Sirnaomics Advances GalAhead™-Based RNAi Therapies for Complement-Mediated Diseases, Files Prospectus

Gaithersburg, Maryland, and Suzhou, ChinaAugust 26, 2022/PR Newswire/ --Sirnaomics Ltd.（"Company"，"Sirnaomics, Stock Code: 2257.HK), is a leading biopharmaceutical company focused on the discovery and development of RNAi therapeutics. Sirnaomics announced that the company is actively advancing its GalAhead™ RNAi delivery platform within its novel therapeutic product pipeline, concentrating on the treatment of complement-mediated related diseases. Currently, among the four candidate products based on the GalAhead™ delivery platform, STP144G targeting complement factor B has entered the IND preparation stage, while the other three products, STP146G, STP145G, and STP247G, are entering the candidate nomination stage.

Sirnaomics' proprietary GalNAc-RNAi therapeutic delivery platform, GalAhead™, is based on a unique RNA structure that can "silence" single or multiple mRNA targets. This is particularly enabled by two key technical components in its structure: mxRNA™ (miniaturized RNAi triggers) and muRNA™ (multi-unit RNAi triggers). mxRNA consists of single-stranded oligonucleotides approximately 30 nt in length, designed to downregulate a single class of genes. muRNA, on the other hand, is composed of multiple single-stranded oligonucleotides, allowing for the simultaneous "silencing" of two or more targets.

Complement-mediated related diseases are one of the core therapeutic areas for Sirnaomics' products based on the GalAhead delivery platform. The design of mxRNA inhibitors has been validated to exhibit sustained therapeutic activity in rodent and non-human primate models. Based on this, the company has developed three new mxRNA-based projects and one muRNA-based project, which have now reached the nomination stage for candidate therapeutic drugs. Among them, STP144G, targeting complement factor B, is the first of such candidates to enter the IND preparation stage and is expected to enter clinical research in the second half of 2023. STP146G, targeting complement factor C3, and STP145G, targeting complement factor C5, have been fully validated in primary cell cultures and rodent models. In addition, the company has also developed a dual-target muRNA drug compound, STP247G, which can simultaneously inhibit complement factor C and complement factor B, making it suitable for treating complement-mediated immune-related diseases. Due to the simplified structure of the aforementioned candidate drugs, their chemistry, manufacturing, and control processes after becoming drugs demonstrate favorable toxicological properties.

Sirnaomics Chief Technology Officer Dr. Dmitry Samarsky stated, "We are very satisfied with the performance of our proprietary GalAhead delivery platform. Based on this new technology, we are able to rapidly, reproducibly, and predictably expand and advance the product pipeline of this delivery platform."

"We are delighted by the rapid expansion of our pipeline based on the GalAhead delivery platform. By leveraging this unique GalNAc analog along with liver hepatocyte-targeting drug candidates, the company is entering a new therapeutic area focused on complement-mediated diseases," said Dr. Yang Lu, Founder, Chairman of the Board, Executive Director, President, and CEO of Sirnaomics. "We anticipate that the first drug candidate utilizing this delivery platform will enter clinical studies in the second half of 2023."

Complement-Mediated Related Diseases
Complement factors are a component of the immune system that protects the host from invasion and proliferation of various microorganisms. In addition, complement factors are also involved in the clearance of the body's own damaged or mutated cells. The activation of the complement system is a highly precise process strictly controlled by regulatory proteins present in plasma and on the surface of host cells. Excessive activation of complement proteins has been identified as a cause of many diseases. These diseases include 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).

AboutSirnaomics
Sirnaomics is an RNA therapeutics biopharmaceutical company with candidate products in preclinical and clinical stages, focusing on exploring and developing innovative drugs for the treatment of indications with unmet medical needs and significant market opportunities. Sirnaomics is the first clinical-stage RNA therapeutics biopharmaceutical company to hold a strong market position in both China and the United States, and it is also the first company to achieve positive Phase IIa clinical results with its core product STP705, an RNAi therapy in the field of oncology. For more information about the company, please visit:www.sirnaomics.com。

Contact Information:
Dr. Dmitry Samarsky
Chief Technology Officer of Sirnaomics
Email:DmitrySamarsky@sirnaomics.com

Investor Relations:
Ye Yongji
CFO of Sirnaomics China
Email:NigelYip@sirnaomics.com

U.S. Media Inquiry:
Alexis Feinberg
Phone: +1 203 939 2225
Email:Alexis.Feinberg@westwicke.com

Asia Media Inquiry:
Li Yaorong
Phone: +852 3150 6707
Email:sirnaomics.hk@pordahavas.com