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MMB

Phase 2

Primary Myelofibrosis (PMF) | Small molecule | Hematology |GSK plc|Last Updated: Jun 18, 2023

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
UNCONTROLLEDBiomarker
Total Trials1
Total Enrollment41
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02515630Momelotinib in Transfusion-Dependent Adults With Primary Myelofibrosis (PMF) or Post-polycythemia Vera or Post-essential Thrombocythemia Myelofibrosis (Post-PV/ET MF)PHASE2 COMPLETED 41Jan 29, 2016Aug 15, 2017Jun 18, 202313 United States, Canada
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Study Endpoints
Primary Endpoints
Transfusion Independence Response by Week 24
From baseline to Week 24

The percentage of subjects who became transfusion independent for ≥ 12 weeks at any time on study. A subject was considered transfusion independent on study if no RBC transfusion occurred in any 12-week period during the 24-week treatment period.

Secondary Endpoints
Transfusion Response Rate by Week 24
From baseline to Week 24
Splenic Response Rate at Week 24
Measured at Week 24
Response Rate in Total Symptom Score (TSS) at Week 24
Measured at Week 24
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
MomelotinibEXPERIMENTALMMB for 24 weeks (± 7 days)
Interventions
NameTypeDescription
MMBDRUGMomelotinib (MMB) tablet administered orally once daily
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites13

Key Inclusion Criteria: * Diagnosis of PMF or Post PV/ET-MF * Requires myelofibrosis therapy, in the opinion of the investigator * High risk OR intermediate-2 risk defined by dynamic international prognostic scoring system (DIPSS) OR intermediate-1 risk defined by DIPSS and associated with symptoma...

Countries:United StatesCanada
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