DeepMed Research

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

C3G/Primary IC-MPGN EAP

Conditions: C3G
IC-MPGN
C3 Glomerulopathy
C3 Glomerulonephritis
Complement 3 Glomerulopathy
Complement 3 Glomerulopathy (C3G)
Complement 3 Glomerulonephritis
Dense Deposit Disease
DDD
Membranoproliferative Glomerulonephritis

First Posted Date: 2021-01-28

Last Posted Date: 2024-07-26

Lead Sponsor: Apellis Pharmaceuticals, Inc.

Registration Number: NCT04729062

MERIDIAN: A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Adults With Amyotrophic Lateral Sclerosis (ALS)

Phase 2

Terminated

Conditions: Amyotrophic Lateral Sclerosis
Motor Neuron Disease

Interventions: Drug: Pegcetacoplan (APL-2)
Other: Placebo

First Posted Date: 2020-10-08

Last Posted Date: 2025-04-24

Lead Sponsor: Apellis Pharmaceuticals, Inc.

Target Recruit Count: 249

Registration Number: NCT04579666

Locations: 🇦🇺
Brain and Mind Centre, Camperdown, New South Wales, Australia
🇺🇸
Johns Hopkins, Baltimore, Maryland, United States
🇺🇸
Hospital for Special Surgery, New York, New York, United States

and more 57 locations

Study Assessing the Safety and Efficacy of Pegcetacoplan in Post-Transplant Recurrence of C3G or IC-MPGN

Phase 2

Active, not recruiting

Conditions: Renal Transplant
Dense Deposit Disease (DDD)
Membranoproliferative Glomerulonephritis (MPGN)
Complement 3 Glomerulonephritis
Membranoproliferative Glomerulonephritis
C3 Glomerulonephritis
C3G
Complement 3 Glomerulopathy
Complement 3 Glomerulopathy (C3G)
Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN)

Interventions: Drug: Pegcetacoplan

First Posted Date: 2020-10-01

Last Posted Date: 2025-03-30

Lead Sponsor: Apellis Pharmaceuticals, Inc.

Target Recruit Count: 13

Registration Number: NCT04572854

Locations: 🇮🇹
Istituto di Ricerche Farmacologiche Mario Negri IRCCS, Ranica, Italy
🇺🇸
Children's Hospital Colorado, Aurora, Colorado, United States
🇺🇸
Mayo Clinic Arizona, Phoenix, Arizona, United States

and more 22 locations

A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With PNH

Phase 3

Completed

Conditions: Paroxysmal Nocturnal Hemoglobinuria

Interventions: Drug: APL-2

First Posted Date: 2019-09-11

Last Posted Date: 2022-10-21

Lead Sponsor: Apellis Pharmaceuticals, Inc.

Target Recruit Count: 53

Registration Number: NCT04085601

Locations: 🇹🇭
Thammasat University Hospital, Pathum Thani, Thailand
🇵🇭
The Medical City, Pasig City, Philippines
🇵🇱
Independent Public Clinical Hospital, Lubin, Poland

and more 26 locations

Study to Evaluate the Safety of Intravitreal APL-2 in Patients Diagnosed With Geographic Atrophy

Phase 1

Completed

Conditions: Geographic Atrophy

Interventions: Drug: PEGCETACOPLAN (APL-2)

First Posted Date: 2018-12-17

Last Posted Date: 2021-11-10

Lead Sponsor: Apellis Pharmaceuticals, Inc.

Target Recruit Count: 19

Registration Number: NCT03777332

Locations: 🇺🇸
Retina Consultants of Houston, The Woodlands, Texas, United States
🇺🇸
Ophthalmic Consultants of Boston, Boston, Massachusetts, United States
🇺🇸
California Retina Consultants, Bakersfield, California, United States

and more 2 locations

A Phase IIa Study to Assess the Safety, Efficacy, and Pharmacokinetics of Subcutaneously Administered Pegcetacoplan (APL-2) in Subjects With PNH

Phase 2

Completed

Conditions: PNH

Interventions: Drug: Pegcetacoplan

First Posted Date: 2018-07-20

Last Posted Date: 2020-12-22

Lead Sponsor: Apellis Pharmaceuticals, Inc.

Target Recruit Count: 4

Registration Number: NCT03593200

Locations: 🇧🇬
Acibadem City Clinic MHAT Tokuda EAD Sofia, Sofia, Bulgaria
🇷🇸
Klinički centar Srbije, Belgrade, Serbia
🇧🇬
Specialized Hospital for Active Treatment of Hematologic Diseases EAD, Sofia, Sofia, Bulgaria

Pegcetacoplan Long Term Safety and Efficacy Extension Study

Phase 3

Active, not recruiting

Conditions: PNH

Interventions: Drug: Pegcetacoplan

First Posted Date: 2018-05-21

Last Posted Date: 2025-03-28

Lead Sponsor: Apellis Pharmaceuticals, Inc.

Target Recruit Count: 160

Registration Number: NCT03531255

Locations: 🇧🇬
Acibadem City Clinic Tokuda Hospital, Sofia, Bulgaria
🇺🇸
Baptist Cancer Center, Memphis, Tennessee, United States
🇯🇵
Japanese Red Cross Nagoya Daini Hospital, Showa-ku, Aichi, Japan

and more 59 locations

A Study to Compare the Efficacy and Safety of Intravitreal APL-2 Therapy With Sham Injections in Patients With Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration

Phase 3

Completed

Conditions: Geographic Atrophy

Interventions: Drug: APL-2
Other: Sham Procedure

First Posted Date: 2018-05-15

Last Posted Date: 2023-07-06

Lead Sponsor: Apellis Pharmaceuticals, Inc.

Target Recruit Count: 637

Registration Number: NCT03525613

Locations: 🇺🇸
Southeast Retina Center, PC, Augusta, Georgia, United States
🇺🇸
Mid Atlantic Retina Specialists, Hagerstown, Maryland, United States
🇺🇸
National Ophthalmic Research Institute (Retina Consultants of Southwest Florida), Fort Myers, Florida, United States

and more 108 locations

Study to Compare the Efficacy and Safety of Intravitreal APL-2 Therapy With Sham Injections in Patients With Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration

Phase 3

Completed

Conditions: Geographic Atrophy

Interventions: Drug: APL-2
Other: Sham Procedure

First Posted Date: 2018-05-15

Last Posted Date: 2023-06-18

Lead Sponsor: Apellis Pharmaceuticals, Inc.

Target Recruit Count: 621

Registration Number: NCT03525600

Locations: 🇺🇸
Long Island Vitreoretinal Consultants, Great Neck, New York, United States
🇺🇸
Associated Retinal Consultants PC, Traverse City, Michigan, United States
🇺🇸
Sierra Eye Associates, Reno, Nevada, United States

and more 136 locations

Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Phase 3

Completed

Conditions: Paroxysmal Nocturnal Hemoglobinuria

Interventions: Drug: Pegcetacoplan
Drug: Soliris

First Posted Date: 2018-04-18

Last Posted Date: 2022-03-25

Lead Sponsor: Apellis Pharmaceuticals, Inc.

Target Recruit Count: 80

Registration Number: NCT03500549

Locations: 🇫🇷
Centre Hospitalier de Saint-Quentin, Saint-Quentin, France
🇺🇸
Cancer Specialists of North Florida, Jacksonville, Florida, United States
🇺🇸
Cancer & Hematology Centers of Western Michigan, Grand Rapids, Michigan, United States

and more 49 locations

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