Details of the SBP

General Information of Synthetic Binding Protein (SBP) (ID: SBP000296)
SBP Name	Macrocyclic peptide Zilucoplan
Synonyms	RA-101495
Design Method	Traditional methods (Site-directed mutagenesis and/or Directed evolution)
Highest Status	Phase III

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 C5	BTS Info	Inhibitor	Myasthenia gravis [ICD-11: 8C60.Z]	N.A.	Ra Pharmaceuticals	[1] , [2]

Clinical Trial Information of This SBP
NCT03030183	Click to show the Detail
Indication	Paroxysmal Nocturnal Hemoglobinuria (PNH)
Phase	Phase II
Title	A Phase 2 Multicenter, Open-Label, Uncontrolled Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of RA101495 in Subjects With Paroxysmal Nocturnal Hemoglobinuria Who Have an Inadequate Response to Eculizumab
Status	Completed
Sponsor	Ra Pharmaceuticals
NCT03078582	Click to show the Detail
Indication	Paroxysmal Nocturnal Hemoglobinuria (PNH)
Phase	Phase II
Title	Phase 2 Multicenter, Open-Label, Uncontrolled Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of RA101495 in Subjects With Paroxysmal Nocturnal Hemoglobinuria
Status	Completed
Sponsor	Ra Pharmaceuticals
NCT03225287	Click to show the Detail
Indication	Paroxysmal Nocturnal Hemoglobinuria (PNH)
Phase	Phase II
Title	A Multicenter, Open-label, Uncontrolled, Extension Study of RA101495 in Subjects With Paroxysmal Nocturnal Hemoglobinuria Who Have Completed a RA101495 Clinical Study
Status	Active, not recruiting
Sponsor	Ra Pharmaceuticals
NCT03315130	Click to show the Detail
Indication	Generalized Myasthenia Gravis
Phase	Phase II
Title	A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of RA101495 in Subjects With Generalized Myasthenia Gravis
Status	Completed
Sponsor	Ra Pharmaceuticals
NCT04025632	Click to show the Detail
Indication	Immune-Mediated Necrotizing Myopathy
Phase	Phase II
Title	A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, and Efficacy of?Zilucoplan?in Subjects With Immune-Mediated Necrotizing Myopathy
Status	Active, not recruiting
Sponsor	Ra Pharmaceuticals
NCT04115293	Click to show the Detail
Indication	Generalized Myasthenia Gravis
Phase	Phase III
Title	A Phase 3, Multicenter, Randomized, Double Blind, Placebo-Controlled Study to Confirm the Safety, Tolerability, and Efficacy of?Zilucoplan?in Subjects With Generalized Myasthenia Gravis
Status	Recruiting
Sponsor	Ra Pharmaceuticals
NCT04225871	Click to show the Detail
Indication	Generalized Myasthenia Gravis
Phase	Phase III
Title	A Phase 3, Multicenter, Open-Label Extension Study of?Zilucoplan?in Subjects With Generalized Myasthenia Gravis
Status	Recruiting
Sponsor	Ra Pharmaceuticals
NCT04436497	Click to show the Detail
Indication	Amyotrophic Lateral Sclerosis
Phase	Phase II; Phase III
Title	HEALEY ALS Platform Trial-Regimen A?Zilucoplan
Status	Enrolling by invitation
Sponsor	Merit E. Cudkowicz
NCT05514873	Click to show the Detail
Indication	Generalized Myasthenia Gravis
Phase	Phase III
Title	An Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of Subcutaneous Zilucoplan in Participants With Generalized Myasthenia Gravis Who Were Previously Receiving Intravenous Complement Component 5 Inhibitors
Status	Active, not recruiting
Sponsor	UCB Biopharma SRL
NCT06055959	Click to show the Detail
Indication	Generalized Myasthenia Gravis
Phase	Phase II; Phase III
Title	A Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis
Status	Not yet recruiting
Sponsor	UCB Biopharma SRL

References

1	ClinicalTrials.gov (NCT04436497) HEALEY ALS Platform Trial - Regimen A Zilucoplan
2	ClinicalTrials.gov (NCT04225871) Open-Label Extension of Zilucoplan in Subjects With Generalized Myasthenia Gravis