Ractigen Therapeutics, a clinical-stage pharmaceutical company dedicated to developing innovative therapies, announced promising clinical data from an Investigator-Initiated Trial (IIT) of RAG-17, a small interfering RNA (siRNA) targeting the Superoxide Dismutase 1 (SOD1) gene. The study revealed encouraging results in the treatment of amyotrophic lateral sclerosis (ALS) associated with SOD1 mutations (ALS-SOD1).

The trial was led by Dr. Yilong Wang and conducted at Beijing Tiantan Hospital, one of China's leading centers for neurological diseases. The trial enrolled six ALS-SOD1 patients and primarily focused on assessing the safety of RAG-17. Results demonstrated that RAG-17, administered intrathecally, was well-tolerated across all dose levels. All adverse events were mild. Comprehensive safety evaluations, including laboratory assessments, vital signs, and electrocardiograms, further supported the favorable safety profile.

Encouragingly, early signs of clinical benefit were also evident. Notable changes in clinical outcomes and key biomarkers indicate the efficacy of RAG-17 within this patient population. These positive clinical findings align with Ractigen's robust preclinical data, which demonstrated significant therapeutic effects of RAG-17 in SOD1-G93A ALS mouse and rat models, including delayed disease progression and improved survival.

"These initial clinical results are truly encouraging and bring us one step closer to our goal of offering new hope to ALS patients," stated 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."

This promising data will be presented at three upcoming conferences: the 27th National Conference of Neurology in China this September, followed by 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 dedicated to 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 Investigational New Drug (IND) was cleared for clinical trials in the U.S.. Additionally, 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.