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SPD602

Phase 2

Transfusional Iron Overload | Small molecule | Hematology |Takeda Pharmaceutical Company Limited|Last Updated: Jun 10, 2021

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
RandomizedCONTROLLEDBiomarker
Total Trials1
Total Enrollment51
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01186419Safety and Pharmacodynamic Study of an Oral Iron Chelator Given for 6 Months to Patients With Iron OverloadPHASE2 COMPLETED 51Aug 13, 2010Jan 8, 2013Jun 10, 20219 United States, Italy +3
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Study Endpoints
Primary Endpoints
Change From Baseline in Liver Iron Concentration (LIC) at 96 Weeks
Baseline and 96 weeks

LIC was determined by R2 Magnetic Resonance Imaging (MRI).

Secondary Endpoints
Maximum Plasma Concentration (Cmax) of SPD602
92 weeks
Area Under The Steady-state Plasma Concentration-time Curve (AUC) of SPD602
92 weeks
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Study Design & Arms
AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
SPD602 (16mg)EXPERIMENTAL -
SPD602 (32mg)EXPERIMENTAL -
Interventions
NameTypeDescription
SPD602 (FBS0701, SSP-004184)DRUGOral FBS0701 taken one time daily for up to 96 weeks.
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Eligibility Criteria
Age Range18 Years — 60 Years
SexALL
Healthy VolunteersNo
Study Sites9

Inclusion Criteria: * Transfusional iron overload due to: hereditary anemias such as sickle cell disease, β-thalassemia and Diamond-Blackfan anemia; acquired anemias such as Myelodysplastic Syndrome and other forms of bone marrow failure. Patients must also be transfusion-dependent and require chro...

Countries:United StatesItalyThailandTurkey (Türkiye)United Kingdom
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