Sirnaomics Advances GalAhead™-Based RNAi Therapeutics for Treatment of Complement-Related Diseases
Gaithersburg, MD, USA and Suzhou Biobay, China, August 26, 2022 – Sirnaomics Ltd. (the “Company” or “Sirnaomics”, stock code: 2257.HK), a leading biopharmaceutical company in discovery and development of RNAi therapeutics, announced today the advancement of its GalAhead™ RNAi delivery platform for developing novel therapeutic products focused on complement-related diseases. Among four product candidates developed with GalAhead™, STP144G, which targets Complement Factor B, has entered into an IND enabling study, while the other three—STP146G, STP145G, and STP247G—are moving into candidate nomination stage.
Sirnaomics’ proprietary GalNAc-RNAi therapeutic platform, GalAhead™, 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 nucleotide long oligonucleotides to downregulate individual genes, while muRNA molecules are comprised of multiple oligonucleotides to silence two or more targets simultaneously.
Complement-related diseases are amongst the important therapeutic areas Sirnaomics has selected for developing GalAhead-based treatments. Based on the validation of the mxRNA inhibitor design, with a long-lasting therapeutic activity observed using rodent and non-human primate animal models, the Company has developed three new mxRNA-based and one muRNA-based programs into the therapeutic candidate nomination phase. STP144G, which targets Complement Factor B, is the first drug candidate that has entered into an IND-enabling study and is expected to advance into clinical study in the second half of 2023. STP146G, which targets Complement Component C3, and STP145G, which targets Complement Component C5, have been validated with both primary cell culture and rodent animal models. Sirnaomics has also developed a dual-targeting muRNA drug compound, STP247G, to inhibit both Complement Component C5 and Complement Factor B for the potential treatment of the complement-mediated immunologic disorders. Due to the simplified structure of these drug candidates, the chemistry, manufacturing and control process of the drug product is well established with a favorable toxicological profile.
Dr. Dmitry Samarsky, Sirnaomics Chief Technology Officer, commented: “We are highly satisfied with the performance of our proprietary GalAhead platform, which allows us to quickly, reproducibly, and predictably expand and progress our therapeutic pipeline based on this novel technology.”
“We are seeing a quick expansion of our product pipeline developed with the GalAhead platform. Our drug candidates are comprised of GalNAc-alike structure and liver hepatocyte targeting property, which enable Sirnaomics to advance into a new frontier to treat complement-related disorders,” said Dr. Patrick Lu, Sirnaomics founder, Chairman of the Board, Executive Director, President and CEO. “We expect that the first drug candidate based on this delivery platform to be in clinical study in the second half of 2023.”
About Complement-Related Diseases
Complement is an integral part of the immune system protecting the host organism against invasion and proliferation of various microorganisms. It is also involved in the removal of the body's own damaged and altered cells. Activation of the complement system is a very precise process and it is strictly controlled by regulatory proteins present in both plasma and at host cells' surfaces. The excessive activation of complement proteins is often discovered to be the reason for many diseases. These include e.g. autoimmune diseases, Alzheimer's syndrome, schizophrenia, atypical hemolytic-uremic syndrome, angioedema, macular degeneration, and Crohn's disease. (Adv Clin Exp Med, 2012 Jan-Feb;21(1):105-14).
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 China and the United States, and also the first company to achieve positive Phase IIa clinical outcomes in oncology for an RNAi therapeutic for its core product, STP705. Learn more at www.sirnaomics.com.
Dr. Dmitry Samarsky
Chief Technology Officer, Sirnaomics
Chief Financial Officer, China, Sirnaomics
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