CVI Pharmaceuticals Announces First Patient Dosed in Phase II Clinical Trial of CVI-LM001, a First-in-Class Oral Small Molecule PCSK9 Inhibitor for Hyperlipidemia

California, USA and Shanghai, China—August 19, 2020-- CVI Pharmaceuticals Limited (hereinafter referred to as “CVI”), a global biopharmaceutical company focused on the research and development of innovative drugs for cardiovascular and hepatic metabolic diseases, today announced that the first patient with hyperlipidemia has been enrolled in its Phase II clinical trial of the investigational drug CVI-LM001 in China. CVI-LM001 is a Class 1 novel small-molecule lipid-lowering drug independently developed by CVI, and it is the world’s first oral small-molecule PCSK9 synthesis inhibitor to enter Phase II clinical studies.

This multicenter, Phase II clinical study (ClinicalTrials.gov NCT04438096) of CVI-LM001 tablets in subjects with hypercholesterolemia, designed as a randomized, double-blind, multi-dose, placebo-controlled trial to evaluate efficacy and safety, aims to further assess the safety and efficacy of oral CVI-LM001 in Chinese subjects with hyperlipidemia.

CVI has successfully completed the single-ascending dose, food-effect, and multiple-ascending dose tolerance and pharmacokinetic studies of CVI-LM001 tablets in healthy subjects, as well as an exploratory study on the mechanism of pharmacodynamic action in patients with hyperlipidemia (Phase Ib).

These study results demonstrated the excellent safety, tolerability, and favorable pharmacokinetic profile of CVI-LM001, and provided key mechanistic proof-of-concept data showing reduced levels of PCSK9 protein in the blood of both healthy subjects and individuals with hyperlipidemia. Meanwhile, a Phase Ib exploratory efficacy study conducted in subjects with hyperlipidemia showed that oral administration of CVI-LM001 led to a significant reduction in blood LDL-C levels compared with the placebo group.

CVI-LM001, independently developed by CVI, is a Class 1 innovative lipid-lowering drug and the first oral small-molecule PCSK9 synthesis inhibitor worldwide to enter Phase II clinical trials. Extensive studies have shown that statins increase PCSK9 gene transcription and upregulate PCSK9 expression, thereby attenuating their own lipid-lowering efficacy.

In contrast to statins, CVI-LM001 inhibits PCSK9 gene expression; therefore, it exhibits a synergistic therapeutic effect with statins in terms of mechanism of action, thereby achieving superior clinical outcomes in patients who fail to reach treatment targets with statin therapy or who are intolerant to statins.

Currently, all lipid-lowering drugs targeting PCSK9 on the market are antibodies administered via injection, which poses inconvenience and high costs for patients with hyperlipidemia. CVI-LM001 is a globally leading small-molecule oral drug that lowers LDL-C by targeting PCSK9, offering greater convenience in administration. The development and commercialization of CVI-LM001 will provide new and improved treatment options for patients with hyperlipidemia.

CVI Therapeutics (CVI) is a biopharmaceutical company currently in the clinical research stage, dedicated to the development of innovative drugs for cardiovascular and hepatic metabolic diseases. The company has established modern R&D centers, as well as teams for preclinical drug development and clinical research management, in California, USA, and Shanghai, China. Its R&D projects have received multiple government grants, including funding from the National “11th Five-Year Plan” Major New Drug Creation Special Fund (2011), the National “12th Five-Year Plan” Major New Drug Creation Special Fund (2014), the Technology Innovation Fund, Key Scientific and Technological Breakthrough Projects, and High-Tech Enterprise Incubation Programs.

CVI’s operations management team comprises clinicians with extensive experience in hepatic metabolism and cardiovascular diseases, biologists with over two decades of specialized expertise in lipid metabolism, and clinicians with broad professional knowledge in the treatment of dyslipidemia. The team possesses comprehensive experience spanning product development, clinical trials, and new drug approval. The company’s advisory board consists of internationally renowned academic leaders in cardiovascular disease research, executives responsible for preclinical and clinical development of innovative drugs in hepatic metabolism and cardiovascular diseases at major pharmaceutical companies overseas and in China, as well as a team of distinguished clinicians.