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Prochymal

Phase 3

Graft Versus Host Disease | Small molecule | Immunology |Mesoblast Limited|Last Updated: Oct 30, 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-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials3
Total Enrollment463
FDA Designations
No designations recorded
Clinical Trials (3)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT00562497Efficacy and Safety of Prochymal® Infusion in Combination With Corticosteroids for the Treatment of Newly Diagnosed Acute Graft Versus Host Disease (GVHD)PHASE3 COMPLETED 192Jan 31, 2008May 20, 2010Jan 19, 202253 United States, Australia +1
NCT00366145Efficacy and Safety of Adult Human Mesenchymal Stem Cells to Treat Steroid Refractory Acute Graft Versus Host Disease (GVHD)PHASE3 COMPLETED 260Aug 17, 2006May 28, 2009Feb 10, 202270 United States, Australia +4
NCT00284986Safety and Efficacy of Prochymal for the Salvage of Treatment-Refractory Acute GVHD ParticipantsPHASE2 COMPLETED 11Nov 18, 2005Feb 8, 2007Oct 30, 20251 United States
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Study Endpoints
Primary Endpoints
Percentage of Participants with Treatment Success
90 Days

Treatment was considered a success if all of the following conditions were met: Achieved induction of a complete response (CR) within 28 days after first infusion; CR followed by 28 days maintenance of a clinically meaningful response defined as the response that did not require an increase in corticosteroid dose (methylprednisolone doses \>2 milligram/kilogram/day \[mg/kg/d\] or prednisone doses \>2.5 mg/kg/d) for more than 7 consecutive days; Did not require second line/escalation therapy through Study Day 56; and survived 90 study days.

Percentage of Participants achieving Complete Response of Greater Than or Equal to 28 Days Duration
up to 100 Days post first infusion

A complete response was defined as complete resolution of all clinical signs of Graft versus host disease (GVHD)- that had to be maintained for at least 28 consecutive days (durable complete response \[DCR\]) within 100 days post first infusion.

Response by Day 28
Day 28

Responses included complete response (CR), partial response (PR), failure to respond. CR is defined as a complete resolution of graft-versus-host disease (GVHD). PR is defined as improvement in at least one organ by at least one full stage in the absence of progression in any other organ, or resolution of GVHD in at least one organ with a need for additional treatment because of abnormalities persisting in another organ. Failure to respond is defined as progression of GVHD.

Secondary Endpoints
Percentage of Participants with Overall Response
Day 90
Percentage of Participants with Induction of a 2-grade decrease in (Graft Versus Host Disease) GVHD by Study Day 28 with maintenance of a 2-grade decrease in GVHD through Study Day 56
Up to Day 56
Percentage of Participants with Induction of CR lasting for greater than or equal to 14 Days
Day 14
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Study Design & Arms
AllocationRANDOMIZED
MaskingTRIPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
PlaceboPLACEBO_COMPARATORParticipants will receive 6 infusions of placebo-matching Prochymal® intravenously (IV) during the first 4 weeks of the study. The first infusion will be administered within 72 hours of the start of systemic corticosteroid therapy. Participants will receive 4 infusions during the first 2 weeks (twice weekly at least 3 days apart), followed by 2 infusions administered once weekly over the subsequent 2 weeks up to Day 28.
Prochymal® 2x10^6 hMSC/kgACTIVE_COMPARATORParticipants will receive 6 infusions of Prochymal® 2x10\^6 human mesenchymal stem cells (hMSC)/kg IV during the first 4 weeks of the study. The first infusion will be administered within 72 hours of the start of systemic corticosteroid therapy. Participants will receive 4 infusions during the first 2 weeks (twice weekly at least 3 days apart), followed by 2 infusions administered once weekly over the subsequent 2 weeks up to Day 28.
Prochymal®ACTIVE_COMPARATORParticipants who receive standard of care plus treatment with ex vivo cultured adult human mesenchymal stem cells.
PROCHYMAL™EXPERIMENTAL -
Interventions
NameTypeDescription
Prochymal®DRUGProchymal® intravenous infusion.
PlaceboOTHERPlacebo-matching Prochymal® intravenous infusion.
CorticosteroidOTHERAdministration will be intravenously as prescribed by the caregiver.
Standard of Care for GVHDDRUGInstitutionally defined standard of care (e.g., maintenance of steroid treatment and the addition of a second-line therapy)
ProchymalDRUGIntravenous infusion of ex-vivo cultured adult human mesenchymal stem cells
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Eligibility Criteria
Age Range18 Years — 70 Years
SexALL
Healthy VolunteersNo
Study Sites53

Inclusion Criteria: * Participants must be 18 years to 70 years of age, inclusive * Participants must have received an allogeneic hematopoietic stem cell transplant using either bone marrow, peripheral blood stem cells or cord blood or administered a donor leukocyte infusion. * Participants must ha...

Countries:United StatesAustraliaCanadaItalySwitzerlandUnited Kingdom
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