Synthetic analog · 17 amino acids · 2,442 Da

Rusfertide studies and research data

PepBank has 8 rusfertide studies with results. All of them gave rusfertide, with 78 results, grouped by what the studies measured. In each group, results from studies in people come first. Data as of 2026-10-30.

Searches 78 results and 17 papers. Newer studies from Europe PMC

Results in people

100% of 78 results

Results in animals

0% of 78 results

Results in lab cells

0% of 78 results

From computer models

0% of 78 results

The molecule

Rusfertide identity

Identifiers, parent molecule, targets and development stage, from curated data.

Rusfertide is a synthetic cyclic hepcidin mimetic that blocks ferroportin and reduces iron availability for red blood cell production. Mimrylo received US approval for erythrocytosis in adults with polycythemia vera on August 28, 2026.

Kind: Synthetic analog · 17 residues
Identifiers: ChEMBLCHEMBL4650507 (opens in a new tab)PubChem CID155884410 (opens in a new tab)UNIIXM71MYX0IQ (opens in a new tab)DrugBankDB17724 (opens in a new tab)CAS1628323-80-7KEGGD12064 (opens in a new tab)
Development: Approved in the US (Mimrylo, 2026)Protagonist Therapeutics / TakedaProtagonist sponsors the earlier trials. Takeda sponsors current Japanese and Chinese phase 2 trials. Mimrylo has US FDA approval. Eight distinct product-intervention registrations report actual enrollment. Two additional registrations report estimated enrollment and do not establish treated participants. Source (opens in a new tab)

Targets

Molecular targets with action and evidence source
Target	Action	Evidence
FerroportinSLC40A1Q9NP59 (opens in a new tab)	Inhibitor	FDA prescribing information, section 12.1, and UniProt Q9NP59.Source (opens in a new tab)

Sequence and structure

Residues, differences from the parent, calculated properties and 3D structure. Source values and calculated values are shown separately.

Sequence

17 amino acids · 4 modifications · 1 disulfide bond

THFPCIFE

PRSKGCK

Properties

From identity sources

Formula: C₁₁₄H₁₈₁N₂₇O₂₈S₂
Molecular weight: 2,442 g/mol
Monoisotopic mass: 2,440.3011 Da

PubChem CID 155884410(opens in a new tab)retrieved 2026-10-27
Curated MIMRYLO section 11(opens in a new tab)retrieved 2026-10-27
GSRS UNII XM71MYX0IQ(opens in a new tab)retrieved 2026-10-27
Curated EU/3/20/2330(opens in a new tab)retrieved 2026-10-27

Calculated from the sequence

pKa set

Some values cannot be calculated. A modification changes this value and is not modeled here (for example a lipid, metal or linker).

Average mass: Not calculated
Monoisotopic mass: Not calculated
Isoelectric point (pI): Not calculated
Net charge at pH 7.4: Not calculated
GRAVY (hydropathy): −0.965; Mean Kyte-Doolittle hydropathy · Kyte and Doolittle 1982; Negative: hydrophilic on average.
Extinction coefficient, 280 nm (Cys reduced): 0 M⁻¹ cm⁻¹; 5500 x Trp + 1490 x Tyr · Pace et al. 1995 (ExPASy ProtParam); No Trp or Tyr: absorbance at 280 nm cannot measure this peptide.
Extinction coefficient, 280 nm (all Cys as cystines): 125 M⁻¹ cm⁻¹; 5500 x Trp + 1490 x Tyr + 125 x cystine · Pace et al. 1995 (ExPASy ProtParam)
Instability index: 24.12; Dipeptide instability weights (DIWV) · Guruprasad et al. 1990; 40 or below: classed as stable (ExPASy).
Aliphatic index: 22.9; Mole % A + 2.9 V + 3.9 (I + L) · Ikai 1980
Boman index: 2.55 kcal/mol; Mean solubility value (negated) · Boman 2003; Above 2.48: high protein-binding potential (Boman).
Hydrophobic moment (μH): 0.264; Eisenberg scale, 100° per residue, best 11-residue window · Eisenberg et al. 1982, 1984

Not included: other modification, lipid chain. Mass, charge and pI are not calculated; use the identity-source values.
The N-terminal group is not modeled.
Defined and validated for proteins; use with care for short peptides.
Assumes an ideal alpha helix. Many short peptides are not helical.

@pepdb/chem 0.2.0 · EMBOSS pKa set · ExPASy residue masses

Net charge by pH

No charge curve

A modification changes this value and is not modeled here (for example a lipid, metal or linker).

Composition

Hydrophobic6% (1)I
Aromatic12% (2)F
Positive29% (5)H K R
Negative12% (2)D E
Polar12% (2)S T
Special29% (5)C G P

17 residues. Each residue counts in one class; F and W count as aromatic.

A Ala0 · 0%

I Ile1 · 6%

L Leu0 · 0%

M Met0 · 0%

V Val0 · 0%

F Phe2 · 12%

W Trp0 · 0%

Y Tyr0 · 0%

H His1 · 6%

K Lys3 · 18%

R Arg1 · 6%

D Asp1 · 6%

E Glu1 · 6%

N Asn0 · 0%

Q Gln0 · 0%

S Ser1 · 6%

T Thr1 · 6%

C Cys2 · 12%

G Gly1 · 6%

P Pro2 · 12%

3D structure

No 3D structure

No experimental structure (PDB) and no UniProt entry for an AlphaFold model are listed for this peptide. Synthetic peptides often have neither. The sequence view above is the reference.

2D structure

Rusfertide has 17 residues. A full-atom drawing is hard to read at this size; the sequence view is the reference.

SMILES fromPubChem CID 155884410(opens in a new tab)

CCCCCCCCCCCCCCCC(=O)N[C@@H](CCC(=O)NCCCC[C@H]1C(=O)N[C@H](C(=O)N[C@H](C(=O)N2CCC[C@H]2C(=O)N[C@H](C(=O)N[C@H](C(=O)N[C@H](C(=O)NCC(=O)N[C@@H](CSSC[C@@H](C(=O)N[C@H](C(=O)N1)[C@@H](C)CC)NC(=O)[C@@H]3CCCN3C(=O)[C@H](CC4=CC=CC=C4)NC(=O)[C@H](CC5=CNC=N5)NC(=O)[C@H]([C@@H](C)O)NC(=O)[C@H](CC(=O)O)NC(=O)CC(C)C)C(=O)N[C@@H](CCCCN)C(=O)N)CCCCN)CO)CCCNC(=N)N)CCC(=O)O)CC6=CC=CC=C6)C(=O)O

Helical wheel

An ideal alpha-helix projection. It shows where the hydrophobic residues fall if the peptide folds as a helix. It does not show that it does.

μH, residues shown: 0.060
μH, best 11-residue window: 0.264
Arrow direction: 247°
Residues: 17

HydrophilicHydrophobic

Eisenberg consensus (normalized) scale · + K, R · − D, E

Assumes an ideal α-helix: 100° per residue, 3.6 residues per turn. The arrow is the mean hydrophobic moment (Eisenberg et al. 1984) and points at the hydrophobic face.

Show as table

Position	Residue	Angle	Eisenberg consensus (normalized)
1	D	0°	-0.90
2	T	100°	-0.05
3	H	200°	-0.40
4	F	300°	1.19
5	P	40°	0.12
6	C	140°	0.29
7	I	240°	1.38
8	K	340°	-1.50
9	F	80°	1.19
10	E	180°	-0.74
11	P	280°	0.12
12	R	20°	-2.53
13	S	120°	-0.18
14	K	220°	-1.50
15	G	320°	0.48
16	C	60°	0.29
17	K	160°	-1.50

Results by area

Rusfertide results by year and outcome

Counts are exact and come from studies that gave the peptide.

Studies by year

8 of 17 studies have results (47%), published 2023 to 2026. Top row: every study. Bottom row: studies with results about Rusfertide, by class. The rows use different scales.

Studies
Human
Animal
Lab
Computer model
Natural levels, tool or combination only

Studies17 in all, 2006 to 2026

Studies with results8 in all, 2023 to 2026

Retracted studies are left out. A study with results in two classes counts in both.

AI wrote and checked these results. AI can make mistakes, so check important results against the source.

What the studies measured

People first in each area, then animals, then lab cells. Counts are exact and come from studies that gave the peptide.

Rusfertide studies on blood and clotting

28 results · people 28 · animals 0 · lab 0

Top 5 of 28 results

In healthy subjects, repeated rusfertide doses reduced serum iron.Clinical pharmacology in drug development, 2025
In patients with polycythemia vera, 77% with rusfertide versus 33% with placebo achieved clinical response.Journal of clinical oncology, 2026
In polycythemia vera, 60% responded with rusfertide versus 17% with placebo during randomized withdrawal.The New England journal of medicine, 2024
In patients with polycythemia vera, iron deficiency markers such as ferritin and red blood cell size improved compared with baseline during rusfertide treatment.Leukemia research, 2025
In healthy adult men, single rusfertide doses lowered blood iron in a dose-dependent manner.European journal of haematology, 2024

Rusfertide studies on blood levels and clearance

23 results · people 23 · animals 0 · lab 0

Top 3 of 23 results

In healthy adults, rusfertide reached its median blood concentration peak at 4 hours.Clinical therapeutics, 2025
In healthy subjects, first-dose peak rusfertide levels differed between abdomen, thigh, and arm injections.Clinical pharmacology in drug development, 2025
In healthy adult men, rusfertide peak blood concentration increased less than proportionally with dose.European journal of haematology, 2024

Rusfertide studies on side effects

16 results · people 16 · animals 0 · lab 0

Top 5 of 16 results

In healthy subjects receiving rusfertide, no serious side effects occurred.Clinical pharmacology in drug development, 2025
In healthy subjects receiving rusfertide, injection-site reactions such as redness, hardening and itching were among side effects reported in 2 or more subjects.Clinical pharmacology in drug development, 2025
In healthy subjects receiving rusfertide, heart electrical tests showed no clinically relevant findings.Clinical pharmacology in drug development, 2025
In healthy subjects receiving rusfertide, 2 or more subjects reported headache.Clinical pharmacology in drug development, 2025
In healthy adults, most adverse events during the rusfertide study were mild.Drugs in R&D, 2024

Rusfertide studies on heart and blood vessels

6 results · people 6 · animals 0 · lab 0

Top 3 of 6 results

In healthy adults, rusfertide heart repolarization changes closely followed placebo.Clinical therapeutics, 2025
In healthy adults, rusfertide had no clinically relevant effect on heart rate.Clinical therapeutics, 2025
In healthy adults, rusfertide had no clinically relevant effect on the PR heart conduction interval.Clinical therapeutics, 2025

Rusfertide studies on overall treatment results

2 results · people 2 · animals 0 · lab 0

2 results

In patients with polycythemia vera, rusfertide improved the reported fatigue score versus placebo.Journal of clinical oncology, 2026
In patients with polycythemia vera, rusfertide improved the reported symptom score versus placebo.Journal of clinical oncology, 2026

Rusfertide studies on daily life and fitness

1 result · people 1 · animals 0 · lab 0

1 result

In polycythemia vera patients with moderate or severe initial symptoms, rusfertide was linked to lower symptom scores compared with baseline.The New England journal of medicine, 2024

Rusfertide studies on liver

1 result · people 1 · animals 0 · lab 0

1 result

In HFE-related haemochromatosis, liver iron changed from 1·4 to 1·1 mg per g with rusfertide, without a statistically significant change.The lancet. Gastroenterology & hepatology, 2023

Rusfertide studies on survival and serious illness

1 result · people 1 · animals 0 · lab 0

1 result

In the polycythemia vera trial, no deaths were reported, including with rusfertide.Journal of clinical oncology, 2026

Trials and papers

Rusfertide trials

Registered trials that test the peptide, with phase, status and dates. A dot marks trials with posted results.

StatusPhase

10 trials match. Highest phase first. Data from ClinicalTrials.gov.

Trials on this page (10 of 10)

10 trials · 3 ongoing · 1 not yet recruiting · 6 completed · 2 with posted results

Completed
Ongoing
Not yet recruiting
Stopped
Estimated dates (est.)
Results posted

202020252030

Ph 2
TRANSCEND
NCT03802201
19 Dec 2018 to 31 Jul 2020
Ph 2
TRANSCEND
NCT04054921
10 May 2019 to 31 Jul 2020
Ph 2
Hepcidin Mimetic in Patients With Polycythemia Vera (REVIVE)
NCT04057040
1 Oct 2019 to 17 Jun 2024
Ph 2
PTG-300 in Subjects With Hereditary Hemochromatosis
NCT04202965
19 Mar 2020 to 15 Feb 2022
Ph 1
Pharmacokinetics and Pharmacodynamics of Different PTG-300 Regimens in Healthy Volunteers
NCT04516382
14 Sept 2020 to 30 Jun 2021
Ph 2
PACIFIC
NCT04767802
2 Feb 2021 to 15 Mar 2023
Ph 3
VERIFY
NCT05210790
11 Apr 2022 to est. 31 Aug 2028
Ph 3
THRIVE
NCT06033586
22 Jan 2024 to est. 1 Apr 2027
Ph 2
A Study of Rusfertide in Japanese Adults With Polycythemia Vera
NCT07648030
14 Jul 2026 to est. 17 May 2030
Ph 2
A Study of Rusfertide in Adults With Polycythemia Vera in China
NCT07765602
est. 1 Mar 2027 to est. 1 Aug 2028

10 trials · 3 ongoing · 1 not yet recruiting · 6 completed · 2 with posted results · source ClinicalTrials.gov (opens in a new tab)

Active, not recruitingPhase 3Results postedNCT05210790

A Study of Rusfertide in Adults With Polycythemia Vera (PV)

Protagonist Therapeutics, Inc.n = 29311 Apr 2022 to est. 31 Aug 2028

Polycythemia Vera

Trial page ClinicalTrials.govUpdated 19 Oct 2026

Active, not recruitingPhase 3NCT06033586

Study to Evaluate the Long-term Safety of Rusfertide (PTG-300) in Subjects With Polycythemia Vera

Protagonist Therapeutics, Inc.n = 4622 Jan 2024 to est. 1 Apr 2027

Polycythemia Vera

Trial page ClinicalTrials.govUpdated 6 Aug 2025

RecruitingPhase 2NCT07648030

A Study of Rusfertide in Japanese Adults With Polycythemia Vera

Takedan = 914 Jul 2026 to est. 17 May 2030

Polycythemia Vera

Trial page ClinicalTrials.govUpdated 2 Oct 2026

Not yet recruitingPhase 2NCT07765602

A Study of Rusfertide in Adults With Polycythemia Vera in China

Takedan = 24est. 1 Mar 2027 to est. 1 Aug 2028

Polycythemia Vera

Trial page ClinicalTrials.govUpdated 28 Aug 2026

CompletedPhase 2Results postedNCT04202965

PTG-300 in Subjects With Hereditary Hemochromatosis

Protagonist Therapeutics, Inc.n = 1619 Mar 2020 to 15 Feb 2022

Hereditary Hemochromatosis

Trial page ClinicalTrials.govUpdated 20 Aug 2025

CompletedPhase 2NCT04057040

Hepcidin Mimetic in Patients With Polycythemia Vera (REVIVE)

Protagonist Therapeutics, Inc.n = 701 Oct 2019 to 17 Jun 2024

Polycythemia Vera

Trial page ClinicalTrials.govUpdated 7 Aug 2025

CompletedPhase 2NCT04767802

PTG-300 in Patients With Polycythemia Vera and Elevated Hematocrit

Protagonist Therapeutics, Inc.n = 202 Feb 2021 to 15 Mar 2023

Polycythemia Vera

Trial page ClinicalTrials.govUpdated 18 Mar 2024

CompletedPhase 2NCT03802201

Study of PTG-300 in Non-Transfusion Dependent and Transfusion-Dependent Beta-Thalassemia Subjects With Chronic Anemia

Protagonist Therapeutics, Inc.n = 6319 Dec 2018 to 31 Jul 2020

β-thalassemia · Ineffective Erythropoiesis · Chronic Anemia

Trial page ClinicalTrials.govUpdated 16 Jul 2021

CompletedPhase 2NCT04054921

Safety Study for Beta Thalassemia Subjects on PTG-300

Protagonist Therapeutics, Inc.n = 3410 May 2019 to 31 Jul 2020

β-thalassemia · Ineffective Erythropoiesis

Trial page ClinicalTrials.govUpdated 16 Jul 2021

CompletedPhase 1NCT04516382

Pharmacokinetics and Pharmacodynamics of Different PTG-300 Regimens in Healthy Volunteers

Protagonist Therapeutics, Inc.n = 1214 Sept 2020 to 30 Jun 2021

Healthy Volunteers

Trial page ClinicalTrials.govUpdated 16 Sept 2021

Papers

Studies on this peptide. Some have results. The others have none yet.

Sort

Design

17 studies match. Retracted studies are left out.

Showing 17 of 17 studiesquery qry_5776f799ae4fe244abf90966

Studies with results

8 studies

Randomized controlled trial · 2024

4 results

Rusfertide, a Hepcidin Mimetic, for Control of Erythrocytosis in Polycythemia Vera.

Kremyanskaya et al. 2024The New England journal of medicinen = 70cited by 46

PMID 38381675DOI 10.1056/nejmoa2308809PUB-38381675

Full text at the publisher4 results where Rusfertide was given

Publisher page See results (4)

Non-randomized trial · 2023

10 results

Rusfertide for the treatment of iron overload in HFE-related haemochromatosis: an open-label, multicentre, proof-of-concept phase 2 trial.

Kowdley et al. 2023The lancet. Gastroenterology & hepatologyn = 16cited by 26

PMID 37863080DOI 10.1016/s2468-1253(23)00250-9PUB-37863080

Full text at the publisher10 results where Rusfertide was given

Publisher page See results (10)

Randomized controlled trial · 2024

8 results

Pharmacokinetics, pharmacodynamics, and tolerability of an aqueous formulation of rusfertide (PTG-300), a hepcidin mimetic, in healthy volunteers: A double-blind first-in-human study.

Modi et al. 2024European journal of haematologycited by 8

PMID 38785334DOI 10.1111/ejh.14243PUB-38785334

Full text at the publisher8 results where Rusfertide was given

Publisher page See results (8)

Randomized controlled trial · 2024

14 results

Pharmacokinetics and Pharmacodynamics of Rusfertide, a Hepcidin Mimetic, Following Subcutaneous Administration of a Lyophilized Powder Formulation in Healthy Volunteers.

Modi et al. 2024Drugs in R&Dcited by 5

PMID 39546273PMC11652423DOI 10.1007/s40268-024-00497-zPUB-39546273

Free to read · publisher termsCC BY-NC14 results where Rusfertide was given

Read at source See results (14)

Randomized controlled trial · 2025

19 results

Multiple-Dose Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of Subcutaneous Rusfertide, a Hepcidin Mimetic, in Healthy Subjects.

Modi et al. 2025Clinical pharmacology in drug developmentn = 18cited by 2

PMID 39888264PMC11975206DOI 10.1002/cpdd.1514PUB-39888264

Free to read · publisher termsCC BY-NC-ND19 results where Rusfertide was given

Read at source See results (19)

Non-randomized trial · 2025

9 results

Rusfertide rapidly decreases hematocrit in patients with suboptimally controlled polycythemia vera.

Chew et al. 2025Leukemia researchn = 20cited by 2

PMID 41175501DOI 10.1016/j.leukres.2025.108132PUB-41175501

Full text at the publisher9 results where Rusfertide was given

Publisher page See results (9)

Randomized controlled trial · 2025

8 results

Evaluation of Rusfertide, a Hepcidin Mimetic, on Cardiac Repolarization: A Randomized, Placebo- and Positive-Controlled Crossover Thorough QT Study in Healthy Participants.

Modi et al. 2025Clinical therapeuticsn = 60cited by 1

PMID 41033871DOI 10.1016/j.clinthera.2025.09.002PUB-41033871

Full text at the publisher8 results where Rusfertide was given

Publisher page See results (8)

Randomized controlled trial · 2026

6 results

VERIFY: A Phase III Trial of Rusfertide Versus Placebo in Phlebotomy-Dependent Patients With Polycythemia Vera Receiving Standard-of-Care Therapy.

Kuykendall et al. 2026Journal of clinical oncology : official journal of the American Society of Clinical Oncologyn = 293

PMID 42868322DOI 10.1200/jco-26-00992PUB-42868322

Full text at the publisher6 results where Rusfertide was given

Publisher page See results (6)

More studies

9 studies

PubMed tag: Review · 2023

Hepcidin mimetics in polycythemia vera: resolving the irony of iron deficiency and erythrocytosis.

Handa et al. 2023Current opinion in hematologycited by 22

PMID 36728649PMC9908837DOI 10.1097/moh.0000000000000747PUB-36728649

Rights unknown

Source link

PubMed tag: Review, Case Reports · 2021

β-Thalassemia: evolving treatment options beyond transfusion and iron chelation.

Langer et al. 2021Hematology. American Society of Hematology. Education Programcited by 20

PMID 34889443PMC8791140DOI 10.1182/hematology.2021000313PUB-34889443

Rights unknown

Source link

PubMed tag: Review · 2026

Modulators of the hepcidin pathway in polycythemia vera and myelofibrosis.

Kremyanskaya et al. 2026Bloodcited by 4

PMID 41100735DOI 10.1182/blood.2025028643PUB-41100735

Full text at the publisher

Publisher page

PubMed tag: Review · 2025

Iron deficiency and phlebotomy in patients with polycythemia vera.

Edahiro et al. 2025International journal of hematologycited by 3

PMID 39528901DOI 10.1007/s12185-024-03868-zPUB-39528901

Full text at the publisher

Publisher page

PubMed tag: Letter · 2026

Emerging agents and regimens for myeloproliferative neoplasms: updates from ASH 2025 Annual Meeting.

Zhang et al. 2026Experimental hematology & oncology

PMID 42547880PMC13430699DOI 10.1186/s40164-026-00813-0PUB-42547880

Free to read · publisher termsCC BY-NC-ND

Read at source

PubMed tag: Review · 2026

Targeting the hepcidin-ferroportin axis in polycythemia vera: a complementary approach to clone-directed therapies.

Costa et al. 2026Blood reviews

PMID 42660705DOI 10.1016/j.blre.2026.101420PUB-42660705

Full text at the publisher

Publisher page

PubMed tag: Review · 2026

Ferritin Normalization Is Not Disease Control in HFE-Related Hemochromatosis: Residual Risk, Transferrin Saturation, and the Hepcidin-Ferroportin Axis.

Raja et al. 2026European journal of haematology

PMID 42764193DOI 10.1111/ejh.70327PUB-42764193

Full text at the publisher

Publisher page

PubMed tag: Review · 2006

Rusfertide

National Institute of Child Health and Human Development, Bethesda (MD)

PMID 42766707PUB-42766707

Rights unknown

Source link

PubMed tag: Letter · 2026

Rusfertide for Polycythemia Vera: Incremental Benefit Assessment.

Tefferi et al. 2026American journal of hematology

PMID 42891571DOI 10.1002/ajh.70528PUB-42891571

Full text at the publisher

Publisher page

Newer studies

PepBank reads each paper before it shows the results. This search looks in Europe PMC for newer papers that PepBank has not read yet.

Rusfertide studies: 8 papers with results

Page 1 of 1

VERIFY: A Phase III Trial of Rusfertide Versus Placebo in Phlebotomy-Dependent Patients With Polycythemia Vera Receiving Standard-of-Care Therapy.
Kuykendall AT · Journal of clinical oncology : official journal of the American Society of Clinical Oncology · 2026
Randomized controlled trial · 6 results
Rusfertide, a Hepcidin Mimetic, for Control of Erythrocytosis in Polycythemia Vera.
Kremyanskaya M · The New England journal of medicine · 2024
Randomized controlled trial · 4 results
Evaluation of Rusfertide, a Hepcidin Mimetic, on Cardiac Repolarization: A Randomized, Placebo- and Positive-Controlled Crossover Thorough QT Study in Healthy Participants.
Modi NB · Clinical therapeutics · 2025
Randomized controlled trial · 8 results
Multiple-Dose Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of Subcutaneous Rusfertide, a Hepcidin Mimetic, in Healthy Subjects.
Modi NB · Clinical pharmacology in drug development · 2025
Randomized controlled trial · 19 results
Pharmacokinetics, pharmacodynamics, and tolerability of an aqueous formulation of rusfertide (PTG-300), a hepcidin mimetic, in healthy volunteers: A double-blind first-in-human study.
Modi NB · European journal of haematology · 2024
Randomized controlled trial · 8 results
Pharmacokinetics and Pharmacodynamics of Rusfertide, a Hepcidin Mimetic, Following Subcutaneous Administration of a Lyophilized Powder Formulation in Healthy Volunteers.
Modi NB · Drugs in R&D · 2024
Randomized controlled trial · 14 results
Rusfertide rapidly decreases hematocrit in patients with suboptimally controlled polycythemia vera.
Chew LP · Leukemia research · 2025
Non-randomized trial · 9 results
Rusfertide for the treatment of iron overload in HFE-related haemochromatosis: an open-label, multicentre, proof-of-concept phase 2 trial.
Kowdley KV · The lancet. Gastroenterology & hepatology · 2023
Non-randomized trial · 10 results

Status and safety

Regulatory and safety

Approval and sport status, and the safety outcomes reported in study results. Laws and labels change, so check the regulator for the current status.

Regulatory status by jurisdiction, with date and source
Jurisdiction	Body	Status	As of	Source
US	FDA	Mimrylo · 28 Aug 2026	27 Oct 2026	dailymed.nlm.nih.gov (opens in a new tab)
EU	EMA	EMA records orphan designation EU/3/20/2330 for PTG-300. Orphan designation is not marketing authorisation. Current EU marketing authorisation was not established.	27 Oct 2026	ema.europa.eu (opens in a new tab)
Sport	WADA	Status unknown The complete 2026 List does not name rusfertide, PTG-300, Mimrylo or hepcidin.	2026 List	wada-ama.org (opens in a new tab)
EU	Note	Orphan designation EU/3/20/2330, granted October 19, 2020. This record does not establish marketing authorisation.	27 Oct 2026	ema.europa.eu (opens in a new tab)
UK	Note	MHRA UK API 19819 lists distribution of rusfertide as a starting material. This is not a product marketing authorisation.	27 Oct 2026	cms.mhra.gov.uk (opens in a new tab)

Safety outcomes

21 safety results for Rusfertide.

Human evidence· 21

In the polycythemia vera trial, no deaths were reported, including with rusfertide.
Randomized controlled trialn = 293
In healthy adults, rusfertide placebo-corrected QTcF changes ranged from -2.0 to 1.8 ms and closely followed placebo.
Randomized controlled trialn = 60
Placebo-corrected ΔQTcF (ΔΔQTcF) LS mean
−2 to 1.8 ms
rusfertide 90 mg versus placebo
In healthy adults, rusfertide had no clinically relevant effect on heart rate.
Randomized controlled trialn = 60
In healthy adults, rusfertide had no clinically relevant effect on the PR interval.
Randomized controlled trialn = 60
In healthy adults, rusfertide had no clinically relevant effect on the QRS interval.
Randomized controlled trialn = 60
In healthy adults, predicted placebo-corrected QTcF change at geometric mean peak rusfertide concentration was 0.04 ms.
Randomized controlled trialn = 60
Predicted ΔΔQTcF at geometric mean Cmax
0.04 ms
rusfertide
Predicted ΔΔQTcF 90% CI
−1.21 to 1.29 ms
rusfertide
In healthy subjects receiving rusfertide, no serious adverse events occurred.
Randomized controlled trialn = 18
In healthy subjects receiving rusfertide, injection-site reactions (erythema, induration, pruritus) were among treatment-emergent adverse events reported in 2 or more subjects.
Randomized controlled trialn = 18

Doses studied · 10

The doses that these studies used. They are not recommendations.

90 mgHuman
Species
human
Route
subcutaneous
Duration
Not reported
Design
Randomized controlled trial · n = 60
60 mg once weeklyHuman
Species
human
Route
subcutaneous
Duration
5 weeks
Design
Randomized controlled trial · n = 18
40 and 80 mgHuman
Species
human
Route
subcutaneous
Duration
Not reported
Design
Randomized controlled trial
1, 3, 10, or 20 mgHuman
Species
human
Route
subcutaneous
Duration
Not reported
Design
Randomized controlled trial
1-80 mg single doseHuman
Species
human
Route
subcutaneous
Duration
Not reported
Design
Randomized controlled trial
10-30 mg lyophilized formulationHuman
Species
human
Route
subcutaneous
Duration
Not reported
Design
Randomized controlled trial

Doses studied in the study results, as given in the studies.
Dose as reported	Class	Species	Route	Duration	Design
90 mg	Human	human	subcutaneous	Not reported	Randomized controlled trial · n = 60
60 mg once weekly	Human	human	subcutaneous	5 weeks	Randomized controlled trial · n = 18
40 and 80 mg	Human	human	subcutaneous	Not reported	Randomized controlled trial
1, 3, 10, or 20 mg	Human	human	subcutaneous	Not reported	Randomized controlled trial
1-80 mg single dose	Human	human	subcutaneous	Not reported	Randomized controlled trial
10-30 mg lyophilized formulation	Human	human	subcutaneous	Not reported	Randomized controlled trial

6 of 10 shown

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Data

Data and API

Data version, change history and code for this peptide.

Data version 30856030 Oct 2026

Peptide

Peptide id: PEP-000166
Peptide version: 2
Peptide hash: d5b30a59498b62b4…
Data version: 308560

Latest changes

27 Oct 2026UpdatedData version 287309
27 Oct 2026CreatedData version 256768

For developers

curl -s https://pepbank.io/v1/briefs/rusfertide

Evidence summary for Rusfertide: counts by class, key results with sources, notices, data version.

Peptide as JSON Evidence summary as JSON

Outcome group	Human	Animal	Lab	Computer model	Total
PharmacokineticsPharmacology	Human: 23 results from 4 studies: 9 increase, 4 no significant change, 1 mixed, 9 not reported. Strongest design: Randomized controlled trial	No results	No results	No results	23
Adverse eventsWhole body	Human: 16 results from 4 studies: 4 no significant change, 12 not reported. Strongest design: Randomized controlled trial	No results	No results	No results	16
Blood counts and hematopoiesisWhole body	Human: 15 results from 4 studies: 3 increase, 7 decrease, 1 no significant change, 4 not reported. Strongest design: Randomized controlled trial	No results	No results	No results	15
Blood biochemical markersWhole body	Human: 13 results from 5 studies: 7 decrease, 3 mixed, 3 not reported. Strongest design: Randomized controlled trial	No results	No results	No results	13
Heart functionCardiovascular	Human: 6 results from 1 study: 5 no significant change, 1 not reported. Strongest design: Randomized controlled trial	No results	No results	No results	6
Overall treatment effectWhole body	Human: 2 results from 1 study: 2 not reported. Strongest design: Randomized controlled trial	No results	No results	No results	2
Liver fat and liver functionLiver	Human: 1 result from 1 study: 1 no significant change. Strongest design: Non-randomized trial	No results	No results	No results	1
MortalityWhole body	Human: 1 result from 1 study: 1 not reported. Strongest design: Randomized controlled trial	No results	No results	No results	1
Patient-reported outcomesWhole body	Human: 1 result from 1 study: 1 decrease. Strongest design: Non-randomized trial	No results	No results	No results	1

Outcome group	Human	Animal	Lab	Computer model	Total
PharmacokineticsPharmacology	Human: 23 results from 4 studies: 9 increase, 4 no significant change, 1 mixed, 9 not reported. Strongest design: Randomized controlled trial	No results	No results	No results	23
Adverse eventsWhole body	Human: 16 results from 4 studies: 4 no significant change, 12 not reported. Strongest design: Randomized controlled trial	No results	No results	No results	16
Blood counts and hematopoiesisWhole body	Human: 15 results from 4 studies: 3 increase, 7 decrease, 1 no significant change, 4 not reported. Strongest design: Randomized controlled trial	No results	No results	No results	15
Blood biochemical markersWhole body	Human: 13 results from 5 studies: 7 decrease, 3 mixed, 3 not reported. Strongest design: Randomized controlled trial	No results	No results	No results	13
Heart functionCardiovascular	Human: 6 results from 1 study: 5 no significant change, 1 not reported. Strongest design: Randomized controlled trial	No results	No results	No results	6
Overall treatment effectWhole body	Human: 2 results from 1 study: 2 not reported. Strongest design: Randomized controlled trial	No results	No results	No results	2
Liver fat and liver functionLiver	Human: 1 result from 1 study: 1 no significant change. Strongest design: Non-randomized trial	No results	No results	No results	1
MortalityWhole body	Human: 1 result from 1 study: 1 not reported. Strongest design: Randomized controlled trial	No results	No results	No results	1
Patient-reported outcomesWhole body	Human: 1 result from 1 study: 1 decrease. Strongest design: Non-randomized trial	No results	No results	No results	1

Rusfertide studies and research data

Rusfertide identity

Targets

Sequence and structure

Sequence

Properties

Net charge by pH

Composition

3D structure

2D structure

Helical wheel

Rusfertide results by year and outcome

What the studies measured

Rusfertide studies on blood and clotting

Rusfertide studies on blood levels and clearance

Rusfertide studies on side effects

Rusfertide studies on heart and blood vessels

Rusfertide studies on overall treatment results

Rusfertide studies on daily life and fitness

Rusfertide studies on liver

Rusfertide studies on survival and serious illness

Rusfertide trials

A Study of Rusfertide in Adults With Polycythemia Vera (PV)

Study to Evaluate the Long-term Safety of Rusfertide (PTG-300) in Subjects With Polycythemia Vera

A Study of Rusfertide in Japanese Adults With Polycythemia Vera

A Study of Rusfertide in Adults With Polycythemia Vera in China

PTG-300 in Subjects With Hereditary Hemochromatosis

Hepcidin Mimetic in Patients With Polycythemia Vera (REVIVE)

PTG-300 in Patients With Polycythemia Vera and Elevated Hematocrit

Study of PTG-300 in Non-Transfusion Dependent and Transfusion-Dependent Beta-Thalassemia Subjects With Chronic Anemia

Safety Study for Beta Thalassemia Subjects on PTG-300

Pharmacokinetics and Pharmacodynamics of Different PTG-300 Regimens in Healthy Volunteers

Papers

Studies with results

Rusfertide, a Hepcidin Mimetic, for Control of Erythrocytosis in Polycythemia Vera.

Rusfertide for the treatment of iron overload in HFE-related haemochromatosis: an open-label, multicentre, proof-of-concept phase 2 trial.

Pharmacokinetics, pharmacodynamics, and tolerability of an aqueous formulation of rusfertide (PTG-300), a hepcidin mimetic, in healthy volunteers: A double-blind first-in-human study.

Pharmacokinetics and Pharmacodynamics of Rusfertide, a Hepcidin Mimetic, Following Subcutaneous Administration of a Lyophilized Powder Formulation in Healthy Volunteers.

Multiple-Dose Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of Subcutaneous Rusfertide, a Hepcidin Mimetic, in Healthy Subjects.

Rusfertide rapidly decreases hematocrit in patients with suboptimally controlled polycythemia vera.

Evaluation of Rusfertide, a Hepcidin Mimetic, on Cardiac Repolarization: A Randomized, Placebo- and Positive-Controlled Crossover Thorough QT Study in Healthy Participants.

VERIFY: A Phase III Trial of Rusfertide Versus Placebo in Phlebotomy-Dependent Patients With Polycythemia Vera Receiving Standard-of-Care Therapy.

More studies

Hepcidin mimetics in polycythemia vera: resolving the irony of iron deficiency and erythrocytosis.

β-Thalassemia: evolving treatment options beyond transfusion and iron chelation.

Modulators of the hepcidin pathway in polycythemia vera and myelofibrosis.

Iron deficiency and phlebotomy in patients with polycythemia vera.

Emerging agents and regimens for myeloproliferative neoplasms: updates from ASH 2025 Annual Meeting.

Targeting the hepcidin-ferroportin axis in polycythemia vera: a complementary approach to clone-directed therapies.

Ferritin Normalization Is Not Disease Control in HFE-Related Hemochromatosis: Residual Risk, Transferrin Saturation, and the Hepcidin-Ferroportin Axis.

Rusfertide

Rusfertide for Polycythemia Vera: Incremental Benefit Assessment.

Newer studies

Rusfertide studies: 8 papers with results

Regulatory and safety

Human evidence· 21

Doses studied · 10

Data and API

Peptide

Latest changes

For developers

Related peptides

Rusfertide results by year and outcome

Rusfertide studies on blood and clotting

Rusfertide studies on blood levels and clearance

Rusfertide studies on side effects

Rusfertide studies on heart and blood vessels

Rusfertide studies on overall treatment results

Rusfertide studies on daily life and fitness

Rusfertide studies on liver

Rusfertide studies on survival and serious illness

Rusfertide trials

A Study of Rusfertide in Adults With Polycythemia Vera (PV)

Study to Evaluate the Long-term Safety of Rusfertide (PTG-300) in Subjects With Polycythemia Vera

A Study of Rusfertide in Japanese Adults With Polycythemia Vera

A Study of Rusfertide in Adults With Polycythemia Vera in China

PTG-300 in Subjects With Hereditary Hemochromatosis

Hepcidin Mimetic in Patients With Polycythemia Vera (REVIVE)

PTG-300 in Patients With Polycythemia Vera and Elevated Hematocrit

Study of PTG-300 in Non-Transfusion Dependent and Transfusion-Dependent Beta-Thalassemia Subjects With Chronic Anemia