｜Edited by the Content Team of Sirius TherapeuticsMarch 202520Day，San Diego and Shanghai — Sirius Therapeutics announced that the National Medical Products Administration (NMPA) has approved its Investigational New Drug (IND) application to initiate the first-in-human Phase I clinical trial of SRSD216. SRSD216 is an innovative small interfering nucleotide (siRNA) therapy designed to treat hyperlipoproteinemia(a).Numerous genetic and epidemiological studies have shown an association between elevated lipoprotein(a) levels and atherosclerotic cardiovascular disease (ASCVD). SRSD216 demonstrated significant and sustained pharmacodynamic effects in preclinical studies.The study's effects and favorable safety profile indicate that this novel siRNA therapy holds great promise for the prevention of ASCVD.
Curt Bradshaw, Ph.D., Chief Scientific Officer of Sirius Therapeutics, stated, "SRSD216 is the third siRNA product to enter clinical development since the company's establishment in 2021. This milestone further validates Sirius Therapeutics' exceptional platform capabilities in discovering and efficiently developing next-generation siRNA therapies."Dr. Qunsheng Ji, CEO of Sirius Therapeutics, added: "The approval of SRSD216 by the NMPA is a crucial step in its development process. Achieving this significant milestone is not only the result of our team's continuous hard work but also part of Sirius Therapeutics' overall strategy to develop innovative therapies for cardiometabolic diseases. The company is now preparing to initiate Phase I clinical trials to further evaluate the safety and efficacy of SRSD216 in human subjects."About ASCVD and Hyperlipoproteinemia(a)
Atherosclerotic cardiovascular disease (ASCVD) is the most common cause of death worldwide. Dyslipidemia is considered a key factor in the development of ASCVD. Although low-density lipoprotein cholesterol (LDL-C) has always been regarded as the primary lipid indicator and intervention target for ASCVD, numerous studies have shown that lipoprotein(a) [Lp(a)] is an independent risk factor for ASCVD, unaffected by age, diet, or exercise. Currently available lipid-lowering drugs have limited efficacy in reducing Lp(a). Therefore, there is a clinical need for drugs that directly target Lp(a).with enormous unmet needs.SRSD216 Injection is a novel double-stranded small interfering ribonucleic acid (siRNA). It specifically modulates the LPA gene, reducing hepatic Apo(a) production and lowering circulating Lp(a) levels. Preclinical in vivo studies have shown that a single dose reduced Lp(a) levels by nearly 100%, with effects lasting over six months, and no significant safety events were observed.Sirius Therapeutics is a clinical-stage biotechnology company dedicated to advancing human health and well-being. Focused on the global development of next-generation nucleic acid therapies for cardiometabolic diseases, the company aims to become a leader in revolutionizing the prevention and treatment of chronic conditions. Its current pipeline includes SRSD107, which has entered clinical development for thromboembolic disorders, and SRSD101 for dyslipidemia.
