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marstacimab

Phase 3

Hemophilia A | Small molecule | Hematology |Pfizer, Inc.|Last Updated: May 7, 2026

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
UNCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment100
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05611801A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia BPHASE3 RECRUITING 100 — — Dec 9, 2022Sep 10, 2028May 7, 202634 Argentina, Australia +16
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Study Endpoints
Primary Endpoints
Annualized bleeding rate (ABR) of treated bleeding events
Baseline to end of 12-month treatment period

Derived for each subject for each period (historical and study treatment) by using the following formula: ABR = number of bleeds requiring treatments/ (days on treatment period / 365.25)

Incidence of adverse events and serious adverse events
Screening through end of follow-up period (approximately 14 months)
Incidence and severity of thrombotic events
Baseline to end of 12-month treatment period
Incidence and severity of thrombotic microangiopathy
Baseline to end of 12-month treatment period
Incidence and severity of disseminated intravascular coagulation/consumption coagulopathy events
Baseline to end of 12-month treatment period
Immunogenicity (incidence of ADA and clinically significant persistent NAb against marstacimab)
Baseline to end of 12-month treatment period
Incidence and severity of injection site reaction
Baseline to end of 12-month treatment period
Incidence of severe hypersensitivity and anaphylactic reactions
Baseline to end of 12-month treatment period
Secondary Endpoints
Incidence of joint bleeds (treated)
Baseline to end of 12-month treatment period
Incidence of spontaneous bleeds (treated)
Baseline to end of 12-month treatment period
Incidence of target joint bleeds (treated)
Baseline to end of 12-month treatment period
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
marstacimab (PF-06741086)EXPERIMENTALWeekly subcutaneous injections.
Interventions
NameTypeDescription
marstacimabDRUGmarstacimab
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Eligibility Criteria
Age Range1 Year — 17 Years
SexMALE
Healthy VolunteersNo
Study Sites34

Inclusion Criteria: * Male participants of appropriate age and required minimum weight * Participants aged 12 to 17 years must be at least 25 kgs at time of consent. * Participants aged 6 to 11 years must be at least 19 kgs at time of consent. * Minimum weight requirement for participants aged 1 to...

Countries:ArgentinaAustraliaAustriaCanadaChinaCzechiaDenmarkGermanyItalyJapanMexicoSaudi ArabiaSlovakiaSouth KoreaSpainTaiwanTurkey (Türkiye)United Kingdom
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Competitive Landscape -Hemophilia 41 trials
CompanyTickerTrialsLead PhaseDrugs
Novo Nordisk A/S Sponsored ADR Class BNVO16PHASE3Concizumab, Mim8, NNC0442-0344 A, Turoctocog alfa pegol, Nonacog beta pegol
Sanofi SA Sponsored ADRSNY6PHASE3Fitusiran, Clotting factor concentrates or bypassing agents, Antithrombin, Efanesoctocog Alfa BIVV001, Efanesoctocog alfa
Pfizer Inc.PFE8PHASE3PF-06838435/ fidanacogene elaparvovec, PF-07055480 : Recombinant AAV2/6 Human Factor VIII Gene Therapy, PF-06741086, marstacimab, PF-06838435
BioMarin Pharmaceutical Inc.BMRN3PHASE3Valoctocogene roxaparvovec
Regeneron Pharmaceuticals, Inc.REGN2PHASE1REGV131, LNP1265
Takeda Pharmaceutical Co. Ltd. Sponsored ADRTAK2PHASE1AskBio009, ADYNOVI/ADYNOVATE
Sangamo Therapeutics, Inc.SGMO1 — SB-318, SB-913, SB-FIX
Illumina, Inc.ILMN1 — Undisclosed
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT05611801primaryCompletionDate: changed
LOWMay 24, 2026NCT05611801studyFirstPostDate: changed