Ractigen Therapeutics Receives for beginning Phase 1 Trials for RAG-17

Ractigen Therapeutics Receives for beginning Phase 1 Trials for RAG-17

By, Admin 17 May 2024

Ractigen Therapeutics Receives Approval from China’s NMPA to Commence Phase 1 Clinical Trials for RAG-17 in SOD1-ALS Patients

Overview

Ractigen Therapeutics, a leading clinical-stage pharmaceutical company dedicated to developing innovative therapies, has announced a significant achievement: the approval of its Investigational New Drug (IND) application by the Center for Drug Evaluation (CDE) of China’s National Medical Products Administration (NMPA). This approval allows the initiation of Phase 1 clinical trials in China for RAG-17, a potential treatment for Amyotrophic Lateral Sclerosis (ALS).

Phase I study

The Phase I study, enabled by the clearance of the IND, will be a randomized, double-blind, placebo-controlled trial focused on evaluating the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of RAG-17 in ALS patients carrying the Superoxide Dismutase 1 (SOD1) mutation.

Ractigen on This Milestone

Ractigen, expressed great excitement about this milestone: “This marks a significant moment for our company, as RAG-17 becomes our first siRNA drug targeting the central nervous system to receive approval from the CDE. We are eager to progress into clinical trials for ALS patients and are hopeful about its potential to provide meaningful benefits to those with the SOD1 mutation.”

Words from an ALS Advocate

Lei Cai, a prominent ALS advocate and patient in China, also shared his optimism about RAG-17: “I am hopeful about RAG-17. Its innovative approach and promising initial results give me confidence in its potential. I believe this drug could offer significant hope and tangible benefits to the ALS community, particularly for those with the SOD1 mutation.”

Previous ODD

  • RAG-17 previously received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) in March 2023 and has been granted clearance for clinical trials in the U.S. 
  • Additionally, an ongoing Investigator-Initiated Trial (IIT) study (NCT05903690) for RAG-17 has shown promising preliminary safety and efficacy results, with six patients already recruited and treated.

RAG-17 Related Studies

  • RAG-17 is a therapeutic siRNA designed to suppress the SOD1 gene in ALS patients with pathogenic mutations. 
  • Leveraging 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 demonstrated RAG-17’s significant therapeutic efficacy in improving motor function and extending survival.

ALS

  • ALS is a severe neurodegenerative disease without a cure, significantly reducing life expectancy. 
  • Most patients succumb to respiratory failure within 3-5 years of diagnosis. 
  • Initial symptoms typically include muscle cramps, twitching, and weakness, progressing to movement and speech difficulties, the need for assisted breathing, paralysis, and ultimately death. 
  • Mutations in the SOD1 gene account for approximately 20% of ALS cases.

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