SRSD-101

Data presented at American Society of Hematology annual meeting SAN DIEGO, CA, USA & SHANGHAI, China I December 09, 2024 I Sirius Therapeutics today announced promising preliminary data from its Phase 1 first-in-human clinical trial of SRSD107, a next generation siRNA therapeutics under clinical development for the prevention and treatment of thromboembolic disorders, such as myocardial infarction, ischemic stroke, and venous thromboembolism. The trial data were presented during a poster session at the 66th American Society of Hematology (ASH) Annual Meeting and Exposition, held in San Diego, CA. SRSD107 is designed to inhibit Factor XI (FXI), a protein in the coagulation pathway that has shown potential to reduce thrombosis without significantly increasing the risk of bleeding, a major liability of current drugs. “In this trial, SRSD107 was safe and well tolerated, with pharmacokinetic parameters consistent with a typical siRNA product,” said Dr. Patrick Yue, Sirius’ Chief Medical Officer. “We are encouraged by the marked, prolonged reduction in FXI antigen and activity, and increase in clotting, or thromboplastin time, that are consistent potent anticoagulation over sustained periods and reduced bleeding risk respectively. The trial provides a strong foundation for Phase 2 clinical studies.” Dr. Qunsheng Ji, Sirius’ Chief Executive Officer added that “presentation of these findings at ASH 2024 underscores Sirius Therapeutics’ commitment to advancing innovative treatments for thromboembolic disorders.” The study was a single-site, randomized double-blind, placebo controlled study to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of subcutaneously administered SRSD107 in 40 healthy subjects. Five cohorts, each consisting of eight subjects evaluated eight doses of SRSD107, each receiving either a single SRSD107 dose or placebo. In the trial, SRSD107 was safe and well-tolerated. Significant changes from baseline in PD biomarkers were observed, with maximal reductions in FXI antigen and activity > 90% and an aPTT (thromboplastin time) increase of > 100% (i.e., an aPTT ratio > 2.0) at the highest doses tested. The pharmacodynamic effects were durable, with FXI antigen and activity levels remaining suppressed for more than 16 weeks after dosing. The study is titled, “A Phase 1, Single Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Subcutaneously Administered SRSD107 in Healthy Subjects. For more information on the study and our ongoing clinical trials, please visit Sirius Therapeutics’ website ( www.siriusrna.com ) or here . About Thromboembolic Disorders Thrombosis, or blood clot formation, is the common underlying mechanism of most cases of myocardial infarction, ischemic stroke, and venous thromboembolism. According to a study in The Lancet of regional and global mortality rates, thromboembolic disorders are estimated to cause as many as 1 in 4 deaths worldwide. About SRSD107 SRSD107 is a novel double-stranded small interfering ribonucleic acid (siRNA). Developed by Sirius Therapeutics, SRSD107 specifically targets human coagulation factor XI (FXI) mRNA and inhibits FXI protein expression, thereby blocking the intrinsic coagulation pathway and promoting anticoagulant/anti-thrombotic effects. SRSD107 has been engineered for the potential to enable once or twice-a-year dosing. About Sirius Therapeutics Sirius is an innovative, clinical stage biotech company developing next generation siRNA therapy for global markets. We are dedicated to translating siRNA technology into transformative medicine for patients with chronic diseases. Our most advanced products are SRSD107 for the treatment of thromboembolic disorders, and SRSD101 for the treatment of dyslipidemia. Founded in 2021 by a world-class leadership team and investors, Sirius has established an innovative discovery center in the United States and translational medicine center in China. Sirius has raised nearly US$100 million funding to date from OrbiMed, Creacion Ventures, Hankang Capital, Delos Capital, and the leadership team. SOURCE: Sirius Therapeutics

NEWS 2024年12月10日，中国上海和美国圣地亚哥：靖因药业（上海）有限公司（靖因药业），今天宣布，公司在第66届美国血液学会（ASH）年会上公布了SRSD107的临床一期试验的最新数据。 SRSD107是一种特异性靶向抗凝血因子XI的新一代siRNA疗法，用于预防和治疗血栓栓塞性疾病，如心肌梗死、缺血性中风和静脉血栓栓塞。 靖因药业首席医学官于沛川博士表示：“在此项初步研究中，SRSD107表现出良好的安全性和耐受性，其药代动力学参数特征符合典型的小干扰核糖核酸（siRNA）产品。我们对此项研究结果感到非常鼓舞，包括FXI 抗原及FXI活性显著且持久的抑制以及活化部分凝血活酶时间（aPTT）的延长。这表明SRSD107将更符合新型抗凝剂的需求特点，可在持续长时间内保持强效抗凝作用并降低出血风险。该试验结果为二期临床研究奠定了坚实的基础。”  靖因药业首席执行官冀群升博士补充道：“在 2024 年美国血液学会（ASH）会议上展示这些研究结果，突显了靖因药业致力于推进血栓栓塞性疾病创新疗法的决心。”  该试验是单中心、随机双盲、安慰剂对照研究，旨在评估皮下给药SRSD107在40 名健康受试者中的安全性、耐受性、药代动力学以及药效学特征。研究共有五个队列，每个队列由 8 名受试者组成。在该研究中，SRSD107安全性和耐受性良好，并观察到药效生物标志物相较基线的显著变化。在最高给药剂量下，FXI 抗原及FXI活性的最大降幅超过90%，aPTT增幅超过 100%（即aPTT 比值＞ 2.0）。单次给药后的药效效应持久，维持FXI抗原及FXI活性近90%抑制状态时长超16周。 这项研究的标题是“一项评价SRSD107皮下注射给药在健康受试者中的安全性、耐受性、药代动力学和药效学的随机、双盲、安慰剂对照、单剂量递增、I期临床研究”。有关该研究和我们正在进行的临床试验的更多信息，请访问Sirius Therapeutics的网站（www.siriusrna.com）。 关于血管栓塞 血栓形成是血管内限制血液流动的血凝块，可发生在动脉或静脉循环中，是大多数心肌梗死、缺血性脑卒中和静脉血栓栓塞症（VTE）的共同病理基础。全球有四分之一的人死于血栓栓塞引发的疾病。 关于SRSD107注射液 SRSD107注射液是一款靖因药业拥有自主知识产权的双链小干扰核酸（siRNA）药物。通过特异性肝靶向人凝血因子XI（FXI ）mRNA，抑制FXI的蛋白表达，阻断内源性凝血途径的激活，从而达到抗凝血作用。临床前试验数据显示，单次皮下注射SRSD107，可降低外周血FXI浓度近100%，且持续时间可长达半年，同时未见出血。SRSD107兼具强效持久作用和良好的安全性，有望成为潜在的同类首创（First-in Class）和同类最佳（Best-in-Class）新一代更安全的抗凝药物。 关于靖因药业 靖因药业 (Sirius Therapeutics) 是一家创新生物技术公司，以人类健康福祉为使命，聚焦新一代核酸创新疗法在心血管代谢疾病领域的应用与开发，致力成为颠覆慢病防治的领军者。目前已进入临床开发阶段的产品包括用于治疗血栓栓塞性疾病的SRSD107和用于治疗血脂异常的SRSD101。 成立于2021年，由国际卓越的管理团队和全球知名医疗健康投资机构孵化。公司采取国际化的战略定位，汇聚中美两地在小核酸疗法领域的人才和资源的优势，建立了以美国为源头创新发现中心，中国为全球转化医学中心的布局。利用其小核酸药物研发核心技术平台，开发了多个具有同类首创或同类最佳潜质的差异化全球竞争优势的产品管线。公司已累计融资近1亿美元。