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AAVCAGsCD59

Phase 1

Dry Age-related Macular Degeneration | Monoclonal antibody | Ophthalmology |Johnson & Johnson|Last Updated: Mar 30, 2025

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
CONTROLLEDBiomarker
Total Trials1
Total Enrollment17
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03144999Treatment of Advanced Dry Age Related Macular Degeneration With AAVCAGsCD59PHASE1 COMPLETED 17Mar 29, 2017Dec 9, 2019Mar 30, 20251 United States
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Study Endpoints
Primary Endpoints
Number of participants experiencing ocular and systemic adverse events as graded by CTCAE v4.0
26 Weeks

Measure intraocular inflammation, ocular changes and systemic side effects following a single intraocular injection of AAVCAGsCD59

Secondary Endpoints
Evaluate the change in area of GA in eyes with dry AMD
26 Weeks
Evaluate the rate of growth of GA in eyes with dry AMD
26 Weeks
Incidence of conversion of dry AMD to wet AMD
26 Weeks
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeOTHER
Treatment Arms
ArmTypeDescription
Low DoseEXPERIMENTALAAVCAGsCD59
Mid DoseEXPERIMENTALAAVCAGsCD59
High DoseEXPERIMENTALAAVCAGsCD59
Interventions
NameTypeDescription
AAVCAGsCD59BIOLOGICALAAVCAGsCD59 is administered as a single intravitreal injection in an office setting
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Eligibility Criteria
Age Range50 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: * Men or women 50 years of age or older * Advanced dry AMD with GA in the study eye * BCVA Snellen equivalent of 20/80 or worse in the study eye using ETDRS charts at a starting distance of 4m after the first 3 patients are enrolled and demonstrate favorable safety data * Total ...

Countries:United States
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