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PTG-300

Phase 2

β-thalassemia | Small molecule | Hematology |Protagonist Therapeutics, Inc.|Last Updated: Jul 16, 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
UNCONTROLLEDDMCBiomarker
Total Trials2
Total Enrollment97
FDA Designations
No designations recorded
Clinical Trials (2)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04054921Safety Study for Beta Thalassemia Subjects on PTG-300PHASE2 COMPLETED 34May 10, 2019Jul 31, 2020Jul 16, 202116 United States, Greece +6
NCT03802201Study of PTG-300 in Non-Transfusion Dependent and Transfusion-Dependent Beta-Thalassemia Subjects With Chronic AnemiaPHASE2 COMPLETED 63Dec 19, 2018Jul 31, 2020Jul 16, 202133 United States, Greece +7
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Study Endpoints
Primary Endpoints
Proportion of subjects with side effects and severity of side effects will be tabulated
Over two year Period after receiving PTG-300

the long-term safety and tolerability of PTG-300 in Beta Thalassemia.

NTD: Proportion of responders at each dose
4 week period

NTD subjects who achieve an increase in Hgb without transfusion

TD: Proportion of clinical responders at each dose
8 week period

TD subjects who achieve a reduction in red blood cell (RBC) units required over an 8 week period

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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
InterventionsEXPERIMENTALPTG-300
PTG-300 ActiveEXPERIMENTALDrug: PTG-300 Subcutaneous
Interventions
NameTypeDescription
PTG-300DRUGPTG-300 is a hepcidin Mimetic
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Eligibility Criteria
Age Range12 Years — 65 Years
SexALL
Healthy VolunteersNo
Study Sites16

Inclusion Criteria: * NTD and TD β-thalassemia subjects who completed Week 12 and Week 16 respectively in Study PTG-300-02. Exclusion Criteria: * Subjects who discontinued prematurely from study 300-02 (before Week 12 in NTD and Week 16 in TD).

Countries:United StatesGreeceLebanonMalaysiaThailandTunisiaTurkey (Türkiye)United KingdomItaly
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