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VX-433

Phase 1

Narcolepsy Type 1 (NT1) | Small molecule | Other |Vertex Pharmaceuticals Incorporated|Last Updated: Jun 8, 2026

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 Enrollment118
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT07584434A Phase 1, First-in-human Study of VX-433PHASE1 RECRUITING 118May 21, 2026Mar 5, 2027Jun 8, 20261 United States
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Study Endpoints
Primary Endpoints
Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
From Day 1 up to Day 6
Part B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
From Day 1 up to Day 18
Part C: Maximum Observed Plasma Concentration (Cmax) of Midazolam in the Absence and Presence of VX-433
From Day 1 up to Day 22
Part C: Maximum Observed Plasma Concentration (Cmax) of Bupropion and Hydroxybupropion in the Absence and Presence of VX-433
From Day 1 up to Day 22
Part C: Area Under the Concentration Versus Time Curve (AUC) of Midazolam in the Absence and Presence of VX-433
From Day 1 up to Day 22
Part C: Area Under the Concentration Versus Time Curve (AUC) of Bupropion and Hydroxybupropion in the Absence and Presence of VX-433
From Day 1 up to Day 22
Secondary Endpoints
Part A: Maximum Observed Plasma Concentration (Cmax) of VX-433
From Day 1 up to Day 6
Part B: Maximum Observed Plasma Concentration (Cmax) of VX-433
From Day 1 up to Day 18
Part A: Area Under the Concentration Versus Time Curve (AUC) of VX-433
From Day 1 up to Day 6
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Part A: Single Ascending Dose (SAD)EXPERIMENTALParticipants will be randomized to receive a single dose of VX-433.
Part A: PlaceboPLACEBO_COMPARATORParticipants will be randomized to receive placebo matched to VX-433.
Part B: Multiple Ascending Dose (MAD)EXPERIMENTALParticipants will be randomized to receive multiple doses of VX-433.
Part B: PlaceboPLACEBO_COMPARATORParticipants will be randomized to receive placebo matched to VX-433.
Part C: Drug Drug InteractionEXPERIMENTALParticipants will receive a single dose of midazolam and bupropion on Day 1 in Dosing Period 1, followed by VX-433 administration for 17 days (Days 5 through 21) and then midazolam and bupropion on Day 18 in Dosing Period 2. Part C will be open-label.
Interventions
NameTypeDescription
VX-433DRUGSuspension for Oral Administration
PlaceboDRUGSuspension for Oral Administration
MidazolamDRUGSyrup for Oral Administration
BupropionDRUGCapsule for Oral Administration
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Eligibility Criteria
Age Range18 Years — 55 Years
SexALL
Healthy VolunteersYes
Study Sites1

Key Inclusion Criteria: * Body mass index (BMI) of 18.0 to 30.0 kilogram per meter square (kg/m2), inclusive * A total body weight of more than (\>) 50 kg * Nonsmoker or ex-smoker for at least 3 months before screening with current nonsmoking status confirmed by urine or blood cotinine at screening...

Countries:United States
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Recent Changes (Last 90 Days)
LOWJun 8, 2026NCT07584434Status: NOT_YET_RECRUITING → RECRUITING
LOWJun 8, 2026NCT07584434Status: NOT_YET_RECRUITING → RECRUITING
LOWJun 8, 2026NCT07584434Status: NOT_YET_RECRUITING → RECRUITING
LOWMay 26, 2026NCT07584434primaryCompletionDate: changed
LOWMay 24, 2026NCT07584434studyFirstPostDate: changed
LOWMay 21, 2026NCT07584434NEW_TRIAL: changed
LOWMay 21, 2026NCT07584434NEW_TRIAL: changed