Visirna Announces Dosing of the First Subjects in China Phase I Study of VSA012

SUZHOU, CHINA – January 21st, 2025. Visirna announced that it has dosed the first subjects in a Phase 1 clinical trial of VSA012, the company's first proprietary in-house-developed investigational small interfering RNA (siRNA) in healthy volunteers. Conducted at Peking University Third Hospital, this Phase 1 clinical trial, employing a single-center, randomized, double-blind, and placebo-controlled design, aims to evaluate the safety and tolerability of VSA012 in healthy adult Chinese subjects，followed by evaluation of the safety and tolerability of VSA012 in Chinese patients with paroxysmal nocturnal Hemoglobinuria (PNH). Professor Haiyan Li, M.D., Principal Investigator at Peking University Third Hospital, remarked, "siRNA therapeutics showcase robust and persistent inhibitory effects on specific targets, distinguished by well-defined mechanism of action and unique therapeutic advantages, thereby establishing themselves as vanguards in pharmaceutical R&D. Today, at our institution, VSA012, another pioneering siRNA therapeutic developed by Visirna, achieved its first administration in healthy volunteers, marking a significant milestone." Dr. Xiaoming Zou, CEO of Visirna, expressed, “VSA012 signifies Visirna's inaugural independently developed siRNA therapeutic and the fourth siRNA-based therapeutic to embark on clinical development. Built upon our exclusive next-generation siRNA technology platform, this candidate presents unique advantages in efficacy and safety. Preclinical studies have validated VSA012’s exceptional pharmacodynamic traits alongside its favorable safety and tolerability profile. The progression of VSA012 into clinical trials marks a significant stride in our ability to innovate siRNA therapeutics. This achievement underscores Visirna's leadership in siRNA therapeutics development, setting a robust foundation for expanding our product portfolio strategically. It establishes Visirna as a comprehensive biopharmaceutical entity, equipped with end-to-end competencies spanning research, development , manufacturing, and commercialization within the field of oligonucleotide therapeutics VSA012 is the first in-house-developed siRNA therapy based on Visirna's proprietary siRNA technology platform. This GalNAc-conjugated siRNA precisely targets complement factor B (CFB) mRNA in the liver, effectively reducing CFB protein expression in the circulation. Promising preclinical results highlight VSA012's profile to durably suppress CFB protein levels in the system, leading to a significant inhibition of alternative complement pathway activity while maintaining excellent safety profile. Visirna was founded in 2022 in a strategic partnership with Arrowhead Pharmaceuticals (NASDAQ: ARWR). Headquartered in China with a global perspective, Visirna strives to emerge as a frontrunner in the advancement of siRNA therapeutics. The existing product portfolio comprises four clinical stage siRNA candidates with a focus on cardiometabolic and autoimmune diseases. For more information, please visit www. visirna.com

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