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Remestemcel-L-rknd

Phase 3

GVHD - Graft-Versus-Host Disease | Small molecule | Immunology |Mesoblast Limited|Last Updated: May 5, 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
RandomizedDouble-BlindPLACEBO_CONTROLLEDBiomarker
Total Trials1
Total Enrollment180
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT07568535A Randomized, Double-Blind, Placebo-Controlled, Multicenter Phase III Trial of Remestemcel-L-rknd Added to Ruxolitinib for Grade III-IV Steroid-Refractory Acute Graft-Versus-Host DiseasePHASE3 NOT YET_RECRUITING 180Aug 31, 2026Jul 1, 2028May 5, 2026 -
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Study Endpoints
Primary Endpoints
Overall response
28 days post randomization

The primary clinical endpoint is overall response at Day 28 post-randomization. Overall response is defined as achieving a PR or CR on Day 28 post-randomization without requirement for new intervening systemic immunosuppressive therapies

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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Remestemcel-L-rkndEXPERIMENTALRemestemcel-L will be administered as intravenous infusions
PlaceboPLACEBO_COMPARATORPTM will be administered as intravenous infusions
Interventions
NameTypeDescription
Remestemcel-L-rkndDRUGRemestemcel-L is comprised of ex-vivo cultured adult human MSC
PlaceboOTHERPlacebo is infusion based to be given at the same points as remestemcel-L-rknd
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo

Inclusion Criteria: 1. Age 18 years or older at the time of enrollment. 2. Able to take oral medications 3. Have Grade III-IV SR-aGVHD at the time of enrollment, defined as aGVHD resistant to high dose corticosteroids at a dose of ≥ 1 mg/kg/day methylprednisolone (or equivalent), given alone or in ...

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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT07568535primaryCompletionDate: changed
LOWMay 24, 2026NCT07568535studyFirstPostDate: changed
LOWMay 21, 2026NCT07568535NEW_TRIAL: changed
LOWMay 21, 2026NCT07568535NEW_TRIAL: changed