Ractigen Therapeutics Announces First-in-Human Dosing in Phase I Clinical Trial of RAG-17 for SOD1-ALS

This study is a randomized, double-blind, placebo-controlled Phase I clinical trial designed to evaluate the safety/tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of RAG-17 in ALS patients carrying SOD1 gene mutations. The study is led by Professor Wang Yilong, Chief Scientist of the Neurology Center at Beijing Tiantan Hospital, and Professor Wu Zhiying, Director of the Rare Disease Diagnosis and Treatment Center at the Second Affiliated Hospital of Zhejiang University School of Medicine, with the collaboration of Professor Shang Huifang, Deputy Director of the Department of Neurology at West China Hospital, Sichuan University.

Dr. Longcheng Li, Founder/CEO of Ractigen, stated:"First Participant Dosed in RAG-17 Study, Marking a Significant Step Forward in Our Efforts to Combat Amyotrophic Lateral Sclerosis (ALS), a Devastating Neurodegenerative Disease. This Achievement Highlights Our Unwavering Commitment to Advancing RNA Therapies, Which Hold the Potential to Bring Life-Changing Hope to Patients Suffering from Rare and Severe Conditions Like ALS and Their Families."

RAG-17 was granted Orphan Drug Designation (ODD) by the U.S. FDA in March 2023 and subsequently received clinical trial approvals from both the FDA and China's Center for Drug Evaluation (CDE) of the National Medical Products Administration in 2024. In November 2024, RAG-17 demonstrated promising clinical outcomes in an Investigator-Initiated Trial (IIT), further validating its potential as a revolutionary therapy. These research findings were presented at the 27th Chinese Neurology Congress, Neuroscience 2024, and the 35th International ALS/MND Symposium, garnering significant attention and high praise.

RAG-17 is an innovative double-stranded small interfering RNA (siRNA) drug independently developed by Ractigen Therapeutics for the treatment of amyotrophic lateral sclerosis (ALS) carrying SOD1 gene mutations. The drug, based on Ractigen's proprietary SCAD™ (Smart Chemical-Assisted Delivery) platform, can efficiently inhibit SOD1 gene expression, thereby reducing the production of toxic proteins and protecting neuronal function.

In preclinical efficacy studies, RAG-17 significantly delayed disease onset, extended the survival of model animals, and markedly improved their motor function. Moreover, in the IIT study, intrathecal injection of RAG-17 demonstrated good tolerability and safety at all dose levels, with comprehensive safety assessment results further supporting its clinical development feasibility.

RAG-17 was granted Orphan Drug Designation (ODD) by the U.S. FDA in 2023 and has subsequently received clinical trial approvals from both the U.S. FDA and China's CDE in 2024. Phase I clinical trials have now been initiated in China, bringing new hope for ALS patients.

ALS is a chronic progressive neurodegenerative disease characterized by the damage of upper and lower motor neurons, mainly presenting as muscle weakness, muscle atrophy, bulbar paralysis, and pyramidal tract signs. ALS is an incurable disease, and the survival period of patients after onset is usually 3-5 years. ALS can be divided into sporadic and familial types, with familial ALS often caused by mutations in various genes. SOD1 gene mutation is one of the most common pathogenic factors, accounting for approximately 20% of familial ALS and 5% of sporadic ALS cases caused by SOD1 gene mutations. In China, SOD1 is the most common mutated gene leading to ALS.