Rusfertide

Summary

Rusfertide (CHEMBL4650507) is a phase-3 clinical-stage protein; indicated across 3 conditions including acquired polycythemia vera and hereditary hemochromatosis.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Protein
• Indications: 3 conditions
• Clinical trials: 8

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL4650507
Name	Rusfertide
Type	Protein
Max phase	3

Also known as: Ptg-300, PTG-300, PTG-300FB, Rusfertide, RUSFERTIDE

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

3 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
acquired polycythemia vera	3	MONDO:0009891	EFO:0002429
hereditary hemochromatosis	2	MONDO:0006507	MONDO:0006507

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

Clinical trials

Total trials: 8.

Phase distribution

Phase	Trials
PHASE2	5
PHASE3	2
PHASE1	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT05210790	PHASE3	ACTIVE_NOT_RECRUITING	A Phase 3 Study of Rusfertide in Patients With Polycythemia Vera
NCT06033586	PHASE3	ACTIVE_NOT_RECRUITING	Study to Evaluate the Long-term Safety of Rusfertide (PTG-300) in Subjects With Polycythemia Vera
NCT03802201	PHASE2	COMPLETED	Study of PTG-300 in Non-Transfusion Dependent and Transfusion-Dependent Beta-Thalassemia Subjects With Chronic Anemia
NCT04054921	PHASE2	COMPLETED	Safety Study for Beta Thalassemia Subjects on PTG-300
NCT04057040	PHASE2	COMPLETED	Hepcidin Mimetic in Patients With Polycythemia Vera (REVIVE)
NCT04202965	PHASE2	COMPLETED	PTG-300 in Subjects With Hereditary Hemochromatosis
NCT04767802	PHASE2	COMPLETED	PTG-300 in Patients With Polycythemia Vera and Elevated Hematocrit
NCT04516382	PHASE1	COMPLETED	Pharmacokinetics and Pharmacodynamics of Different PTG-300 Regimens in Healthy Volunteers

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).