Pozelimab

Summary

Pozelimab (CHEMBL4298086) is an approved antibody (ATC L04AJ11); indicated across 4 conditions including protein-losing enteropathy and myasthenia gravis.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Antibody
• ATC class: L04AJ11
• Indications: 4 conditions
• Clinical trials: 21

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL4298086
Name	Pozelimab
Type	Antibody
Max phase	4
ATC	L04AJ11

Also known as: Pozelimab, Pozelimab-bbfg, REGN-3918, Regn3918, REGN3918, Veopoz, POZELIMAB

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

1 approved indication. FDA phase 4, plus an anticancer drug’s labelled cancer uses (which ChEMBL often logs at phase 3).

Indication	Phase	MONDO	EFO
protein-losing enteropathy	4	MONDO:0009174	HP:0002243

2 diseases in clinical trials (phase 1–3, investigational — not approved indications). Highest ChEMBL trial phase per disease; a non-cancer approved use is occasionally logged at phase 3 here.

Disease (in trials)	Phase	MONDO	EFO
myasthenia gravis	3	MONDO:0009688	EFO:0004991
hemoglobinuria	3	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: 21.

Phase distribution

Phase	Trials
PHASE3	7
PHASE1	5
PHASE2	3
Not specified	3
PHASE4	1
PHASE2/PHASE3	1
EARLY_PHASE1	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT07142343	PHASE4	RECRUITING	A Study to Test the Safety of Pozelimab in Pediatric Participants 1 to 5 Years of Age With a Rare Disease Called CHAPLE (Complement Hyperactivation, Angiopathic Thrombosis, Protein-losing Enteropathy) Disease
NCT05070858	PHASE3	ACTIVE_NOT_RECRUITING	A Study to Test How Safe Pozelimab and Cemdisiran Combination Therapy and Cemdisiran Alone Are and How Well They Work in Adult Patients With Generalized Myasthenia Gravis
NCT05133531	PHASE3	ACTIVE_NOT_RECRUITING	A Study to Evaluate How Safe Pozelimab + Cemdisiran Combination Therapy is and How Well it Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Not Recently Received or Have Not Received Complement Inhibitor Treatment
NCT05744921	PHASE3	RECRUITING	A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works
NCT06541704	PHASE3	RECRUITING	A Study Investigating Subcutaneously Administered Pozelimab in Combination With Cemdisiran or Cemdisiran Alone in Adult Participants With Geographic Atrophy
NCT07154745	PHASE3	RECRUITING	A Study to Evaluate How Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Whose Current Treatment is Not Working Efficiently
NCT04162470	PHASE3	TERMINATED	REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate Its Long Term Safety, Efficacy and Tolerability.
NCT04209634	PHASE2/PHASE3	COMPLETED	Open-Label Efficacy and Safety Study of Pozelimab in Patients With CD55-Deficient Protein-Losing Enteropathy (CHAPLE Disease)
NCT05131204	PHASE3	TERMINATED	Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria
NCT03946748	PHASE2	COMPLETED	Study to Evaluate the Efficacy and Safety of REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
NCT04811716	PHASE2	COMPLETED	Pozelimab and Cemdisiran Combination Treatment in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy
NCT04888507	PHASE2	COMPLETED	Pozelimab and Cemdisiran Combination Therapy in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Switch From Eculizumab Therapy
NCT07230834	PHASE1	RECRUITING	Intravitreal (IVT) Pozelimab for Geographic Atrophy (GA) in Adult Participants
NCT03115996	PHASE1	COMPLETED	Study of the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of REGN3918 in Healthy Volunteers
NCT04491838	PHASE1	COMPLETED	Study of a Single Subcutaneous Dose of Pozelimab Produced From Two Different Manufacturing Processes in Healthy Adult Participants
NCT04601844	PHASE1	COMPLETED	Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Pozelimab in Combination With Cemdisiran in Healthy Adult Volunteers
NCT04940364	PHASE1	COMPLETED	A Study to Examine the Safety, Tolerability and Biological Effects of Single Doses of Subcutaneously and Intravenously Administered Pozelimab as Monotherapy and in Combination With Single Doses of Subcutaneously Administered Cemdisiran in Adult Japanese Healthy Volunteers
NCT06479863	EARLY_PHASE1	RECRUITING	Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Sporadic Inclusion Body Myositis
NCT05232110	Not specified	APPROVED_FOR_MARKETING	Compassionate Use of Pozelimab
NCT06003881	Not specified	APPROVED_FOR_MARKETING	Compassionate Use of Pozelimab in Patients With CD55-Deficient Protein-Losing Enteropathy
NCT06028594	Not specified	NO_LONGER_AVAILABLE	Compassionate Use of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria

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).