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ABP 959

Phase 3

Paroxysmal Nocturnal Hemoglobinuria | Small molecule | Other |Amgen Inc.|Last Updated: May 23, 2023

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
RandomizedDouble-BlindCONTROLLEDDMC
Total Trials1
Total Enrollment42
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03818607A Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Participants With PNHPHASE3 COMPLETED 42Jan 22, 2019Jul 12, 2022May 23, 202324 United States, Czechia +12
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Study Endpoints
Primary Endpoints
LDH Level at Week 27 (Parallel Comparison)
Week 27

The primary analysis for the parallel comparison was hemolysis as measured by LDH at Week 27 by initial treatment received (Period 1).

Time-adjusted Area Under the Effect Curve (AUEC) of LDH (Crossover Comparison Per Assigned Treatment)
From Week 13 to Week 27, from Week 39 to Week 53, and from Week 65 to Week 79

The primary analysis for the crossover comparison was hemolysis, as measured by the time-adjusted AUEC of LDH, according to treatment assigned during each of the 14-week assessments during Periods 1 and 2.

Secondary Endpoints
Mean Total Complement (50% Total Hemolytic Complement Activity [CH50])
Baseline, Week 27, Week 39, Week 53, Week 65, and Week 79
Mean Total Hemoglobin Levels
Baseline, Week 27, Week 39, Week 53, Week 65, and Week 79
Mean Serum-free Hemoglobin Levels
Baseline, Week 27, Week 39, Week 53, Week 65, and Week 79
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelCROSSOVER
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
T (ABP 959) / R (eculizumab)OTHERABP 959 for 52 weeks in Period 1 followed by eculizumab for 26 weeks in Period 2
R (eculizumab) / T (ABP 959)OTHEREculizumab for 52 weeks in Period 1 followed by ABP 959 for 26 weeks in Period 2
Interventions
NameTypeDescription
ABP 959DRUGintravenous infusion
EculizumabDRUGintravenous infusion
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites24

Inclusion Criteria: * Men and women ≥ 18 years of age. * Historical diagnosis of PNH. * Administration of eculizumab for ≥ 6 months and currently receiving 900 mg of eculizumab. * Hemoglobin ≥ 9.0 g/dL for at least 6 weeks before randomization. * Lactate dehydrogenase \< 1.5 × the upper limit of no...

Countries:United StatesCzechiaFinlandFranceIrelandItalyNetherlandsNorwayPortugalSloveniaSpainSwedenTurkey (Türkiye)United Kingdom
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