Sirius Therapeutics claims breakthrough results in first-in-human trial of SRSD107

On December 9, 2024, Sirius Therapeutics presented promising preliminary data from its Phase 1 clinical trial of SRSD107, a next-generation siRNA therapeutic aimed at preventing and treating thromboembolic disorders, including myocardial infarction, ischemic stroke, and venous thromboembolism. The trial was conducted at a single site, and involved a randomized, double-blind, placebo-controlled design with 40 healthy participants divided into five cohorts. Each cohort received varying doses of SRSD107, demonstrating the drug's safety and well-tolerated status throughout. Participants monitored in the trial experienced significant pharmacodynamic changes, with reductions in Factor XI antigen and activity surpassing 90%. These results indicate sustained potent anticoagulation effects along with an increase in thromboplastin time, evident more than 16 weeks post-dosing. Key outcomes from the trial, presented during the 66th American Society of Hematology (ASH) Annual Meeting in San Diego, California, suggest a robust safety profile for SRSD107. Dr. Patrick Yue, Chief Medical Officer of Sirius, emphasized the importance of these findings, reflecting a marked and prolonged reduction in FXI antigen and activity, as well as an aPTT increase over 100% at the highest doses. These pharmacokinetic and pharmacodynamic observations highlight SRSD107's potential as an anticoagulant without significantly increasing bleeding risks, a common concern with existing therapies. The study is essential as thromboembolic disorders are a leading cause of mortality worldwide, accounting for an estimated one in four deaths. With current anticoagulants carrying risks of bleeding, SRSD107's targeted action on Factor XI mRNA could represent a significant advancement in treatment options for thromboembolic diseases. Following these initial findings, Sirius Therapeutics plans to advance SRSD107 into Phase 2 clinical studies to further evaluate its clinical efficacy and safety. Founded in 2021, Sirius Therapeutics' commitment to innovation encompasses a strategy that places emphasis on advancing novel treatment mechanisms for severe medical conditions by leveraging siRNA technology. The ongoing development of SRSD107 exemplifies the company's dedication to combating thromboembolic disorders, providing hope for better therapeutic options that minimize overall risk.