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Rusfertide

Phase 3

Polycythemia Vera | Small molecule | Hematology |Protagonist Therapeutics, Inc.|Last Updated: Aug 7, 2025

Target and mechanism

Target class-Tide (Peptide)
ModalitySmall molecule

Also known as Open-label rusfertide

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindCONTROLLEDDMC
Total Trials2
Total Enrollment339

FDA Designations

No designations recorded

Clinical trial landscape

Rusfertide · 2 trials · 1 indication

Phase 3 2
NCT06033586Study to Evaluate the Long-term Safety of Rusfertide (PTG-300) in Subjects With Polycythemia VeraPolycythemia Vera
ACTIVE NOT_RECRUITING46 Analytics
NCT05210790A Phase 3 Study of Rusfertide in Patients With Polycythemia VeraPolycythemia Vera
ACTIVE NOT_RECRUITING293 Analytics
PHASE3ACTIVE NOT_RECRUITING
Study to Evaluate the Long-term Safety of Rusfertide (PTG-300) in Subjects With Polycythemia Vera
Polycythemia VeraUnlock trial analytics
PHASE3ACTIVE NOT_RECRUITING
A Phase 3 Study of Rusfertide in Patients With Polycythemia Vera
Polycythemia VeraUnlock trial analytics

Study Endpoints

Primary Endpoints

Hematocrit
0-2 years

Median hematocrit

Phlebotomies
0-2 years

Number of phlebotomies

Proportion of subjects achieving a response who receive rusfertide compared to placebo.
Week 20 through Week 32

Response is defined as absence of phlebotomy eligibility.

Secondary Endpoints

Comparison of mean number of phlebotomies between rusfertide and placebo.
Week 0 to Week 32
Proportion of subjects with HCT values <45% for rusfertide and placebo.
Week 0 to Week 32
Comparison mean change from baseline in total fatigue score based on PROMIS Short Form between rusfertide and placebo.
Week 32
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Open-label rusfertideEXPERIMENTALOpen-label rusfertide
RusfertideEXPERIMENTALRusfertide (32 Weeks) - Rusfertide (124 Weeks Open-label)
PlaceboEXPERIMENTALPlacebo (32 Weeks) - Rusfertide (124 Weeks Open-label)

Interventions

NameTypeDescription
Open-label rusfertideDRUGsubcutaneous rusfertide
PlaceboDRUGPlacebo
RusfertideDRUGExperimental drug
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: * Subject who has completed at least 12 months of dosing with rusfertide and successfully completed the end of treatment visit of a previous Phase 2 study of rusfertide. * Subject understands the study procedures, is willing and able to adhere to study requirements and agrees to...

Countries:United StatesAustraliaAustriaBelgiumCanadaChileCzechiaFranceGermanyHong KongHungaryIsraelItalyMexicoNetherlandsPolandPortugalSpainTurkey (Türkiye)United Kingdom
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Competitive Landscape -Polycythemia Vera 11 trials

Frequently asked questions about Rusfertide

What is Rusfertide used for in Polycythemia Vera?

Rusfertide is an investigational small molecule being developed for the treatment of Polycythemia Vera, a hematologic condition. It is currently in Phase 3 clinical development and has not been approved by regulatory authorities. The drug is being studied in patients with this condition to evaluate its safety and efficacy.

What does Rusfertide target?

Rusfertide is a peptide-based small molecule, classified under the -tide (peptide) target class. It is being studied for its effects in Polycythemia Vera, a hematologic disorder. The specific molecular target of Rusfertide is not disclosed in the available clinical trial information.

Who makes Rusfertide?

Rusfertide is being developed by Protagonist Therapeutics, Inc., a biopharmaceutical company traded on the stock exchange under the ticker symbol PTGX. The company is conducting Phase 3 clinical trials to evaluate the drug's safety and efficacy in patients with Polycythemia Vera.

What phase is Rusfertide in?

Rusfertide is currently in Phase 3 clinical development. It is an investigational drug and has not been approved by the FDA or other regulatory agencies. The drug is being studied in active clinical trials for the treatment of Polycythemia Vera, with two ongoing Phase 3 studies.

What clinical trials is Rusfertide in?

Rusfertide is being evaluated in two active Phase 3 clinical trials. The first, NCT05210790, is a study in patients with Polycythemia Vera with an enrollment of 293 participants across multiple countries. The second, NCT06033586, is a long-term safety study with 46 participants in the United States.

Is Rusfertide the same as open-label rusfertide?

Rusfertide is also known as open-label rusfertide, referring to the drug when administered in an open-label study design. In the clinical trials, rusfertide is being studied in a randomized, double-blind, controlled setting, and the open-label term may refer to a specific phase of the study where blinding is not applied.