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ARO-C3

Phase 1

C3 Glomerulopathy | Small molecule | Nephrology |Arrowhead Pharmaceuticals, Inc.|Last Updated: May 22, 2026

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
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment62
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05083364Study of ARO-C3 in Adult Healthy Volunteers and Patients With Complement Mediated Renal DiseasePHASE1 COMPLETED 62Feb 1, 2022Sep 10, 2025May 22, 202616 Australia, Georgia +5
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Study Endpoints
Primary Endpoints
Number of Participants with Adverse Events (AEs) and/or Serious Adverse Events (SAEs) at Day 169
up to day 169 (End of Study [EOS])
Secondary Endpoints
Pharmacokinetics (PK) of ARO-C3: Maximum Observed Plasma Concentration (Cmax)
up to 48 hours post-dose
PK of ARO-C3: Area under the Plasma Concentration Versus Time Curve from Zero to 24Hours (AUC0-24)
up to 48 hours post-dose
PK of ARO-C3: Area Under the Plasma Versus Time Concentration Curve from Zero to the Last Quantifiable Plasma Concentration (AUClast)
up to 48 hours post-dose
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ARO-C3 (Healthy Volunteers)EXPERIMENTAL1 or 2 doses of ARO-C3 by subcutaneous (sc) injection
Placebo (Healthy Volunteers)PLACEBO_COMPARATORplacebo calculated volume to match active treatment by sc injection
ARO-C3 (Adult Patients with C3G or IgAN)EXPERIMENTAL3 doses of ARO-C3 by sc injection
Interventions
NameTypeDescription
ARO-C3DRUGARO-C3 for sc injection
PlaceboDRUGsterile normal saline (0.9% NaCl) for sc injection
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Eligibility Criteria
Age Range18 Years — 70 Years
SexALL
Healthy VolunteersYes
Study Sites16

Inclusion Criteria (All Participants): * Willing to provide written informed consent and to comply with study requirements * Female participants must be non-pregnant/non-lactating * Healthy volunteers must be willing to be vaccinated with a meningococcal and pneumococcal vaccine. C3G and IgAN parti...

Countries:AustraliaGeorgiaGermanyNew ZealandSouth KoreaThailandUnited Kingdom
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