Ractigen Reports Positive Trial Results for RAG-17 in ALS-SOD1 Treatment

NANTONG, China and SUZHOU, China I September 10, 2024 I Ractigen Therapeutics, a clinical-stage pharmaceutical company focused on creating advanced therapies, has publicized promising clinical data from an Investigator-Initiated Trial (IIT) of RAG-17, a small interfering RNA (siRNA) aimed at the Superoxide Dismutase 1 (SOD1) gene. The study highlighted positive outcomes in treating amyotrophic lateral sclerosis (ALS) linked to SOD1 mutations (ALS-SOD1).

The trial, spearheaded by Dr. Yilong Wang, took place at Beijing Tiantan Hospital, a premier center for neurological diseases in China. Six patients with ALS-SOD1 were enrolled, with the primary objective being the safety evaluation of RAG-17. The findings revealed that RAG-17, when administered intrathecally, was well-tolerated across all dosages, with only mild adverse events reported. Comprehensive safety assessments, which included laboratory tests, vital signs, and electrocardiograms, corroborated the favorable safety profile.

Moreover, early indicators of clinical benefit were observed. Significant improvements in clinical outcomes and key biomarkers were noted, suggesting the efficacy of RAG-17 in this patient group. These positive clinical results are consistent with Ractigen’s extensive preclinical data, which demonstrated substantial therapeutic effects of RAG-17 in SOD1-G93A ALS mouse and rat models, such as delayed disease progression and enhanced survival rates.

“These initial clinical results are truly encouraging and bring us one step closer to our goal of offering new hope to ALS patients,” commented Dr. Long-Cheng Li, Founder and CEO of Ractigen Therapeutics. “The positive outcomes from this trial underscore the potential of RAG-17 as a disease-modifying therapy for ALS-SOD1. We are fully committed to advancing its clinical development and ultimately delivering this much-needed treatment to patients.”

The promising data will be showcased at three upcoming conferences: the 27th National Conference of Neurology in China this September, Neuroscience 2024 in Chicago, USA in October, and the 35th International Symposium on ALS/MND in Montreal, Canada in December—one of the largest annual gatherings focused on ALS and motor neuron disease research.

RAG-17 received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) in March 2023 and was approved for clinical trials in the U.S. as an Investigational New Drug (IND). In addition, in May 2024, the IND application was approved by the Center for Drug Evaluation (CDE) of China National Medical Products Administration (NMPA) for clinical trials in China.

RAG-17 is a siRNA specifically designed to target and suppress the SOD1 gene in ALS patients with pathogenic mutations. Using Ractigen’s proprietary SCAD™ delivery platform, RAG-17 is conjugated with an accessory oligonucleotide (ACO) for enhanced delivery into the central nervous system (CNS). Preclinical studies, including those using the hSOD1G93A mouse model, have shown significant therapeutic efficacy of RAG-17 in improving motor function and extending survival.

ALS is a severe neurodegenerative disease with no cure, drastically cutting down life expectancy, with most patients losing their lives to respiratory failure within 3-5 years of diagnosis. Initial symptoms generally involve muscle cramps, twitching, and weakness. These symptoms progress to difficulties with movement and speech, the need for assisted breathing, paralysis, and ultimately death. Mutations in the SOD1 gene account for about 20% of familial ALS and 5% of sporadic ALS cases.

Ractigen Therapeutics, a clinical-stage pharmaceutical company, is devoted to developing pioneering oligonucleotide drugs and therapeutic strategies. The company has developed several globally leading oligonucleotide drug delivery platforms, including SCAD™ and LiCO™, and maintains a diverse pipeline of oligonucleotide drugs targeting a wide range of conditions, from neurodegenerative and neuromuscular diseases to cancer, metabolic disorders, and hematological diseases. The company aims to provide innovative therapeutic solutions for previously undruggable targets and address incurable diseases across different medical fields.