Visirna Therapeutics' siRNA Drug VSA003 Granted Breakthrough Therapy Designation in China for Homozygous Familial Hypercholesterolemia

Jan 18, 2024 17:07 CST Updated 17:07

Visirna

siRNA Drug Developer

▎Edited by the WuXi AppTec content team

On January 18, the official website of the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) announced,Visirna's VSA003 Injection Proposed for Inclusion in Breakthrough Therapy Designation, Targeting Indication isAdultHomozygous Familial Hypercholesterolemia（HoFH）, can be used in HoFH patients on the basis of dietary therapy and other lipid-lowering treatments who need further reduction of LDL-C, or HoFH patients who are non-responsive or intolerant to other lipid-lowering treatments. Public information shows,VSA003 is aTreatment of Lipid Metabolism DisordersLiver-targeted small interfering RNA (siRNA)) Drug。

Screenshot source: CDE official website

HoFH is an autosomal dominant genetic disease caused by mutations in key genes responsible for the catabolism of low-density lipoprotein cholesterol (LDL-C) and has been included in China's First List of Rare Diseases. Due to extremely high LDL-C levels from birth, patients with HoFH face a significantly increased risk of developing atherosclerotic cardiovascular diseases, with systemic atherosclerosis occurring early and progressing rapidly., angina pectoris or myocardial infarction may occur during childhood and adolescence.。

Public data shows that, among the known types of gene mutations in HoFH patients,LDLRThe proportion of mutations is as high as 90%. This gene mutation affects the binding of LDL to LDLR, leading to impaired metabolic clearance of LDL and consequently increased LDL-C levels. The mechanisms of action of commonly used lipid-lowering drugs are mostly related to LDLR, but they have limited efficacy in treating HoFH.TargetedAngiopoietin-like Protein 3 (ANGPTL3)Small nucleic acid drugs are expected to reduce lipids through a non-LDLR-dependent mechanism,Is expected to become a new drug treatment option for HoFH patients.

VSA003 isVisirna is developingAn siRNA drug,By targeting the degradation of ANGPTL3 messenger RNA, increasing the clearance of LDL-C and triglycerides (TG), etc., thus having therapeutic potential for unmet medical needs in the cardiovascular field such as hypercholesterolemia, hypertriglyceridemia, and HoFH.

In July 2023, Visirna announced that the first subject had been dosed in the Phase 1 clinical trial of VSA003 injection in China. The principal investigator of the trial,Peking Union Medical College HospitalProfessor Chen RuiIt was previously stated in a press release that VSA003 has the potential to become the world's first innovative small nucleic acid drug targeting the ANG3 pathway, offering significant therapeutic potential for mixed dyslipidemia. As the principal investigator of VSA003’s first clinical study in China, I look forward to the smooth progress of the drug's clinical trials, which will provide more treatment options for patients with dyslipidemia in China.

In addition, according to Visirna's CEODr. Xiaoming ZouAs introduced in an earlier press release, VSA003, one of Visirna's key clinical pipelines in the cardiovascular field, has achieved positive clinical trial data, demonstrating good safety and therapeutic potential.

According to the official website of Visirna, the company was founded in 2022 and established a strategic partnership with Arrowhead Pharmaceuticals, a small nucleic acid drug company. Currently, the company's product pipeline includes three siRNA drugs targeting the cardiovascular and metabolic fields that are in the clinical research and development stage. This is alsoVisirnaThe second product has been proposed for inclusion in the breakthrough therapy category. Previously, the company's targetedAPOC3siRNA DrugsVSA001 InjectionAlready included in the breakthrough therapy category, proposed for developmentUsed forReduce Triglyceride Levels in Adult Patients with Familial Chylomicronemia Syndrome (FCS)。

References:

[1] Official website of the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) of China. Retrieved Jan 18, 2024, from https://www.cde.org.cn/main/xxgk/listpage/9f9c74c73e0f8f56a8bfbc646055026d

[2] Visirna's Small Nucleic Acid New Drug VSA003 Completes First Subject Dosing in China Phase I Clinical Trial. Retrieved July 8 , 2023, from https://mp.weixin.qq.com/s/T08sAVdlKSD04SGivHO9mw

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