASDAQ: ARWR) has established a long-term strategic partnership. The company's product pipeline currently includes three small interfering RNA drugs in clinical development targeting the cardiovascular and metabolic disease fields. The pipeline is in a leading position among similar competing products and adopts a collaborative clinical development and registration strategy between China and the United States, accelerating the registration and market launch of its products under research. At the same time, through comprehensive integration of internal and external resources, the company has formed an entire industrial chain layout from early-stage research and development, clinical development, localized siRNA drug production to commercialization.