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ISU304/CB2679d/Dalcinonacog alfa 75~/kg

Phase 1

Hemophilia B | Monoclonal antibody | Hematology |Catalyst Pharmaceuticals, Inc.|Last Updated: Nov 10, 2020

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
CONTROLLEDDMC
Total Trials1
Total Enrollment11
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03186677Dose-escalation Study to Investigate the Safety, PK, and PD of ISU304/CB2679d in Hemophilia B PatientsPHASE1 COMPLETED 11Jun 3, 2017Feb 22, 2019Nov 10, 20203 South Korea
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Study Endpoints
Primary Endpoints
Number of Adverse Events (AEs) After the Administration of Investigational Products (IP)
Through study completion, an average of 8 days

The number of reported AEs (local/systemic/other) after IP administration was calculated by cohort.

Secondary Endpoints
Maximum Plasma Concentration (Cmax)
0 to 72 hours for Cohorts 1 to 3, 0 to 120 hours for Cohorts 4 and 5
Factor IX Inhibitor
At end of study visit (an average of 8 days)
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Cohort 1EXPERIMENTALSingle intravenous administration of BeneFIX (75 IU/kg) with 72 hours of observation, followed by single intravenous administration of ISU304/CB2679d/Dalcinonacog alfa (75 IU/kg) with 72 hours of observation
Cohort 2EXPERIMENTALSingle intravenous administration of ISU304/CB2679d/Dalcinonacog alfa (75 IU/kg) with 72 hours of observation, followed by single subcutaneous administration of ISU304/CB2679d/Dalcinonacog alfa (75 IU/kg) with 72 hours of observation
Cohort 3EXPERIMENTALSingle intravenous administration of ISU304/CB2679d/Dalcinonacog alfa (75 IU/kg) with 72 hours of observation, followed by single subcutaneous administration of ISU304/CB2679d/Dalcinonacog alfa (150 IU/kg) with 120 hours of observation
Cohort 4EXPERIMENTALOne subcutaneous administration of ISU304/CB2679d/Dalcinonacog alfa (150 IU/kg) per day for 6 days with 240 hours of observation
Cohort 5EXPERIMENTALOne intravenous administration of ISU304/CB2679d/Dalcinonacog alfa (75 IU/kg) followed by subcutaneous administration of ISU304/CB2679d/Dalcinonacog alfa (150 IU/kg) once daily for 9 days with 312 hours of observation
Interventions
NameTypeDescription
ISU304/CB2679d/Dalcinonacog alfa 75~150 IU/kgBIOLOGICALISU304/CB2679d/Dalcinonacog alfa 75\~150 IU/kg by intravenous or subcutaneous
BeneFIXBIOLOGICALBeneFIX 75 IU/kg, intravenous administration
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Eligibility Criteria
Age Range12 Years — 65 Years
SexALL
Healthy VolunteersNo
Study Sites3

Inclusion Criteria: 1. Previously treated male patients with moderate or severe hemophilia B (documented FIX activity ≤ 2% and exposed to any FIX product for ≥ 150 exposure days (estimated) at the time of screening) 2. Patients must be 12 to 65 years old at the time of screening 3. Patients who hav...

Countries:South Korea
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