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ANX005

Phase 2

Warm Autoimmune Hemolytic Anemia (wAIHA) | Small molecule | Hematology |Annexon, Inc.|Last Updated: Jan 19, 2024

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
UNCONTROLLEDBiomarker
Total Trials1
Total Enrollment6
FDA Designations
ORPHAN_DRUGFAST_TRACK
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04691570Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ANX005 in Participants With Warm Autoimmune Hemolytic Anemia (wAIHA)PHASE2 COMPLETED 6Nov 10, 2021Jan 17, 2023Jan 19, 20241 United States
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Study Endpoints
Primary Endpoints
Safety: Treatment-emergent adverse events (TEAEs)
Up to Week 16

Number of participants with TEAEs, defined as any adverse event with an onset on or after the day of infusion through 16 weeks after the infusion

Change in disease activity biomarkers
Baseline to Day 71

Change in hemoglobin, lactate dehydrogenase, bilirubin, reticulocyte count and haptoglobin from baseline

Secondary Endpoints
Plasma concentrations
Up to Day 71
Change in complement system biomarkers
Baseline to Day 71
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ANX005EXPERIMENTALParticipants will receive two once-weekly doses of ANX005 at specific time points
Interventions
NameTypeDescription
ANX005DRUGANX005 is provided as a solution for IV infusion
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: * Male or non-pregnant, non-lactating female ≥18 years of age (no maximum age). * Diagnosis of wAIHA at least 3 months prior to screening with a direct antiglobulin test (DAT) ≥1 positive for immunoglobulin G (IgG)±C3, or a diagnosis of mixed autoimmune hemolytic anemia (AIHA) t...

Countries:United States
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