Study commences following green light from the U.S. FDA for Investigational New Drug Application
HONG KONG, GERMANTOWN, Md. and SUZHOU, China, April 12, 2023 /PRNewswire/ -- Sirnaomics Ltd. (the "Company", Stock Code: 2257.HK, together with its subsidiaries, the "Group" or "Sirnaomics"), a leading biopharmaceutical company in discovery and development of RNAi therapeutics, today announced the launch of a Phase I clinical trial of STP122G based on the Group's GalNAc Factor XI Program. This Factor XI program is applicable across a broad range of disease indications as an anticoagulant therapeutic. This study marks the first time that Sirnaomics is utilizing its proprietary GalNAc RNAi platform technology, GalAhead™, in one of its siRNA-based candidates and conducting a trial for a patient population with high unmet need in anticoagulation disorders.
Factor XI (FXI) is an enzyme produced predominantly by hepatocytes in the liver and it plays an important role in the body's blood clotting cascade. The recent increase in the interest in FXI for use as an anticoagulant is attributable to the very promising clinical results and an excellent safety profile due to the fact that reduction of FXI impacts only the intrinsic coagulation pathway while preserving the extrinsic pathway and bleeding time. The site of production for FXI also makes it an ideal target for GalNAc-based siRNA therapeutics. Sirnaomics' Phase I, single-center, randomized, double-blind, placebo-controlled, sequential cohort study is designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of the GalNAc-siRNA treatment STP122G, administered subcutaneously in healthy volunteers. By targeting FXI, the Group has the potential to target multiple diseases that require anticoagulation such as atrial fibrillation, pulmonary embolism, deep vein thrombosis (DVT), and deep venous thrombosis prophylaxis for surgical procedures.
"Our hope in pursuing a treatment program that focuses on FXI, a liver enzyme that plays a key role in blood clotting, is to explore treatments for anticoagulation disorders that are life threatening," said Dr. Michael Molyneaux, M.D., Executive Director and Chief Medical Officer of Sirnaomics. "This IND gives us our first opportunity to explore a treatment developed with our GalNAc platform, after receiving safe to proceed letter from the U.S. Food and Drug Administration (the "U.S. FDA"). Our goal is to determine the safety and tolerability of FXI-GalNAc-siRNA to inform dosage in future studies. We anticipate dosing our first volunteer in mid-2023."
"We have initiated the development of GalAhead™ technology since 2019 and we have demonstrated its reproducible and robust performance in numerous animal studies," commented Dr. Dmitry Samarsky, Chief Technology Officer of Sirnaomics. "Our current therapeutic pipeline based on GalAhead™ comprises ten programs in the areas of hematologic, cardiometabolic, immunologic and rare diseases. Entering Phase I clinical trial with its frontrunner Factor XI program marks a major milestone for the GalAhead™ technology and Sirnaomics."
"Having clinical programs based on two different technology platforms marks a major milestone for Sirnaomics, allowing the Group to expand into much broader therapeutic areas," commented Dr. Patrick Lu, Founder, Chairman of the Board, Executive Director, President and CEO of Sirnaomics. "The proprietary GalAhead™ technology provides a unique opportunity which helps the Group's clinical programs go beyond the oncology and fibrosis fields. STP122G's long lasting safety and efficacy effects observed with a non-human primate model warrants its clinical application on anticoagulant therapy."
About the GalAhead™ Technology
The GalAhead™ is a proprietary technology platform for RNAi therapeutics, developed by Sirnaomics. The GalAhead™ platform relies on unique RNA structures that allow the knockdown of single or multiple distinct mRNA targets, specifically two key technological components: mxRNA™ (miniaturized RNAi triggers) and muRNA™ (multi-unit RNAi triggers). mxRNAs™ are comprised of single ~30 nt long oligonucleotides to downregulate individual genes, while muRNA™ molecules are comprised of multiple oligonucleotides to silence two or more targets simultaneously. The targeted delivery technology has demonstrated specific liver hepatocyte targeting via a cell surface receptor ASGPR.
About Sirnaomics
Sirnaomics is an RNA therapeutics biopharmaceutical company with product candidates in preclinical and clinical stages that focuses on the discovery and development of innovative drugs for indications with medical needs and large market opportunities. Sirnaomics is the first clinical-stage RNA therapeutics company to have a strong presence in both Asia and the United States. Based on its proprietary delivery technologies: Polypeptide Nanoparticle Formulation and the 2nd generation of GalNAc conjugation, the Group has established very enriched drug candidate pipeline. Sirnaomics is currently holding a leadership position on advancing RNAi therapeutics for oncology application with multiple successes of its clinical programs for STP705 and STP707. STP122G represents the first drug candidate of GalAhead™ technology entering clinical development. With establishment of the Group's manufacturing facility, Sirnaomics currently is undergoing a transition from a biotech company to a biopharma corporation. Learn more at: www.sirnaomics.com.
