Details of the SBP

General Information of Synthetic Binding Protein (SBP) (ID: SBP003220)
SBP Name	Macrocyclic peptide Pegcetacoplan
Synonyms	APL-2
Design Method	Traditional methods (Site-directed mutagenesis and/or Directed evolution)
Highest Status	Marketed

Protein Scaffold Information of This SBP
Scaffold ID	PS044 Scaffold Info	[1] , [2]
Scaffold Name	Macrocyclic peptide
Scaffold Class	Non-Antibody
Fold Type	Beta-Sheets + Loops

Binding Target(s) of This SBP (BTS)

BTS Name	Details	Mechanism	Application	Affinity	Research Organization	Ref
Complement C3	BTS Info	Inhibitor	Paroxysmal nocturnal hemoglobinuria [ICD-11: 3A21.0]; Nephritis [ICD-11: GB40]; Age-related macular degeneration [ICD-11: 9B75.0]; Amyotrophic lateral sclerosis [ICD-11: 8B60.0]; Autoimmune haemolytic anaemia [ICD-11: 3A20.0]; IgA nephropathy [ICD-11: MF8Y]; Membranous glomerulonephritis [ICD-11: MF8Y]	N.A.	Apellis Pharmaceuticals	[1] , [2]

Clinical Trial Information of This SBP
NCT02264639	Click to show the Detail
Indication	Paroxysmal Nocturnal Hemoglobinuria (PNH)
Phase	Phase I
Title	An Open Label, Single and Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of APL-2 as an Add-On to Standard of Care in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH).
Status	Completed
Sponsor	Apellis Pharmaceuticals
NCT02461771	Click to show the Detail
Indication	Neovascular Age-related Macular Degeneration
Phase	Phase I
Title	Assessment of Safety, Tolerability and Pharmacokinetics of Intravitreal APL-2 Therapy for Neovascular Age-Related Macular Degeneration (AMD)
Status	Completed
Sponsor	Apellis Pharmaceuticals
NCT02503332	Click to show the Detail
Indication	Geographic Atrophy (GA)
Phase	Phase II
Title	A Phase II, Multicenter, Randomized, Single-Masked, Sham-Controlled Study of Safety, Tolerability and Evidence of Activity of Intravitreal APL-2 Therapy in Patients With Geographic Atrophy (GA)
Status	Completed
Sponsor	Apellis Pharmaceuticals
NCT02588833	Click to show the Detail
Indication	Paroxysmal Nocturnal Hemoglobinuria (PNH)
Phase	Phase I
Title	A Phase Ib, Open Label, Multiple Ascending Dose, Pilot Study to Assess the Safety, Preliminary Efficacy and Pharmacokinetics of Subcutaneously Administered APL-2 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Status	Completed
Sponsor	Apellis Pharmaceuticals
NCT03226678	Click to show the Detail
Indication	Warm Autoimmune Hemolytic Anemia; Cold Agglutinin Disease
Phase	Phase II
Title	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)
Status	Completed
Sponsor	Apellis Pharmaceuticals, Inc.
NCT03453619	Click to show the Detail
Indication	IgA Nephropathy; Lupus Nephritis; Membranous Nephropathy; C3 Glomerulonephritis; Dense Deposit Disease
Phase	Phase II
Title	Phase II Study Assessing Safety and Efficacy of APL-2 in Glomerulopathies
Status	Active, not recruiting
Sponsor	Apellis Pharmaceuticals, Inc.
NCT03465709	Click to show the Detail
Indication	Neovascular Age-related Macular Degeneration
Phase	Phase I; Phase II
Title	An 18-Month Phase Ib/II Multi-Center, Open Label Study to Evaluate the Safety of Intravitreal APL-2 Therapy in Patients With Neovascular Age-Related Macular Degeneration (AMD)
Status	Terminated
Sponsor	Apellis Pharmaceuticals
NCT03500549	Click to show the Detail
Indication	Paroxysmal Nocturnal Hemoglobinuria
Phase	Phase III
Title	Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Status	Completed
Sponsor	Apellis Pharmaceuticals, Inc.
NCT03525600	Click to show the Detail
Indication	Geographic Atrophy
Phase	Phase III
Title	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
Status	Active, not recruiting
Sponsor	Apellis Pharmaceuticals Inc
NCT03525613	Click to show the Detail
Indication	Geographic Atrophy
Phase	Phase III
Title	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
Status	Active, not recruiting
Sponsor	Apellis Pharmaceuticals Inc
NCT03531255	Click to show the Detail
Indication	Paroxysmal Nocturnal Hemoglobinuria (PNH)
Phase	Phase III
Title	An Open Label, Non-Randomized, Multi-Center Extension Study to Evaluate the Long-Term Safety and Efficacy of?Pegcetacoplan?in the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH)
Status	Enrolling by invitation
Sponsor	Apellis Pharmaceuticals
NCT03593200	Click to show the Detail
Indication	Paroxysmal Nocturnal Hemoglobinuria (PNH)
Phase	Phase II
Title	Phase IIa, Open Label, Multiple Dose Study to Assess the Safety, Efficacy and Pharmacokinetics of Subcutaneously Administered APL-2 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH).
Status	Completed
Sponsor	Apellis Pharmaceuticals
NCT03777332	Click to show the Detail
Indication	Geographic Atrophy Secondary to Age-related Macular Degeneration
Phase	Phase I
Title	A 24-Month Phase Ib Multi-Center, Open Label, Single Arm Study to Evaluate the Safety of Intravitreal APL-2 Therapy in Patients Diagnosed With Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration (AMD)
Status	Active, not recruiting
Sponsor	Apellis Pharmaceuticals
NCT04085601	Click to show the Detail
Indication	Paroxysmal Nocturnal Hemoglobinuria
Phase	Phase III
Title	A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With PNH
Status	Active, not recruiting
Sponsor	Apellis Pharmaceuticals
NCT04572854	Click to show the Detail
Indication	Renal Transplant; Complement 3 Glomerulopathy (C3G); Dense Deposit Disease; Membranoproliferative Glomerulonephritis (MPGN); Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN)
Phase	Phase II
Title	Study Assessing the Safety and Efficacy of Pegcetacoplan in Post-Transplant Recurrence of C3G or IC-MPGN
Status	Recruiting
Sponsor	Apellis Pharmaceuticals
NCT04579666	Click to show the Detail
Indication	Amyotrophic Lateral Sclerosis (ALS)
Phase	Phase II
Title	MERIDIAN: A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Adults With Amyotrophic Lateral Sclerosis (ALS)
Status	Recruiting
Sponsor	Apellis Pharmaceuticals
NCT04729062	Click to show the Detail
Indication	Paroxysmal Nocturnal Hemoglobinuria (PNH)
Phase	.
Title	Pegcetacoplan Early Access Program for PNH
Status	Not Available
Sponsor	Apellis Pharmaceuticals
NCT04770545	Click to show the Detail
Indication	Geographic Atrophy Secondary to Age-related Macular Degeneration
Phase	Phase III
Title	An Extension Study to Evaluate the Long-term Safety and Efficacy of Pegcetacoplan (APL-2) in Subjects With Geographic Atrophy Secondary to AMD
Status	Enrolling by invitation
Sponsor	Apellis Pharmaceuticals
NCT04901936	Click to show the Detail
Indication	Paroxysmal Nocturnal Hemoglobinuria (PNH); Paroxysmal Hemoglobinuria
Phase	Phase II
Title	A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Status	Recruiting
Sponsor	Apellis Pharmaceuticals Inc
NCT04919629	Click to show the Detail
Indication	Fallopian Tube Carcinosarcoma; Fallopian Tube Adenocarcinoma; Fallopian Tube Serous Adenocarcinoma; Ovarian Carcinosarcoma; Ovarian Clear Cell Adenocarcinoma; Ovarian Endometrioid Adenocarcinoma; Ovarian Serous Adenocarcinoma; Primary Peritoneal Adenocarcinoma; Recurrent Fallopian Tube Carcinoma; Recurrent Ovarian Carcinoma; Recurrent Primary Peritoneal Carcinoma
Phase	Phase II
Title	APL-2 and Pembrolizumab Versus APL-2, Pembrolizumab and Bevacizumab Versus Bevacizumab Alone for the Treatment of Recurrent Ovarian, Fallopian Tube, or Primary Peritoneal Cancer and Malignant Effusion
Status	Not Recruiting
Sponsor	Roswell Park Cancer Institute

References

1	C3 inhibition with pegcetacoplan in subjects with paroxysmal nocturnal hemoglobinuria treated with eculizumab. Am J Hematol. 2020 Nov;95(11):1334-1343.
2	Pegcetacoplan versus Eculizumab in Paroxysmal Nocturnal Hemoglobinuria. N Engl J Med. 2021 Mar 18;384(11):1028-1037.