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Rusfertide

Phase 3

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

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindCONTROLLEDDMCBiomarker
Total Trials2
Total Enrollment339
FDA Designations
No designations recorded
Clinical Trials (2)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06033586Study to Evaluate the Long-term Safety of Rusfertide (PTG-300) in Subjects With Polycythemia VeraPHASE3 ACTIVE NOT_RECRUITING 46Jan 22, 2024Apr 1, 2027Aug 6, 20251 United States
NCT05210790A Phase 3 Study of Rusfertide in Patients With Polycythemia VeraPHASE3 ACTIVE NOT_RECRUITING 293Apr 1, 2022Jun 1, 2027Aug 7, 2025184 United States, Australia +18
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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 — 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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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT05210790primaryCompletionDate: changed
LOWMay 26, 2026NCT06033586primaryCompletionDate: changed
LOWMay 24, 2026NCT05210790studyFirstPostDate: changed
LOWMay 24, 2026NCT06033586studyFirstPostDate: changed