Ractigen Announces Positive Results for RAG-17 in ALS-SOD1 Treatment Trial
Wednesday, September 11, 2024
Ractigen Therapeutics has announced encouraging clinical data from an investigator-initiated trial (IIT) for its therapy RAG-17.
RAG-17 is a siRNA designed to target the SOD1 gene in ALS patients with harmful mutations. Using Ractigen’s SCAD™ delivery platform, RAG-17 is paired with an accessory oligonucleotide (ACO) to improve delivery into the central nervous system (CNS). Preclinical trials, particularly in mouse models of ALS, showed that RAG-17 could improve motor function and extend survival.
ALS is a serious neurodegenerative disease with no known cure. Life expectancy after diagnosis is typically 3-5 years, with most patients dying from respiratory failure. Early symptoms include muscle cramps, twitching, and weakness, which progress to difficulties with movement, speech, and eventually paralysis. Mutations in the SOD1 gene are responsible for around 20% of familial ALS cases and 5% of sporadic cases.
This small interfering RNA (siRNA) targets the Superoxide Dismutase 1 (SOD1) gene, which is linked to amyotrophic lateral sclerosis (ALS) in patients with SOD1 mutations.
The trial took place at Beijing Tiantan Hospital, a leading neurological centre in China. Six patients with ALS-SOD1 took part, and the study mainly evaluated the safety of RAG-17. Results showed that the drug, delivered through the spine (intrathecal), was well-tolerated at all doses, with only mild side effects. Further tests, including lab work, vital sign checks, and electrocardiograms, supported its safety.
In addition to safety, early signs of clinical benefit were noted. Changes in patient outcomes and key biomarkers indicated the potential effectiveness of RAG-17. These results reflect earlier preclinical studies in ALS-SOD1 mouse and rat models, where the drug showed significant benefits, such as slowing disease progression and extending survival.
Ractigen Therapeutics initial findings bring us closer to providing new hope for ALS patients. The positive outcomes highlight the potential of RAG-17 as a disease-modifying treatment for ALS-SOD1.
The trial results will be presented at several upcoming conferences, including the 27th National Conference of Neurology in China in September, Neuroscience 2024 in Chicago, USA, in October, and the 35th International Symposium on ALS/MND in Montreal, Canada, in December.
RAG-17 received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) in March 2023, with its Investigational New Drug (IND) application cleared for clinical trials in the U.S. In May 2024, the China National Medical Products Administration (NMPA) also approved the IND application for trials in China.
Source: ractigen.com
