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KB408

Phase 1

Alpha 1-Antitrypsin Deficiency | Small molecule | Rare Disease |Krystal Biotech, Inc.|Last Updated: Jul 22, 2025

Target and mechanism

ModalitySmall molecule

Also known as KB408 (Nebulization)

Success Probability

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Market & Valuation

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Trial Design

CONTROLLED
Total Trials1
Total Enrollment15

FDA Designations

ORPHAN_DRUG

Clinical trial landscape

KB408 · 1 trial · 1 indication

Phase 1 1
NCT06049082A Study of KB408 for the Treatment of Alpha-1 Antitrypsin DeficiencyAlpha 1-Antitrypsin Deficiency
RECRUITING15 Analytics
PHASE1RECRUITING
A Study of KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency
Alpha 1-Antitrypsin Deficiency
NCT0604908215
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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 to 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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Frequently asked questions about KB408

What is KB408 used for?

KB408 is an investigational small molecule being developed for the treatment of Alpha 1-Antitrypsin Deficiency, a rare genetic condition. It is currently in Phase 1 clinical development and has not been approved by the FDA. The drug is administered via nebulization.

Who makes KB408?

KB408 is being developed by Krystal Biotech, Inc., a biopharmaceutical company traded on the Nasdaq under the ticker symbol KRYS. The company is conducting a Phase 1 clinical trial of KB408 for Alpha 1-Antitrypsin Deficiency in the United States.

What phase is KB408 in?

KB408 is in Phase 1 clinical development. It is an investigational drug and has not received FDA approval. The ongoing Phase 1 study is currently recruiting participants with Alpha 1-Antitrypsin Deficiency in the United States.

What clinical trials is KB408 in?

KB408 is being studied in a Phase 1 clinical trial registered as NCT06049082, titled "A Study of KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency." The trial is recruiting 15 participants aged 18 years and older in the United States and is currently active.

Is KB408 the same as KB408 (Nebulization)?

Yes, KB408 is also known as KB408 (Nebulization), reflecting its route of administration. The drug is delivered via nebulization for the treatment of Alpha 1-Antitrypsin Deficiency. It is currently in Phase 1 clinical development by Krystal Biotech.

Has KB408 received FDA orphan drug designation?

Yes, KB408 has received orphan drug designation from the FDA for the treatment of Alpha 1-Antitrypsin Deficiency. This designation is granted to drugs intended for the treatment of rare diseases and may provide certain development incentives.