#China #ALS #Ractigen Therapeutics #RAG-17 #Nantong

Ractigen's RAG-17 ALS Data Impresses at AAN 2025

In a significant milestone for Ractigen Therapeutics, the company presented groundbreaking data on RAG-17, a novel siRNA therapy for Amyotrophic Lateral Sclerosis (ALS), at the American Academy of Neurology (AAN) 2025 Annual Meeting in San Diego, California. The research showcased at the conference highlighted promising results from the Investigator-Initiated Trial (IIT), which is in its early phases, but already indicating favorable outcomes.

The presentation, titled "RAG-17 a Novel siRNA Therapy for SOD1-ALS Safety and Preliminary Efficacy from a First-in-human Trial," was delivered on April 7, 2025, by Dr. Weiqi Chen from Beijing Tiantan Hospital, representing the team led by Dr. Yilong Wang. The significance of this presentation was underlined by the recognition of the abstract as an “Abstract of Distinction” by the AAN Science Committee, affirming its quality and relevance within the field of neurology.

Key Findings from the RAG-17 IIT Study

The IIT, which is a first-in-human, open-label, dose-escalation study involving six patients suffering from SOD1-ALS over a period of 240 days, revealed several critical outcomes:

1. Safety and Tolerability: RAG-17 was generally well tolerated with intrathecal administration. Importantly, no dose-limiting toxicities (DLTs) or serious adverse events (SAEs) were reported, and most adverse events experienced were mild and transient.

2. Target Engagement: The treatment with RAG-17 led to a significant reduction in SOD1 protein levels in the cerebrospinal fluid (CSF), exceeding 50% in five out of six subjects, reinforcing the therapy's effectiveness in the central nervous system.

3. Biomarker Improvements: Significant reductions in plasma neurofilament light chain (NfL) levels—a crucial biomarker for neuroaxonal damage—were recorded, indicating a positive potential effect on neurodegeneration.

4. Clinical Efficacy: Encouraging trends were observed in clinical outcome measures. During the study, there was an average decrease of 2.17 points in the ALS Functional Rating Scale-Revised (ALSFRS-R) score, suggesting that RAG-17 might aid in slowing down functional decline in ALS patients. Additionally, the Forced Vital Capacity (FVC) remained stable in most participants, with some showing notable improvements from baseline.

Phase I Trial Progress

Aside from the IIT results, Ractigen has reported on the ongoing Phase I clinical trial for RAG-17 involving randomized, double-blind, placebo-controlled dose escalation methods. This trial aims to further ascertain the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy particularly for SOD1-ALS patients. Following the initiation of the first patient dosing in December 2024, progress has been swift, with two cohorts successfully completing their dosing.

This trial is being conducted across various sites with contributions from esteemed investigators, including Dr. Yilong Wang, Dr. Zhiying Wu, and Dr. Huifang Shang, reinforcing the collaborative nature of this groundbreaking work.

Leadership Remarks

Dr. Long-Cheng Li, President and CEO of Ractigen Therapeutics, expressed enthusiasm regarding the recognition of their research, stating, "We are thrilled that our RAG-17 program has been honored with an Abstract of Distinction at the AAN 2025 meeting. This recognition highlights the significance of our work in addressing the unmet needs of SOD1-ALS patients." He further emphasized the implications of the positive findings from the IIT, showcasing not only safety but also encouraging preliminary signals regarding efficacy.

In support of these findings, Dr. Yilong Wang noted that the outcomes presented at AAN 2025 emphasize RAG-17's potential to significantly enhance the quality of life for SOD1-ALS patients. He reiterated the exciting prospects of moving forward with the Phase I study to validate these results and bring RAG-17 closer to clinical application.

About RAG-17 and ALS

RAG-17 is an investigational siRNA therapeutic candidate utilizing Ractigen's proprietary SCAD™ delivery platform, focus on silencing the superoxide dismutase 1 (SOD1) gene, the mutations of which underpin a significant percentage of familial ALS cases. As ALS is a progressive neurodegenerative disease with limited treatment options, advancements such as RAG-17 could potentially alter the treatment landscape significantly.

Ractigen Therapeutics, committed to addressing pressing medical needs, continues fostering innovation in RNA therapeutics, and the recently unveiled data demonstrate promise for a brighter future for ALS sufferers. For more information, visit Ractigen's website.