Ractigen Therapeutics, a clinical-stage biopharmaceutical company developing RNA-based medicines, recently announced the final positive results from the Investigator-Initiated Trial (IIT) of RAG-17, a novel siRNA therapy targeting SOD1 for Amyotrophic Lateral Sclerosis (ALS). The results were presented for the first time during an oral session at the American Academy of Neurology (AAN) 2025 Annual Meeting in San Diego, CA. The presentation, titled “RAG-17: A Novel siRNA Therapy for SOD1-ALS – Safety and Preliminary Efficacy from a First-in-human Trial,” was delivered by Dr. Weiqi Chen on behalf of the study’s Principal Investigator, Dr. Yilong Wang.

The abstract was selected for the prestigious "Abstracts of Distinction" award, highlighting the research's exceptional quality and relevance to the neurologic community. This follows the positive initial data previously announced in September 2024.

The final data from the IIT, a first-in-human, open-label, dose-escalation study with six SOD1-ALS patients, showed favorable safety and tolerability, with no dose-limiting toxicities or serious adverse events reported. Treatment with RAG-17 led to a substantial reduction of SOD1 protein levels in cerebrospinal fluid (CSF) in five out of six patients, confirming effective target engagement. The study also observed significant reductions in plasma neurofilament light chain (NfL) levels, suggesting a potential impact on neurodegeneration. Encouraging trends in clinical efficacy were seen, including a slowing of functional decline and stability in Forced Vital Capacity (FVC) in most patients.

Ractigen also reported that its Phase I trial for RAG-17 is progressing smoothly. The trial, which is randomized, double-blind, and placebo-controlled, aims to further evaluate safety, pharmacokinetics, pharmacodynamics, and preliminary efficacy in SOD1-ALS patients. Two cohorts have successfully completed, and the study is ongoing at multiple sites.

Dr. Long-Cheng Li, President and CEO of Ractigen, expressed excitement over the recognition and progress of the RAG-17 program. Dr. Yilong Wang, Principal Investigator of the IIT study, also commented on the promising results and the potential for RAG-17 to significantly improve the lives of SOD1-ALS patients, with further validation expected in the ongoing Phase I trial.

Source: https://www.ractigen.com/ractigens-rag-17-als-data-shines-at-aan-2025-earns-top-award-phase-i-advances