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Biological: narsoplimab

Phase 2

Thrombotic Microangiopathies | Small molecule | Other |Omeros Corporation|Last Updated: Mar 25, 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
UNCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment18
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05855083Efficacy and Safety Study of Narsoplimab in Pediatric Patients With High-Risk Hematopoietic Stem Cell Transplant TMAPHASE2 RECRUITING 18May 1, 2023Dec 1, 2025Mar 25, 202516 United States, Germany +3
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Study Endpoints
Primary Endpoints
100-day survival rate following high-risk HSCT-TMA diagnosis.
100 days

Percentage of patients alive at 100 days following the diagnosis of high-risk HSCT-TMA

Secondary Endpoints
Number of participants with treatment-emergent adverse events assessed by CTCAE v5.0
52 weeks
Percentage of patients meeting protocol definition of clinical response
52 weeks
52 week survival rate following high-risk HSCT-TMA diagnosis
52 weeks
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Narsoplimab single arm-treatmentEXPERIMENTALNarsoplimab 4 mg/kg
Interventions
NameTypeDescription
Biological: narsoplimabDRUGTreatment with narsoplimab 4 mg/kg will be administered
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Eligibility Criteria
Age Range28 Days — 17 Years
SexALL
Healthy VolunteersNo
Study Sites16

Inclusion Criteria: 1. Age at least 28 days and less than 18 years prior to informed consent (Visit 0). 2. Have informed consent from at least one parent or legal guardian as required by local law and regulation. Patient informed consent will be required if the patient has reached the local legal a...

Countries:United StatesGermanyIsraelNetherlandsSpain
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT05855083primaryCompletionDate: changed
LOWMay 24, 2026NCT05855083studyFirstPostDate: changed