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ANX005

Phase 3

Guillain-Barre Syndrome | Small molecule | Neurology |Annexon, Inc.|Last Updated: Jun 30, 2026

Success Probability
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials1
Total Enrollment242
FDA Designations
ORPHAN_DRUGFAST_TRACK
Clinical trial landscape

ANX005 · 5 trials · 5 indications

Phase 3 1Phase 2 3Phase 1 1
NCT04701164Efficacy and Safety of ANX005 in Subjects With Guillain-Barré SyndromeGuillain-Barre Syndrome
COMPLETED242 Analytics
PHASE3COMPLETED
Efficacy and Safety of ANX005 in Subjects With Guillain-Barré Syndrome
Guillain-Barre SyndromeUnlock trial analytics
Study Endpoints
Primary Endpoints
GBS Disability Score (GBS-DS) at Week 8
Week 8
Number of Participants with Adverse Events
Through Month 6

Number participants recently diagnosed with GBS who experience adverse events.

Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Day 1 through Day 71

An adverse event (AE) was any untoward medical occurrence in a participant who had been administered a pharmaceutical product. An AE did not necessarily have a causal relationship with the product and therefore could be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a pharmaceutical product. An AE could arise with any use, route of administration, formulation, dose (including an overdose), or when used in combination with another pharmaceutical product. A TEAE was an AE with an onset date/time after the first infusion of ANX005 until the end of the study. A summary of serious and all other non-serious adverse events regardless of causality is located in the Adverse Events module.

Maximum Change From Baseline in Hemoglobin Levels
Baseline up to Day 71

Maximum change from Baseline was calculated as the maximum post-Baseline value observed up to Day 71 minus the Baseline value. Baseline was defined as the last non-missing observation recorded before the first dose of ANX005.

Change From Baseline in Lactate Dehydrogenase Levels at Day 71
Baseline, Day 71

Change from Baseline was calculated as the post-Baseline value at Day 71 minus the Baseline value. Baseline was defined as the last non-missing observation recorded before the first dose of ANX005.

Change From Baseline in Percentage of Reticulocytes/Total Cells Count at Day 71
Baseline, Day 71

Change from Baseline was calculated as the post-Baseline value at Day 71 minus the Baseline value. Baseline was defined as the last non-missing observation recorded before the first dose of ANX005.

Change From Baseline in Haptoglobin Levels at Day 71
Baseline, Day 71

Change from Baseline was calculated as the post-Baseline value at Day 71 minus the Baseline value. Baseline was defined as the last non-missing observation recorded before the first dose of ANX005.

Change From Baseline in Total Bilirubin Levels at Day 71
Baseline, Day 71

Change from Baseline was calculated as the post-Baseline value at Day 71 minus the Baseline value. Baseline was defined as the last non-missing observation recorded before the first dose of ANX005.

Change From Baseline in Indirect Bilirubin Levels at Day 71
Baseline, Day 71

Change from Baseline was calculated as the post-Baseline value at Day 71 minus the Baseline value. Baseline was defined as the last non-missing observation recorded before the first dose of ANX005.

Number of Participants Who Experienced Treatment-Emergent Adverse Events
Up to Week 36
Safety and tolerability of intravenous ANX005 administered for up to 22 weeks in subjects with, or at risk for, manifest Huntington's Disease
Up to Week 36

As measured by incidence of TEAEs, SAEs, AEs related to ANX005, SAEs related to ANX005, Grade 3 or higher AEs, Grade 3 or higher AEs related to ANX005, AEs leading to study or treatment discontinuation.

Pharmacokinetics (PK) of ANX005
Up to Week 36

As measured by ANX005 serum and cerebrospinal fluid concentrations

Pharmacodynamics (PD) effects of ANX005
Up to Week 36

As measured by C1q, C4a, and NfL levels in blood and/or cerebrospinal fluid concentrations

Safety and tolerability of ANX005 when administered in combination with IVIg: incidence of TEAEs, SAEs, AE's
6 months

As measured by incidence of TEAEs, SAEs, AE's related to ANX005, SAE's related to ANX005, Grade 3 or higher AEs, Grade 3 or higher AEs related to ANX005, AEs leading to study or treatment discontinuation.

Secondary Endpoints
Medical Research Council (MRC) Sum Score at Week 8
Week 8
MRC Sum Score at Day 8
Day 8
Duration (Days) of Ventilation Support Over 26 Weeks
26 weeks
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ANX005 Treatment Group - Dose 1EXPERIMENTALParticipants will receive a single IV infusion of ANX005 (Dose 1) on Day 1.
ANX005 Treatment Group - Dose 2EXPERIMENTALParticipants will receive a single IV infusion of ANX005 (Dose 2) on Day 1.
Placebo GroupPLACEBO_COMPARATORParticipants will receive a single IV infusion of placebo on Day 1.
ANX005EXPERIMENTALParticipants will receive two once-weekly doses of ANX005 at specific time points
Open Label Treatment ArmEXPERIMENTALOne (1) dose of ANX005, 75 mg/kg, will be administered IV. IVIg, 0.4 g/kg, will be administered for 5 consecutive Days.
Interventions
NameTypeDescription
ANX005DRUGSolution for intravenous infusion
PlaceboDRUGSolution for intravenous infusion
Intravenous immunoglobulinDRUGinvestigational drug
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Eligibility Criteria
Age Range16 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites11

Inclusion Criteria: * Diagnosis of GBS according to the National Institute of Neurological Disorders and Stroke Diagnostic Criteria for Guillain-Barré Syndrome. * Onset of GBS-related weakness ≤10 days prior to start of infusion. * GBS-DS score of 3,4, or 5 at screening and at Day 1 prior to infusi...

Countries:BangladeshPhilippinesUnited StatesAustraliaAustriaBulgariaCanadaDenmark
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