Ractigen Completes Enrollment for RAG-17 in SOD1-ALS, Tanruprubart Shows Rapid Responses in GBS, Building a Multidisciplinary SMA Clinic

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Welcome to the Neurology News Network, my name is Louie Pasculli and here’s a look at some of the top stories in Neurology.

Beginning with positive clinical results, Ractigen Therapeutics announced the completion of patient enrollment and initial dosing across all cohorts of 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).¹

The phase 2 trial (NCT06556394) 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. It 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 pivotal achievement for Ractigen as we advance RAG-17 along a defined, accelerated regulatory pathway.”¹

Following up with more positive 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).¹ Overall, all patients showed rapid, clinically meaningful improvement in muscle strength within 4 days of a single 30 mg/kg infusion, with the company describing the outcomes as consistent with previously reported phase 3 and real-world evidence findings.²

The first reported cohort included both male and female patients ranging from 12 to 78 years old and covering the spectrum of disease from moderate to severe. Four patients who were bedbound early in their disease course walked with or without assistance between days 2 and 8, and the 1 patient who required mechanical ventilation early in the disease course came off the ventilator within 4 days. The remaining 5 patients all showed marked gains in function within 48 hours.

Douglas Love, president and chief executive officer of Annexon, said this in a statement.² “The early and marked improvements in this initial data from FORWARD are consistent with our Phase 3 and Real World Evidence findings. That consistency continues to strengthen the significant functional outcomes being achieved with our differentiated C1q-focused approach targeting neuroinflammation at its source.”

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. Assembling and sustaining this type of care team, particularly outside academic centers, remains a practical barrier, and one that determines whether patients reach the clinicians equipped to treat them.

Sandeep Rana, MD, is a neurophysiologist and director of the ALS Center at the Allegheny Health Network Neuroscience Institute, where he also served as program director of the neurology residency program, and serves as a professor of neurology at Drexel University. His multidisciplinary clinic, established for amyotrophic lateral sclerosis (ALS) and later expanded to serve patients with SMA, has operated for close to 2 decades.

In recognition of SMA Awareness Month, held annually throughout August, NeurologyLive^® spoke with Rana about how his clinic is structured, how the arrival of disease-modifying therapies shifted the team’s priorities toward wellness and long-term function, and why he considers the assembly of a multidisciplinary team an organizational problem as much as a clinical one. He also discussed the specific difficulty of staffing intrathecal administration, and what he would ask of neurologists practicing in community settings.

To read the full pieces and to get more direct access to expert insight, head to NeurologyLive.com. Be sure to tune in next week to remain informed on the latest in neurology. I’m Louie Pasculli, thanks for watching Neurology News Network.

REFERENCES
1. Ractigen Therapeutics Announces Completion of Enrollment and First Dosing in Phase II Clinical Trial of RAG-17 for SOD1-ALS. News release. Ractigen Therapeutics. August 6, 2026. Accessed August 11, 2026. https://www.prnewswire.com/news-releases/ractigen-therapeutics-announces-completion-of-enrollment-and-first-dosing-in-phase-ii-clinical-trial-of-rag-17-for-sod1-als-302844916.html
2. Annexon Reports First U.S. and European Patient Cohort of GBS FORWARD Study Demonstrated Rapid, Positive Clinical Responses in All Tanruprubart Treated Patients Within One Week. News release. Annexon, Inc. August 6, 2026. Accessed August 10, 2026. https://ir.annexonbio.com/news-releases/news-release-details/annexon-reports-first-us-and-european-patient-cohort-gbs-forward