Ractigen Therapeutics Gets IND Nod from China's NMPA for Phase 1 Trials of RAG-17 in SOD1-ALS

SUZHOU, China, May 15, 2024 -- Ractigen Therapeutics, a company focused on pioneering therapies, has announced that China's National Medical Products Administration (NMPA) Center for Drug Evaluation (CDE) approved their Investigational New Drug (IND) application. This approval allows the initiation of Phase 1 clinical trials in China for RAG-17, a treatment targeting Amyotrophic Lateral Sclerosis (ALS).

The Phase I trial will be randomized, double-blind, and placebo-controlled, assessing the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of RAG-17 in ALS patients with the Superoxide Dismutase 1 (SOD1) mutation.

Dr. Long-Cheng Li, the Founder, President, and CEO of Ractigen, emphasized the importance of this approval: "This is a pivotal moment for us. RAG-17 is our first siRNA drug targeting the central nervous system to receive CDE approval. We are eager to move forward with clinical trials for ALS patients and believe in the potential of RAG-17 to provide significant benefits to those with the SOD1 mutation."

Lei Cai, a prominent ALS advocate in China who also battles the disease, expressed his hopes for RAG-17: "I am very optimistic about RAG-17. Its innovative approach and promising early results make me confident in its potential. I believe this drug could offer substantial hope and tangible benefits to the ALS community, particularly for those with the SOD1 mutation."

Previously, RAG-17 received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) in March 2023 and was cleared for clinical trials in the U.S. An ongoing Investigator-Initiated Trial (IIT) study (NCT05903690) for RAG-17 has already recruited and treated six patients, showing promising preliminary safety and efficacy results.

RAG-17 is a therapeutic siRNA designed to suppress the SOD1 gene in ALS patients with pathogenic mutations. Using Ractigen’s proprietary SCAD™ delivery platform, RAG-17 combines siRNA with an accessory oligonucleotide (ACO) to enhance its effectiveness in central nervous system (CNS) tissues. Preclinical studies, including those using the hSOD1G93A mouse model, have shown that RAG-17 significantly improves motor function and prolongs survival.

Amyotrophic Lateral Sclerosis (ALS) is a severe neurodegenerative disease with no cure, drastically reducing life expectancy. Most patients succumb to respiratory failure within 3-5 years of diagnosis. Initial symptoms often include muscle cramps, twitching, and weakness, progressing to movement and speech difficulties, assisted breathing, paralysis, and eventually death. Mutations in the SOD1 gene account for about 20% of ALS cases among more than 50 genes associated with the disease.

Ractigen Therapeutics is a clinical-stage pharmaceutical company dedicated to developing innovative oligonucleotide drugs and therapeutic approaches. The company has developed several leading oligonucleotide drug delivery platforms, including SCAD™ and LiCO™, and has a diverse pipeline of oligonucleotide drugs. Their drugs target various conditions, including neurodegenerative and neuromuscular diseases, cancer, metabolic disorders, and hematological diseases. Ractigen aims to deliver groundbreaking therapeutic solutions for previously undruggable targets and incurable diseases across various fields.

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