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VX-634

Phase 1

Alpha 1-Antitrypsin Deficiency | Small molecule | Other |Vertex Pharmaceuticals Incorporated|Last Updated: Mar 12, 2024

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_CONTROLLEDBiomarker
Total Trials1
Total Enrollment127
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05579431A Phase 1, First-in-human Study of VX-634PHASE1 COMPLETED 127Oct 13, 2022Nov 13, 2023Mar 12, 20245 United States
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Study Endpoints
Primary Endpoints
Part A and B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Day 1 up to Day 20
Secondary Endpoints
Part A and B: Maximum Observed Plasma Concentration (Cmax) of VX-634
Day 1 up to Day 20
Part A and B: Area Under the Concentration Versus Time Curve (AUC) of VX-634
Day 1 up to Day 20
Part A and B: Urine Concentration of VX-634
Part A Cohort A3: Days 1 and 5; Part B Cohort B3: Days 1, 10 and 11
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Part AEXPERIMENTALParticipants grouped in different cohorts will receive a single ascending dose of VX-634.
Placebo Part APLACEBO_COMPARATORParticipants will be randomized to receive placebo matched to VX-634.
Part BEXPERIMENTALParticipants grouped in different cohorts will receive multiple doses of VX-634.The dose levels will be determined based on the data from Part A.
Placebo Part BPLACEBO_COMPARATORParticipants will be randomized to receive placebo matched to VX-634.
Interventions
NameTypeDescription
VX-634DRUGSuspension for oral administration.
PlaceboDRUGSuspension for oral administration.
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Eligibility Criteria
Age Range18 Years — 55 Years
SexALL
Healthy VolunteersYes
Study Sites5

Key Inclusion Criteria: * Participants of age between 18 to 55 years (inclusive) * Body mass index (BMI) of 18.0 to 32.0 kilogram per meter square (kg/m\^2) * A total body weight of more than (\>)50 kg * Participants of non childbearing potential * Non smoker or ex-smoker for at least 3 months befo...

Countries:United States
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