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LJPC-401

Phase 2

Hereditary Hemochromatosis | Small molecule | Other |Innoviva, Inc.|Last Updated: Jun 9, 2022

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_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment70
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03395704A Study of LJPC-401 for the Treatment of Iron Overload in Adult Patients With Hereditary HemochromatosisPHASE2 COMPLETED 70Nov 29, 2017Oct 28, 2019Jun 9, 202231 United States, Australia +2
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Study Endpoints
Primary Endpoints
Effect of LJPC-401 Versus Placebo on Blood Iron Levels
16 Weeks

Percentage change in transferrin saturation (TSAT) as measured by blood laboratory tests.

Secondary Endpoints
Effect of LJPC-401 Versus Placebo on Number of Phlebotomies
16 Weeks
Effect of LJPC-401 Versus Placebo on Blood Iron Levels
16 Weeks
Effect of LJPC-401 Versus Placebo on the Total Number of Treatment-emergent Adverse Events
20 Weeks
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Study Design & Arms
AllocationRANDOMIZED
MaskingSINGLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
LJPC-401ACTIVE_COMPARATORLJPC-401 solution for subcutaneous injection only, 5mg/1 mL (5mg/mL) or 10mg/1mL (10mg/mL) single use vial
PlaceboPLACEBO_COMPARATOR0.9% Sodium Chloride Injection, USP, or equivalent
Interventions
NameTypeDescription
LJPC-401DRUGLJPC-401 subcutaneous injection, up to 20 mg weekly for 16 weeks. The minimum weekly dose will be 5 mg and the maximum weekly dose of LJPC-401 will be 20 mg.
PlaceboDRUG0.9% Sodium Chloride Injection, USP, or equivalent
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites31

Inclusion Criteria: 1. Patients with clinical diagnosis of hereditary hemochromatosis 2. Patients who are prescribed therapeutic phlebotomy for treatment of hereditary hemochromatosis 3. Patients with serum ferritin and TSAT levels above treatment guidelines 4. Female patients of child bearing pote...

Countries:United StatesAustraliaFranceUnited Kingdom
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