Recent Updates
Recently added Catalysts

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

Subscribe to view

Market & Valuation

Subscribe to view

Trial Design

UNCONTROLLED
Total Trials1
Total Enrollment44

FDA Designations

No designations recorded

Clinical trial landscape

APL-2, Pegcetacoplan · 1 trial · 1 indication

Phase 3 1
NCT07214740Study to Evaluate a Pegcetacoplan (Syfovre®) Prefilled SyringeGeographic Atrophy Secondary to Age-related Macular Degeneration
COMPLETED44 Analytics
PHASE3COMPLETED
Study to Evaluate a Pegcetacoplan (Syfovre®) Prefilled Syringe
Geographic Atrophy Secondary to Age-related Macular DegenerationUnlock trial analytics

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
Unlock Study Endpoints

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
Unlock Study Design Details

Eligibility Criteria

Age Range60 Years to 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
Unlock Eligibility Criteria

Frequently asked questions about APL-2, Pegcetacoplan

What is APL-2 used for?

APL-2, also known as pegcetacoplan, is an investigational therapy being studied for paroxysmal nocturnal hemoglobinuria, geographic atrophy, warm autoimmune hemolytic anemia, and IgA nephropathy. It is in Phase 3 clinical development for these conditions.

What does APL-2 target?

APL-2 targets the complement system. It is designed to inhibit complement component C3, which plays a role in the immune response. This mechanism is being investigated for conditions like geographic atrophy and paroxysmal nocturnal hemoglobinuria.

Who makes APL-2?

APL-2 is being developed by Apellis Pharmaceuticals, Inc., a biopharmaceutical company. The company's stock is traded under the ticker symbol APLS.

What phase is APL-2 in?

APL-2 is in Phase 3 clinical development. It is an investigational drug and has not been approved by regulatory authorities. Clinical trials are ongoing to evaluate its safety and efficacy for various indications.

What clinical trials is APL-2 in?

APL-2 has been studied in several Phase 3 trials. These include NCT03525600 and NCT03525613 for geographic atrophy secondary to age-related macular degeneration, NCT04085601 for paroxysmal nocturnal hemoglobinuria, and NCT07214740 for geographic atrophy using a prefilled syringe.

Is APL-2 the same as pegcetacoplan?

Yes, APL-2 is also known as pegcetacoplan. The drug is referred to by both names in clinical research and development contexts.