Synerk Achieves Key Milestone with First-in-Human Dosing of Its Proprietary siRNA Drug Candidate in China

SuzhouApril 27, 2023PR Newswire -- April 27, Synerk, an innovative biopharmaceutical company dedicated to the development of RNA-targeted therapies, announced that the first subject has been successfully dosed in the Phase I clinical trial of its first small interfering RNA (siRNA) candidate product under investigation. According to publicly available data, this marks the first siRNA therapeutic candidate targeting a specific site in China to enter the clinical dosing stage, with Synerk holding full independent intellectual property rights.

This siRNA candidate drug, developed based on Synerk's patented and internationally leading GalNAc liver-targeting delivery platform technology, can safely and efficiently deliver siRNA to hepatocytes. By utilizing the natural process of RNA interference, it directly intervenes in the synthesis of liver target proteins at the mRNA level, thereby achieving therapeutic purposes. Compared with earlier siRNA drugs, this candidate drug maintains excellent activity and good safety while demonstrating enhanced stability in vivo.

This Phase I, multi-center, randomized, double-blind, placebo-controlled study aims to evaluate the safety and tolerability of the drug injection in subjects, while also exploring pharmacokinetics and preliminary efficacy. Dr. Lan Tao, CEO of Synerk, stated that novel nucleic acid therapies represented by siRNA drugs have significant market potential with many industry participants, though most remain in the preclinical stage. "The successful first-in-human dosing in this study signifies that this siRNA new drug has entered the clinical trial phase, marking an important milestone for Synerk in exploring siRNA-based treatments."

The completion of this milestone event is attributed to Synerk's decades of technical accumulation and excellent execution capabilities in the field of nucleic acid therapy. "At present, the Synerk team may be the only team in China with complete experience in bringing siRNA drugs to market, possessing first-hand experience in siRNA design, synthesis and screening, CMC, preclinical research, and clinical trials," said Dr. Lan Tao, CEO of Synerk. "We will use this siRNA product as a breakthrough to lay the foundation for the subsequent development of innovative therapies targeting other disease indications. Synerk has multiple siRNA targeted drugs under research, covering cardiovascular/metabolic diseases, liver diseases, infectious diseases, complement, coagulation-related diseases, and CNS diseases. The company has an innovative delivery technology platform that specifically targets the liver and extrahepatic organs. Currently, we are actively advancing several preclinical R&D projects, and we expect more breakthrough small nucleic acid drugs to enter the clinical stage in the future."

Dr. Jiang Weiwen, co-founder and scientific research director of Synerk, stated, "Based on decades of experience in nucleic acid pharmaceuticals and extensive in vivo and in vitro experimental screening, the Synerk team has meticulously designed this siRNA candidate drug and has applied for patents worldwide. In animal model experiments, the efficacy of this candidate drug, including biomarker change indicators and duration, has reached an internationally advanced level. In preclinical trials, it also demonstrated safety comparable to or better than other marketed siRNA drugs."

"Compared with small molecule and antibody drugs, siRNA drugs have a longer duration of efficacy and lower frequency of administration, which can significantly improve patient medication adherence," said Dr. Gang Xing, Head of Clinical Research at Synerk. "This Phase I clinical trial will provide us with sufficient safety, tolerability, and PK/PD data to support future mid-to-late stage clinical trials. We will advance the clinical trials as quickly as possible, striving to provide patients with new treatment options as soon as possible."