BRISBANE - Annexon, Inc. (Nasdaq: ANNX), today announced positive results from the completed pivotal Phase 3 trial of C1q-targeted immunotherapy, ANX005, in Guillain-Barre Syndrome (GBS) at the 2024 Peripheral Nerve Society (PNS) Annual Meeting in Montreal, Canada.

Leading global experts in the GBS field highlighted the significant unmet need and opportunity to transform the GBS treatment landscape with a targeted immunotherapy approach, as well as additional Phase 3 analyses of early and durable treatment effects important to patients and the medical community.

'ANX005 rapidly suppressed neuroinflammation and validated the role of C1q inhibition in GBS during the active phase of disease, leading to highly statistically significant improvements across multiple endpoints and over multiple timepoints versus placebo,' said Douglas Love, president and chief executive officer of Annexon. 'Having successfully completed the first placebo-controlled GBS trial in decades, we were honored to present the pivotal Phase 3 results showing accelerated and durable recovery of GBS patients treated with ANX005 compared to placebo in the plenary Symposium at PNS. With these favorable results, we are laser focused on bringing ANX005 to GBS patients worldwide as quickly as possible.'

Dr. Quazi Deen Mohammad, Principal Investigator of the trial and Founding Director of the National Institute of Neurosciences and Hospital (NINS), Bangladesh added, 'This well-designed and well-executed study demonstrated that acute suppression of C1q with ANX005 enabled patients to get better sooner, which translated into continued long-term benefits and a significantly higher likelihood of full recovery versus placebo. Notably, patients came off ventilation and walked one month earlier, regained their independence faster, and got back to a normal way of life sooner. In addition, Phase 3 patients with baseline characteristics consistent with North American and European GBS patients had a more pronounced treatment effect, being three times more likely versus placebo to be in a good state of health with ANX005 treatment. These compelling data reinforce the therapeutic potential of ANX005 to be the first targeted immunotherapy treatment for GBS.'

Summary of Phase 3 Data with ANX005 30 mg/kg Treatment

GBS-Disability Scale (GBS-DS)

Primary endpoint at Week 8: 2.41-fold higher likelihood of being in a better state of health with ANX005 vs. placebo (p = 0.0058)

Week 1: 7.22-fold higher likelihood of being in a better state of health with ANX005 vs. placebo (p < 0.0001)

Week 4: 2.49-fold higher likelihood of being in a better state of health with ANX005 vs. placebo (p = 0.0073)

Week 26: 2.5 times more patients had fully recovered to a normal / pre-disease state of health (GBS-DS = 0) with ANX005 (21.5%) vs. placebo (8.6%) (OR 4.14, p = 0.0092)

Week 8 responder analysis (pre-specified sensitivity analysis): 2-times more patients improved 3 points or more with ANX005 (28.2%) vs. placebo (13.6%) (p = 0.0309)

Week 8 dichotomy analysis (pre-specified sensitivity analysis): 2.5-times more patients were able to run or better ANX005 (29%) vs. placebo (12%) (OR 3.34, p = 0.0065)

Functional Measures

Walking 31 days earlier with ANX005 treatment (56 days) vs. placebo (87 days) (p = 0.0211)

Off ventilation 28 days earlier with ANX005 treatment (20 days) vs. placebo (48 days) (p = 0.0356)

Patients with North American and European baseline characteristics

Week 1: 8.8-point improvement in muscle strength measured by Medical Research Council (MRC) sumscore with ANX005 vs. placebo (p

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