The long-awaited positive results of Annexon Inc.’s Phase 3 trial investigating ANX005 for Guillain-Barré syndrome (GBS) was presented at the PNS 2024 Annual Meeting.
Several leading global GBS experts spoke of the significant need to enact changes in treatment of these patients through targeted immunotherapy.
Douglas Love M.D., Annexon CEO, said that the company has completed the first successful placebo-controlled trial for the syndrome in several decades. The Phase 3 results exhibited durable recovery from GBS compared to the placebo.
The Principal Trial Investigator, Quazi Deen Mohammad M.D. of Bangladesh, added the study showed that suppression of C1q by ANX005 allowed patients to recover sooner. This translates into longer-term benefits and more of a likelihood for full recovery as compared to placebo.
Key Findings
• Patients treated with ANX005 30 mg/kg showed a faster and a more complete recovery through Week 26 on both primary and pre-specified endpoints
• The study defined the highest and best treatment timing during the most active phase of acute GBS
• Robust, durable, and early treatment facilitated patient recovery
• Adverse advents were mild to moderate showing no increase in infections
• Vaccinations and prophylactic antibiotics were not required
About Guillain-Barre Syndrome (GBS)
GBS is an acute, rare neurological disorder that results in over 22,000 people being hospitalized in Europe and the U.S. every year. This cost translates to a multi-billion-dollar annual burden for the U.S. and Europe. It is a condition whereby the immune system attacks the nerves, causing weakness or paralysis. The cause is unknown. Most people are able to walk within months after the onset of GBS. Some cases may be severe but most initially require hospitalization.
About ANX005
ANX005 has been designed to reduce nerve damage and inflammation by precluding C1q activity in the nervous system. ANX005 finds its C1q target and prevents it from binding to the peripheral nerves. The drug is delivered intravenously and is known to act immediately. ANX005 has earned orphan drug and fast track designations from the FDA and orphan designations from the EMA.
About Annexon Biosciences
The biopharmaceutical company advances therapies for people with devastating neuroinflammatory diseases. Annexon’s novel approach targets C1q and blocks it before it takes hold.
