Ractigen Therapeutics has announced the completion of patient enrollment and initial dosing in its phase 2 clinical trial evaluating RAG-17, an investigational small interfering RNA (siRNA) therapy targeting superoxide dismutase 1 (SOD1) mutations in amyotrophic lateral sclerosis (ALS).
The trial is a randomized, double-blind, placebo-controlled, multiple ascending dose study evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of repeated intrathecal RAG-17 injections in patients with SOD1 mutations.
Phase 2 Trial Details
The phase 2 trial is being conducted across 5 Chinese sites, including Beijing Tiantan Hospital, West China Hospital of Sichuan University, and the First Affiliated Hospital of Sun Yat-sen University.
Long-Cheng Li, MD, founder and chief executive officer of Ractigen, said in a statement, “Completing enrollment and first-dose administration in our Phase II MAD trial marks a key achievement for Ractigen as we advance RAG-17 along a defined, accelerated regulatory pathway.”
Clinical Trials Results
Annexon reported early results from the first 10 US and European patients treated with tanruprubart in its ongoing, open-label FORWARD study for Guillain-Barré syndrome (GBS). Overall, all patients showed rapid, clinically meaningful improvement in muscle strength within 4 days of a single 30 mg/kg infusion.
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Douglas Love, president and chief executive officer of Annexon, said this in a statement: “The early and marked improvements in this initial data from FORWARD are consistent with our Phase 3 and Real World Evidence findings.
Coordinated multidisciplinary management has long been identified as central to treatment in SMA, spanning physical and occupational therapy, respiratory care, nutrition, speech-language pathology, and psychosocial support.
Multidisciplinary Management
Sandeep Rana, MD, is a neurophysiologist and director of the ALS Center at the Allegheny Health Network Neuroscience Institute. His multidisciplinary clinic has operated for close to 2 decades, and he discussed the specific difficulty of staffing.
As the ALS community recognizes SMA Awareness Month, Rana’s clinic is an example of how coordinated care can make a difference in patient outcomes.
The completion of enrollment and initial dosing in the phase 2 trial of RAG-17 is a significant step forward for Ractigen Therapeutics, and the early results from the FORWARD study are a positive sign for Annexon.