{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,22]],"date-time":"2026-09-22T14:34:21Z","timestamp":1790087661091,"version":"4.0.1"},"reference-count":32,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2025,2,25]],"date-time":"2025-02-25T00:00:00Z","timestamp":1740441600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["J Peripheral Nervous Sys"],"published-print":{"date-parts":[[2025,3]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Background and Aims<\/jats:title>\n                    <jats:p>Guillain\u2013Barr\u00e9 syndrome (GBS) is an acute autoimmune peripheral neuropathy driven by autoantibodies and classical complement pathway activation. Despite treatments with intravenous immunoglobulin or plasma exchange, GBS remains characterized by variability in recovery and high incidence of residual disabilities. This randomized, double\u2010blind, placebo\u2010controlled Phase 1 trial evaluated the safety, tolerability, and pharmacokinetics of ANX005, a novel therapeutic targeting the classical complement cascade.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>Patients with recent\u2010onset GBS, who had no access to intravenous immunoglobulin or plasma exchange, received escalating doses of ANX005 or placebo as a single IV infusion. Primary objectives included assessments of safety. Secondary objectives included determination of pharmacokinetic and pharmacodynamic profiles and clinical outcomes through Week 8. Exploratory objectives included an evaluation of serum and cerebrospinal fluid (CSF) complement and tissue damage biomarkers.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      Fifty patients were randomized to receive either ANX005 (\n                      <jats:italic>n<\/jats:italic>\n                      \u2009=\u200938) or placebo (\n                      <jats:italic>n<\/jats:italic>\n                      \u2009=\u200912). ANX005 was well\u2010tolerated across all doses with no dose\u2010limiting toxicities, suggesting an acceptable safety profile. Pharmacodynamic data showed effective C1q inhibition and a reduction in neurofilament light chain levels, suggesting nerve damage mitigation. Exploratory endpoints evaluating clinical outcomes included improvements in Medical Research Council sum scores, GBS\u2010Disability Score, and Overall Neuropathy Limitations Scale with ANX005 compared to placebo, particularly in patients receiving doses that inhibited serum C1q for \u2265\u20091\u2009week and provided C1q blockade in the CSF.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Interpretation<\/jats:title>\n                    <jats:p>These results support ANX005 as a targeted therapy for GBS that modulates the classical complement pathway. Further investigation in a larger Phase 3 trial is warranted to confirm these results and assess the long\u2010term benefits of complement inhibition in patients with GBS.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1111\/jns.70009","type":"journal-article","created":{"date-parts":[[2025,3,12]],"date-time":"2025-03-12T03:15:28Z","timestamp":1741749328000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":21,"title":["Results From a Phase 1 Study Evaluating the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of\n                    <scp>ANX005<\/scp>\n                    , a C1q Inhibitor, in Patients With Guillain\u2013Barr\u00e9 Syndrome"],"prefix":"10.1111","volume":"30","author":[{"given":"Quazi Deen","family":"Mohammad","sequence":"first","affiliation":[{"name":"National Institute of Neurosciences &amp; Hospital  Dhaka Bangladesh"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0935-8079","authenticated-orcid":false,"given":"Zhahirul","family":"Islam","sequence":"additional","affiliation":[{"name":"Laboratory of Gut\u2013Brain Axis, icddr,b  Dhaka Bangladesh"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nowshin","family":"Papri","sequence":"additional","affiliation":[{"name":"Laboratory of Gut\u2013Brain Axis, icddr,b  Dhaka Bangladesh"},{"name":"Department of Neurology Erasmus MC, University Medical Center  Rotterdam the Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shoma","family":"Hayat","sequence":"additional","affiliation":[{"name":"Laboratory of Gut\u2013Brain Axis, icddr,b  Dhaka Bangladesh"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Israt","family":"Jahan","sequence":"additional","affiliation":[{"name":"Laboratory of Gut\u2013Brain Axis, icddr,b  Dhaka Bangladesh"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Khan Abul Kalam","family":"Azad","sequence":"additional","affiliation":[{"name":"Dhaka Medical College and Hospital  Dhaka Bangladesh"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dean R.","family":"Artis","sequence":"additional","affiliation":[{"name":"Annexon Bioscience  Brisbane California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Benjamin","family":"Hoehn","sequence":"additional","affiliation":[{"name":"Annexon Bioscience  Brisbane California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eric","family":"Humphriss","sequence":"additional","affiliation":[{"name":"Annexon Bioscience  Brisbane California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ping","family":"Lin","sequence":"additional","affiliation":[{"name":"Annexon Bioscience  Brisbane California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ted","family":"Yednock","sequence":"additional","affiliation":[{"name":"Annexon Bioscience  Brisbane California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Henk\u2010Andr\u00e9","family":"Kroon","sequence":"additional","affiliation":[{"name":"Annexon Bioscience  Brisbane California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2025,2,25]]},"reference":[{"key":"e_1_2_8_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(16)00339-1"},{"key":"e_1_2_8_3_1","doi-asserted-by":"publisher","DOI":"10.1038\/nrneurol.2014.121"},{"key":"e_1_2_8_4_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.lpm.2013.02.328"},{"key":"e_1_2_8_5_1","doi-asserted-by":"publisher","DOI":"10.1093\/brain\/awt285"},{"key":"e_1_2_8_6_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0b013e3182104407"},{"key":"e_1_2_8_7_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.54.12.2311"},{"key":"e_1_2_8_8_1","doi-asserted-by":"publisher","DOI":"10.1093\/brain\/awy232"},{"key":"e_1_2_8_9_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41582-019-0250-9"},{"key":"e_1_2_8_10_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0b013e3182904fcc"},{"issue":"9","key":"e_1_2_8_11_1","article-title":"Intravenous Immunoglobulin for Guillain\u2010Barr\u00e9 Syndrome","volume":"2014","author":"Hughes R. 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