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Remestemcel-L

Phase 3

Grade B Acute Graft Versus Host Disease | Monoclonal antibody | Immunology |Mesoblast Limited|Last Updated: Mar 16, 2022

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 Enrollment32
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02652130Safety Follow-up of Treatment With Remestemcel-L in Pediatric Participants Who Have Failed to Respond to Steroid Treatment for Acute GVHDPHASE3 COMPLETED 32Oct 28, 2015Jun 15, 2018Mar 16, 202220 United States
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Study Endpoints
Primary Endpoints
Overall Survival Rate Through Day 180
From Baseline Day 1 in the Study MSB-GVHD001 up to Day 180 in Study MSB-GVHD002 (180 days)

The overall survival rate is defined as the percentage of participants alive at the given time point. OS is defined as the time to death from the start of drug therapy.

Secondary Endpoints
Overall Survival Rate at Day 180 for Participants Who Had Overall Response (OR) at Day 28 of Study MSB-GVHD001
From Baseline (Day 1) in the Study MSB-GVHD001 up to Day 180 in the Study MSB-GVHD002 (180 days)
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeOTHER
Treatment Arms
ArmTypeDescription
Safety populationEXPERIMENTALAll participants who were enrolled and had received at least 1 dose of remestemcel-L in Study MSB-GVHD001.
Interventions
NameTypeDescription
Remestemcel-LBIOLOGICALNo intervention was given in Study MSB-GVHD002 (NCT02652130). It was a safety follow-up trial of remestemcel-L-treated participants from Study MSB-GVHD001.
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Eligibility Criteria
Age Range2 Months — 17 Years
SexALL
Healthy VolunteersNo
Study Sites20

Inclusion Criteria: * Participants must have participated in MSB-GVHD001 and have received at least one infusion of remestemcel-L. * Participant or participant's authorized representative must be capable of providing written informed consent. Assent, if applicable, must also be collected when requi...

Countries:United States
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