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HMPL-523

Phase 3

Primary Immune Thrombocytopenia (ITP) | Small molecule | Hematology |HUTCHMED (China) Limited|Last Updated: May 4, 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_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment272
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05029635Phase III Study on HMPL-523 for Treatment of ITPPHASE3 COMPLETED 272Oct 27, 2021Sep 18, 2025May 4, 202637 China
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Study Endpoints
Primary Endpoints
the durable response rate in the primary study
treatment period Week14-Week24

Platelet count ≥50×10\^9 /L on at least 4 of 6 scheduled visits of Week14-Week24 in the primary study

Secondary Endpoints
the overall response rate in the primary study
treatment period Week1-Week24 in the primary study
Incidence of treatment emergent adverse events
treatment period Week1-Week24 in the primary study
Plasma concentration at steady state 2 hours post dose (C2h,ss)
treatment period Week1-Week24 in the primary study
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Drug: HMPL-523ACTIVE_COMPARATORPrimacy study (Randomized, Double-Blind Phase): Eligible subjects receive 300 mg HMPL-523 treatment once daily for 24 weeks. Sub study (Open-label Phase): Eligible subjects receive 300 mg HMPL-523 treatment once daily for 76 weeks after the enrollment of the last patient enrolled in open-label phase.
Drug: placeboPLACEBO_COMPARATORPrimacy study (Randomized, Double-Blind Phase): Eligible subjects will receive 300 mg HMPL-523 matched placebo treatment once daily for 24 weeks.
Interventions
NameTypeDescription
HMPL-523DRUGHMPL-523(300mg PO QD)
PlaceboDRUGPlacebo(300mg PO QD)
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Eligibility Criteria
Age Range18 Years — 75 Years
SexALL
Healthy VolunteersNo
Study Sites37

Inclusion Criteria for double-blind phase: 1. Voluntary signature of written informed consent form; 2. Male or female aged 18\~75 years; 3. Performance Status score \[Eastern Cooperative Oncology Group (ECOG) score\] 0\~1; 4. Having been diagnosed as ITP prior to randomization, and duration of dise...

Countries:China
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