Zilucoplan

Summary

Zilucoplan (CHEMBL4298207) is a phase-3 clinical-stage protein (ATC L04AJ06); indicated across 5 conditions including myasthenia gravis and severe acute respiratory syndrome.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Protein
• ATC class: L04AJ06
• Indications: 5 conditions
• Clinical trials: 16

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL4298207
Name	Zilucoplan
Type	Protein
Max phase	3
ATC	L04AJ06

Also known as: RA101495, Zilucoplan, ZILUCOPLAN

Targets

Targets

No target linkage available.

Bioactivity

No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

5 indications (0 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

Indication	Trial phase	MONDO	EFO
myasthenia gravis	3	MONDO:0009688	EFO:0004991
severe acute respiratory syndrome	3	MONDO:0005091	MONDO:0100096
amyotrophic lateral sclerosis	2	MONDO:0004976	MONDO:0004976
hemoglobinuria	2	MONDO:0003656	MONDO:0100244

1 further indication record had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 16.

Phase distribution

Phase	Trials
PHASE3	6
PHASE2	5
PHASE2/PHASE3	3
PHASE1	2

Top trials by phase / activity

NCT	Phase	Status	Title
NCT04225871	PHASE3	ACTIVE_NOT_RECRUITING	Open-Label Extension of Zilucoplan in Subjects With Generalized Myasthenia Gravis
NCT04297683	PHASE2/PHASE3	RECRUITING	HEALEY ALS Platform Trial - Master Protocol
NCT06055959	PHASE2/PHASE3	RECRUITING	A Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis
NCT06435312	PHASE3	ENROLLING_BY_INVITATION	An Open-label Extension Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis
NCT04115293	PHASE3	COMPLETED	Safety, Tolerability, and Efficacy of Zilucoplan in Subjects With Generalized Myasthenia Gravis
NCT04436497	PHASE2/PHASE3	COMPLETED	HEALEY ALS Platform Trial - Regimen A Zilucoplan
NCT04590586	PHASE3	COMPLETED	Study of Multiple Candidate Agents for the Treatment of COVID-19 in Hospitalized Patients
NCT05514873	PHASE3	COMPLETED	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
NCT06471361	PHASE3	COMPLETED	A Study to Evaluate the Safe and Effective Use of a Zilucoplan Auto-injector by Study Participants With Generalized Myasthenia Gravis
NCT03030183	PHASE2	COMPLETED	Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients Who Have an Inadequate Response to Eculizumab
NCT03078582	PHASE2	COMPLETED	Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients
NCT03225287	PHASE2	TERMINATED	Extension Study of RA101495 for Patients With PNH Who Have Completed a Zilucoplan (RA101495) Clinical Study
NCT03315130	PHASE2	COMPLETED	Safety and Efficacy Study of RA101495 in Subjects With Generalized Myasthenia Gravis
NCT04025632	PHASE2	TERMINATED	Safety and Efficacy Study of Zilucoplan in Subjects With Immune-Mediated Necrotizing Myopathy
NCT06961747	PHASE1	RECRUITING	A Study to Assess Zilucoplan Concentration in Breast Milk of Healthy Lactating Women
NCT06511076	PHASE1	COMPLETED	A Study to Evaluate Zilucoplan Injected Subcutaneously Either by a Prefilled Syringe or an Auto-injector in Healthy Adult Participants

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).