Guoxin Pharma Supports First-in-Human Dosing of ART104 in Alagille Syndrome Trial, Advancing a Potential First-in-Class Therapy

On September 15, 2025, Guoxin Pharmaceutical Technology (Beijing) Co., Ltd. ("Guoxin Pharmaceutical") and Arnatar Therapeutics reached a significant milestone — ART104, a potential "first-in-class" antisense oligonucleotide (ASO) drug for Alagille Syndrome (ALGS), jointly advanced by both parties, has successfully completed the first dosing in an investigator-initiated clinical trial (IIT) conducted at the Children's Hospital of Fudan University on September 1. It was reported that the first patient experienced no discomfort throughout the process, and no drug-related side effects were found during the first follow-up visit after administration, demonstrating good safety. As the core partner of the project, Guoxin Pharmaceutical provided strong support for this key breakthrough in the treatment of rare diseases with its professional service capabilities.

ART104 is the first ASO therapeutic drug designed to upregulate Jag1 protein expression. Relying on Arnatar's self-developed technology, it treats ALGS—a severe multisystem disease characterized by bile duct paucity and liver dysfunction—by addressing the genetic root cause, which is Jag1 protein deficiency. ART104 has previously been granted Orphan Drug Designation (ODD) and Pediatric Rare Disease Designation (PRDD) by the U.S. FDA. Preclinical studies have demonstrated significant advantages in restoring hepatic bile duct development and showing good tolerability, with the potential to change the current treatment standards for ALGS.

This IIT trial, led by Professor Wang Jianguo from the Children's Hospital of Fudan University, will focus on evaluating the safety, pharmacokinetics/pharmacodynamics, and efficacy of ART104. Throughout the project's advancement, Guoxin Pharmaceutical Technology (Beijing) Co., Ltd. provided core support in multiple aspects to ensure the orderly conduct of the trial.

• Full-process Collaboration Support:Deeply involved in the overall advancement of the trial, coordinating various resources to ensure smooth transitions across all stages from trial initiation to the first dose administration, guaranteeing the efficient implementation of the project.

• Key Professional Empowerment in Critical Areas:Focusing on the core areas of clinical trials, we provide professional services tailored to project needs, injecting expertise into key aspects such as trial design and advancement.

• Compliance and Security Safeguards:Based on compliance standards, focus on safety control during the trial process to provide strong support for the standardized conduct of the trial.

The completion of the first dosing of ART104 with this support is another practical achievement for Guoxin Pharmaceutical Technology (Beijing) Co., Ltd. in its deep cultivation of professional services in the rare disease treatment field and empowerment of innovative R&D. In the future, Guoxin Pharmaceutical will continue to leverage its strengths in the clinical research field, deepen collaboration with global innovative pharmaceutical companies, and focus on more rare diseases like ALGS and other major disease areas. With higher quality and efficient services, it aims to accelerate the development of more innovative drugs, helping more patients gain access to safe and effective treatment options as soon as possible, thereby contributing to the improvement of human health.