#China #Shanghai #coronary artery disease #Ribo Life Science #Vortosiran

Promising Outcomes from Ribo's Vortosiran Phase 2a Trial

Suzhou Ribo Life Science Co., Ltd. recently shared significant findings from their Phase 2a trial of Vortosiran, a novel siRNA therapy targeting Factor XI (FXI) for patients diagnosed with chronic coronary artery disease. The announcement, made during the China Pharmaceutical Innovation Conference in Shanghai, highlights the crucial advancements in cardiovascular treatment.

Vortosiran, known by its developmental code RBD4059, was evaluated in a randomized, double-blind, placebo-controlled study that aimed to assess its safety and effectiveness. Results indicated that Vortosiran, when administered to patients already receiving standard aspirin therapy, was generally well-tolerated. Notably, the therapy achieved a profound, dose-dependent, and lasting suppression of FXI activity, which could lead to groundbreaking changes in anticoagulant treatment options for these patients.

The study specifically involved individuals with a history of chronic coronary artery disease and myocardial infarction. With high doses of Vortosiran, patients demonstrated a remarkable average reduction of FXI activity by 92%, lasting for several months post-treatment. This reduction vastly exceeds the effects seen with existing small molecule treatments, generally given on a daily basis but currently in Phase 3 trials.

Importantly, the trial reported no severe adverse events related to the treatment, nor were there any major or clinically relevant minor bleeding incidents. These findings provide the first global proof of concept for siRNA-mediated FXI suppression to treat patients with coronary artery conditions. The data strongly supports Vortosiran's potential as a distinctive, long-acting antithrombotic therapy designed to mitigate thrombotic risks while maintaining hemostasis, addressing a significant unmet medical need in cardiovascular care.

Dr. Li-Ming Gan, Joint CEO and Global Head of R&D at Ribo, stated, “Preventing blood clot formation without increasing bleeding risks has been a long-standing goal in cardiovascular medicine. The Phase 2a data, acquired within our target patient population, reinforces our belief that Vortosiran presents a safe and highly differentiated approach to FXI inhibition in thromboembolic diseases. Based on these results, we have initiated the ORBIT-XI program (Optimizing RNA-Based Inhibition of Thrombosis), which includes multiple Phase 2b trials aimed at advancing to Phase 3 development across various indications.”

About Vortosiran (RBD4059)

Vortosiran is a GalNAc-conjugated siRNA candidate developed using the proprietary RiboGalSTAR™ platform targeting the liver. By selectively suppressing FXI synthesis, Vortosiran inhibits the intrinsic pathway of coagulation and showcases strong antithrombotic action. Genetic evidence strongly suggests that individuals born with naturally lower levels of FXI enjoy protection against thrombotic disease without incurring a higher risk of spontaneous bleeding. This makes FXI inhibition a particularly attractive therapeutic avenue with the potential for effective thrombosis prevention and an enhanced safety profile compared to existing anticoagulant therapies.

About Suzhou Ribo Life Science Co., Ltd. and Ribocure Pharmaceuticals AB

Suzhou Ribo Life Science Co., Ltd. (06938.HK) is a pioneering clinical R&D company dedicated to advancing nucleic acid-based drug development stemming from RNA interference technology. With innovative R&D capabilities and vertically integrated tech platforms, Ribo has established a robust product pipeline focused on addressing serious diseases with unmet medical needs.

As a subsidiary of Suzhou Ribo Life Science, Ribocure Pharmaceuticals AB specializes in the global development of life-saving oligonucleotide therapies, concentrating on advancing product portfolios and novel therapeutic targeting ideas while also enhancing clinical trial capabilities.

For more information, visit www.ribolia.com and www.ribocure.com.