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KB408

Phase 1

Alpha 1-Antitrypsin Deficiency | Small molecule | Other |Krystal Biotech, Inc.|Last Updated: Jul 22, 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
CONTROLLEDBiomarker
Total Trials1
Total Enrollment15
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06049082A Study of KB408 for the Treatment of Alpha-1 Antitrypsin DeficiencyPHASE1 RECRUITING 15 — — Feb 15, 2024Dec 1, 2026Jul 22, 20253 United States
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Study Endpoints
Primary Endpoints
To evaluate safety and tolerability of KB408 based upon assessment of adverse events (frequency, severity, relatedness), and changes from baseline in vital signs, ECG, spirometry, and clinical laboratory test results
2 months (Cohorts 1, 2, 3); 3 months (Cohort 2b)

Number of subjects with treatment related adverse events as assessed by NCI-CTCAE v5

Secondary Endpoints
To assess the effect of KB408 on serum alpha-1 antitrypsin (AAT) concentration
2 months (Cohorts 1, 2, 3); 3 months (Cohort 2b)
To assess the effect of KB408 on plasma neutrophil elastase (NE) concentration
2 months (Cohorts 1, 2, 3); 3 months (Cohort 2b)
To evaluate the effect of KB408 on AAT concentration in the lung
2 months (Cohorts 1, 2, 3); 3 months (Cohort 2b)
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Cohort 1: Low dose KB408EXPERIMENTALSingle dose of KB408 (low dose)
Cohort 2: Mid dose KB408EXPERIMENTALSingle dose of KB408 (mid dose)
Cohort 3: High dose KB408EXPERIMENTALSingle dose of KB408 (high dose)
Cohort 2b: Mid dose KB408EXPERIMENTALMultiple doses of KB408 (mid dose)
Interventions
NameTypeDescription
KB408 (Nebulization)DRUGNebulized solution of KB408, a replication-defective HSV-1 vector expressing full length human SERPINA1
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Eligibility Criteria
Age Range18 Years — 70 Years
SexALL
Healthy VolunteersNo
Study Sites3

Inclusion Criteria: 1. The subject or legally authorized representative must have read, understood, and signed an Institutional Review Board (IRB) approved Informed Consent Form and must be willing and able to comply with study procedures and instructions. 2. Subject is aged ≥18 to ≤70 years, at th...

Countries:United States
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT06049082primaryCompletionDate: changed
LOWMay 24, 2026NCT06049082studyFirstPostDate: changed