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MOD-5014

Phase 1

Hemophilia A or B With Inhibitors | Monoclonal antibody | Hematology |Opko Health Inc.|Last Updated: Sep 30, 2019

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
RandomizedPLACEBO_CONTROLLEDDMC
Total Trials1
Total Enrollment32
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02919800A Single-dose, Dose-escalation Study of a Long-acting MOD-5014 in Healthy Adult MalePHASE1 COMPLETED 32Jan 22, 2017Feb 21, 2018Sep 30, 20191 Israel
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Study Endpoints
Primary Endpoints
Composite safety and tolerability parameters as measured by adverse events, electrocardiograms (ECG), Immunogenicity, laboratory results, vital signs and injection site reactions
within 30 days of injection
Secondary Endpoints
Cmax of MOD-5014
within 30 days of injection
Tmax of MOD-5014
within 30 days of injection
AUC(0-t) of MOD-5014
within 30 days of injection
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Study Design & Arms
AllocationRANDOMIZED
MaskingSINGLE
ModelPARALLEL
PurposeOTHER
Treatment Arms
ArmTypeDescription
MOD-5014EXPERIMENTALMOD-5014 longevity is the result of fusion of three consecutive C-terminal peptide (CTP) domains to the C-terminus of FVII. CTP technology is based on a natural peptide, the C-terminal peptide of the beta chain of human chorionic gonadotropin (hCG), which provides hCG with the required longevity to maintain pregnancy (initial half-life \[t1/2\] \~10 h, terminal t1/2 \~37 h). The beta chain of luteinizing hormone (LH), a gonadotropin that triggers ovulation, is almost identical to hCG but does not include the CTP. As a result, LH has a significantly shorter half-life in blood (initial t1/2 \~1 h, terminal t1/2 \~10 h) (Fares et al., 1992).
MOD-5014 PlaceboPLACEBO_COMPARATORPlacebo solution for SC injection containing the same inactive ingredients used in the active drug product at matching volumes
Interventions
NameTypeDescription
MOD-5014BIOLOGICALMOD-5014, a long-acting modified recombinant Factor VIIa
MOD-5014 PlaceboOTHERPlacebo solution for SC injection containing the same inactive ingredients used in the active drug product at matching volumes
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Eligibility Criteria
Age Range18 Years — 50 Years
SexMALE
Healthy VolunteersYes
Study Sites1

Inclusion Criteria: 1. Men, 18-50 years of age, inclusive, at the screening visit. 2. Subjects must provide written informed consent prior to participating in the study. 3. Considered healthy based on medical history, physical examination and clinical laboratory results. 4. Body Mass Index (BMI) 19...

Countries:Israel
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