Pegcetacoplan

Generic Name: Pegcetacoplan

Brand Names: Empaveli, Syfovre, Aspaveli

Drug Type: Biotech

CAS Number: 2019171-69-6

Unique Ingredient Identifier: TO3JYR3BOU

Background: Pegcetacoplan is a complement inhibitor indicated in the treatment of paroxysmal nocturnal hemoglobinuria (PNH). Prior to its FDA approval, patients with PNH were typically treated with the C5 inhibiting monoclonal antibody eculizumab. Patients given eculizumab experienced less hemolysis caused by the membrane attack complex, but were still somewhat susceptible to hemolysis caused by C3b opsonization. Pegcetacoplan was developed out of a need for an inhibitor of complement mediated hemolysis further upstream of C5. Pegcetacoplan is a pegylated C3 inhibitor that can disrupt the processes leading to both forms of hemolysis that threaten patients with PNH.

Pegcetacoplan for subcutaneous use was granted FDA approval on 14 May 2021. In February 2023, pegcetacoplan for intravitreal use was approved by the FDA for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration.

Indication: Pegcetacoplan is indicated to treat adults with paroxysmal nocturnal hemoglobinuria (PNH). It is also indicated to treat geographic atrophy (GA) secondary to age-related macular degeneration.

Associated Conditions: Geographic Atrophy Secondary to Age-related Macular Degeneration, Paroxysmal Nocturnal Haemoglobinuria (PNH)

Clinical Trials

Pegcetacoplan (APL-2) in Neovascular AMD

Phase 1

Terminated

Conditions: Neovascular Age-related Macular Degeneration

Interventions: Drug: Pegcetacoplan

First Posted Date: 2018-03-14

Last Posted Date: 2020-09-16

Lead Sponsor: Apellis Pharmaceuticals, Inc.

Target Recruit Count: 17

Registration Number: NCT03465709

Locations: 🇺🇸
Apellis Clinical Site, Houston, Texas, United States

Phase II Study Assessing Safety and Efficacy of APL-2 in Glomerulopathies

Phase 2

Completed

Conditions: IgA Nephropathy
Membranous Nephropathy
Dense Deposit Disease
Lupus Nephritis
C3 Glomerulonephritis

Interventions: Drug: APL-2

First Posted Date: 2018-03-05

Last Posted Date: 2025-02-13

Lead Sponsor: Apellis Pharmaceuticals, Inc.

Target Recruit Count: 21

Registration Number: NCT03453619

Locations: 🇺🇸
Horizon Research Group, Coral Gables, Florida, United States
🇺🇸
Northwest Louisiana Nephrology LLC, Shreveport, Louisiana, United States
🇺🇸
Washington Nephrology Associates, Alexandria, Virginia, United States

and more 11 locations

Study to Assess the Safety, Tolerability, Efficacy and PK of APL-2 in Patients With Warm Type Autoimmune Hemolytic Anemia (wAIHA) or Cold Agglutinin Disease (CAD)

Phase 2

Completed

Conditions: Cold Agglutinin Disease
Warm Autoimmune Hemolytic Anemia

Interventions: Drug: APL-2

First Posted Date: 2017-07-24

Last Posted Date: 2024-12-12

Lead Sponsor: Apellis Pharmaceuticals, Inc.

Target Recruit Count: 24

Registration Number: NCT03226678

Locations: 🇺🇸
East Carolina University, Greenville, North Carolina, United States
🇺🇸
Mayo Clinic, Rochester, Minnesota, United States
🇺🇸
University of Tennessee Medical Center, Knoxville, Tennessee, United States

and more 5 locations

Pilot Study to Assess Safety, Preliminary Efficacy and Pharmacokinetics of S.C. Pegcetacoplan (APL-2) in PNH Subjects.

Phase 1

Completed

Conditions: Paroxysmal Nocturnal Hemoglobinuria

Interventions: Drug: Pegcetacoplan

First Posted Date: 2015-10-28

Last Posted Date: 2021-01-11

Lead Sponsor: Apellis Pharmaceuticals, Inc.

Target Recruit Count: 23

Registration Number: NCT02588833

Locations: 🇳🇿
Waikato Hospital, Hamilton, Waikato, New Zealand
🇲🇾
Hospital Ampang, Ampang, Selangor, Malaysia
🇹🇭
Ramathibodi Hospital, Bangkok, Thailand

and more 4 locations

Study of Pegcetacoplan (APL-2) Therapy in Patients With Geographic Atrophy

Phase 2

Completed

Conditions: Geographic Atrophy

Interventions: Other: Sham Procedure
Drug: Pegcetacoplan

First Posted Date: 2015-07-20

Last Posted Date: 2020-10-06

Lead Sponsor: Apellis Pharmaceuticals, Inc.

Target Recruit Count: 246

Registration Number: NCT02503332

Locations: 🇺🇸
The Gavin Herbert Eye Institute/UC Irvine, Irvine, California, United States
🇺🇸
University of Southern California - USC Eye Institute, Los Angeles, California, United States
🇺🇸
Associated Retinal Consultants, PC, Traverse City, Michigan, United States

and more 42 locations

Assessment of Safety, Tolerability and Pharmacokinetics of Intravitreal Pegcetacoplan (APL-2) for Patients With Wet AMD

Phase 1

Completed

Conditions: Neovascular Age-Related Macular Degeneration

Interventions: Drug: Pegcetacoplan

First Posted Date: 2015-06-03

Last Posted Date: 2020-10-06

Lead Sponsor: Apellis Pharmaceuticals, Inc.

Target Recruit Count: 13

Registration Number: NCT02461771

Locations: 🇺🇸
United States, Florida, Miami, Florida, United States
🇺🇸
United States, California, Beverly Hills, California, United States
🇦🇺
Australia, New South Wells, Parramatta, New South Wales, Australia

and more 1 locations

A Phase I Study to Assess the Safety of Pegcetacoplan (APL-2) as an Add-On to Standard of Care in Subjects With PNH

Phase 1

Completed

Conditions: Paroxysmal Nocturnal Hemoglobinuria (PNH)

Interventions: Drug: Pegcetacoplan

First Posted Date: 2014-10-15

Last Posted Date: 2021-01-08

Lead Sponsor: Apellis Pharmaceuticals, Inc.

Target Recruit Count: 9

Registration Number: NCT02264639

Locations: 🇺🇸
University of Southern California Norris Comprehensive Cancer Center, Los Angeles, California, United States
🇺🇸
Lakes Research, Miami Lakes, Florida, United States
🇺🇸
Cleveland Clinic, Cleveland, Ohio, United States

and more 4 locations

Drug Development Updates

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