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APL-2, Pegcetacoplan

Phase 3

Geographic Atrophy Secondary to Age-related Macular Degeneration | Small molecule | Ophthalmology |Apellis Pharmaceuticals, Inc.|Last Updated: Dec 17, 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
UNCONTROLLEDBiomarker
Total Trials1
Total Enrollment44
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT07214740Study to Evaluate a Pegcetacoplan (Syfovre®) Prefilled SyringePHASE3 COMPLETED 44Oct 24, 2025Dec 12, 2025Dec 17, 20255 United States
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Study Endpoints
Primary Endpoints
To evaluate safety and tolerability of intravitreal (IVT) pegcetacoplan administered via PFS by assessing incidence of ocular treatment-emergent adverse events (TEAEs) in the study eye
Up to Day 30
Secondary Endpoints
The number of IVT pegcetacoplan injections successfully administered using PFS as reported by retina specialists immediately following the injection
At Day 1
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Pegcetacoplan, 15 mg/100 μL (single dose), intravitreal injection via prefilled syringeEXPERIMENTALThis is a 30-day, phase 3b, single-arm, open-label, multicenter study to evaluate the safety of IVT pegcetacoplan administered via PFS in participants with geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The study consists of a screening/baseline visit at day 1, a follow-up call at day 7, and an end of study visit at day 30.
Interventions
NameTypeDescription
APL-2, PegcetacoplanDRUGComplement (C3) Inhibitor
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Eligibility Criteria
Age Range60 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites5

Inclusion Criteria: 1. Participants must be aged at least 60 years and have a diagnosis of GA secondary to AMD. 2. Participants must have adequate clarity of ocular media, adequate pupillary dilation, and fixation to permit the collection of good quality images in the study eye as determined by the...

Countries:United States
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