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Alpha 1-Antitrypsin

Phase 3

Alpha 1-Antitrypsin Deficiency | Small molecule | Other |Kamada Ltd.|Last Updated: Sep 4, 2025

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-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment220
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04204252Phase III, Efficacy and Safety of "Kamada-AAT for Inhalation"PHASE3 RECRUITING 220Nov 25, 2019Jun 1, 2031Sep 4, 20259 Belgium, Finland +4
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Study Endpoints
Primary Endpoints
FEV1 post bronchodilator
104 weeks

Change from baseline in post bronchodilator FEV1 at 104 weeks

Secondary Endpoints
CT densitometry whole-lung 15th percentile lung density (PD15) at total lung capacity (TLC).
104 weeks
FEV1 % of predicted
104 weeks
FEV1/FVC %
104 weeks
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Inhaled AATACTIVE_COMPARATORDaily inhalation of 80 mg/day "Kamada-AAT for Inhalation" for 104 weeks
PlaceboPLACEBO_COMPARATORDaily inhalation of a solution of NaCl in phosphate buffer solution with 0.01% TWEEN-80
Interventions
NameTypeDescription
Alpha 1-AntitrypsinDRUGKamada's alpha 1-antitrypsin product given by inhalation using the eFlow® electronic nebulizer manufactured by PARI Pharma GmbH
PlaceboDRUGPreparation of NaCl in phosphate buffer solution with 0.01% TWEEN-80
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Eligibility Criteria
Age Range18 Years — 65 Years
SexALL
Healthy VolunteersNo
Study Sites9

Inclusion Criteria 1. Diagnosis of severe AAT deficiency, i.e. patients with either Pi(ZZ), Pi(Z/Null), or Pi(Null/Null) genotypes. 2. Serum AAT levels ≤ 11 µM at screening. 3. Lung disease with clinical evidence of airflow limitation (post bronchodilator FEV1/SVC≤70%) at screening. 4. 40% ≤ FEV1 ≤...

Countries:BelgiumFinlandIrelandNetherlandsSwedenUnited Kingdom
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT04204252primaryCompletionDate: changed
LOWMay 24, 2026NCT04204252studyFirstPostDate: changed