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APL-2, Pegcetacoplan

Apellis Pharmaceuticals, Inc. · Phase 3 active Small molecule

Pegcetacoplan is a complement C3 inhibitor that blocks the activation and amplification of the complement cascade by targeting C3.

Pegcetacoplan is a complement C3 inhibitor that blocks the activation and amplification of the complement cascade by targeting C3. Used for Geographic atrophy (age-related macular degeneration), C3 glomerulopathy, Post-transplant thrombotic microangiopathy.

At a glance

Generic nameAPL-2, Pegcetacoplan
SponsorApellis Pharmaceuticals, Inc.
Drug classComplement C3 inhibitor
TargetComplement C3
ModalitySmall molecule
Therapeutic areaImmunology / Rare Disease
PhasePhase 3

Mechanism of action

APL-2 (pegcetacoplan) is a synthetic cyclic peptide that binds to complement component C3 and prevents its cleavage into C3a and C3b, thereby inhibiting both the classical and alternative complement pathways. By blocking C3 activation, the drug reduces downstream complement-mediated inflammation and tissue damage, which is implicated in several rare and systemic diseases.

Approved indications

Common side effects

Key clinical trials

Primary sources

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SourceUsed for
ClinicalTrials.govTrial enrolment, design, endpoints, results