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VX-407

Phase 2

Autosomal Dominant Polycystic Kidney Disease (ADPKD) | Small molecule | Nephrology |Vertex Pharmaceuticals Incorporated|Last Updated: May 13, 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 Trials4
Total Enrollment289
FDA Designations
No designations recorded
Clinical Trials (4)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT07161037Phase 2a Study of VX-407 in Participants With ADPKD Who Have a Subset of PKD1 Gene Variants (AGLOW)PHASE2 RECRUITING 24Nov 19, 2025Dec 31, 2027May 13, 202642 United States, Belgium +6
NCT07074327Effects of VX-407 on the Pharmacokinetics of Oral Contraceptives in Healthy ParticipantsPHASE1 COMPLETED 74Jul 11, 2025Feb 27, 2026Mar 30, 20261 United States
NCT07022119A Phase 1 Study Evaluating Safety, Tolerability, and Pharmacokinetics of VX-407 in Healthy ParticipantsPHASE1 COMPLETED 32Jun 12, 2025Feb 24, 2026Mar 27, 20261 United States
NCT06345755A Phase 1 Study to Evaluate Safety, Tolerability, and Pharmacokinetics of VX-407 in Healthy ParticipantsPHASE1 COMPLETED 159Apr 17, 2024Jun 13, 2025Jul 14, 20252 United States, Canada
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Study Endpoints
Primary Endpoints
htTKV on MRI Over Time
Baseline up to End of Study (Week 52)
Part A: Maximum Observed Plasma Concentration (Cmax) of LNG and EE in the Absence and Presence of VX-407
From Day 1 up to Day 7 and Day 21 up to Day 27
Part A: Area Under the Concentration Versus Time Curve From the Time of Dosing Extrapolated to Infinity (AUC0-inf) of LNG and EE in the Absence and Presence of VX-407
From Day 1 up to Day 7 and Day 21 up to Day 27
Part B (Optional): Cmax of Norelgestromin (NGMN) and Norgestrel (NG) (Active Metabolites of NGM) and EE in the Absence and Presence of VX-407
From Day 1 up to Day 9 and Day 23 up to Day 31
Part B (Optional): AUC0-inf of NGMN and NG (Active Metabolites of NGM) and EE in the Absence and Presence of VX-407
From Day 1 up to Day 9 and Day 23 up to Day 31
Part C (Optional): Cmax of NET and EE in the Absence and Presence of VX-407
From Day 1 up to Day 5 and Day 19 up to Day 23
Part C (Optional): AUC0-inf of NET and EE in the Absence and Presence of VX-407
From Day 1 up to Day 5 and Day 19 up to Day 23
Part D (Optional): Cmax of DRSP and EE in the Absence and Presence of VX-407
From Day 1 up to Day 7 and Day 21 up to Day 27
Part D (Optional): AUC0-inf of DRSP and EE in the Absence and Presence of VX-407
From Day 1 up to Day 7 and Day 21 up to Day 27
Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
From Enrollment up to Day 10
Part B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
From Enrollment up to Day 23
Secondary Endpoints
Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
From Day 1 up to Week 54
Maximum Observed Plasma Concentration (Cmax) of VX-407
From Day 1 up to Week 52
Area Under the Concentration Versus Time Curve (AUC) of VX-407
From Day 1 up to Week 52
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
VX-407EXPERIMENTALParticipants will receive VX-407 for up to 52 weeks.
Part A: VX-407 With Levonorgestrel/Ethinyl Estradiol (LNG/EE)EXPERIMENTALParticipants will receive a single dose of LNG/EE on Days 1 and 21 in fasted state. Participants will also receive VX-407 every 12 hours (q12h) from Days 8 through 26 in fasted state.
Part B (Optional): VX-407 With Norgestimate/Ethinyl Estradiol (NGM/EE)EXPERIMENTALParticipants will receive a single dose of NGM/EE on Days 1 and 23 in fasted state. Participants will also receive VX-407 q12h from Days 10 through 30 in fasted state.
Part C (Optional): VX-407 With Norethindrone/Ethinyl Estradiol (NET/EE)EXPERIMENTALParticipants will receive a single dose of NET/EE on Days 1 and 19 in fasted state. Participants will also receive VX-407 q12h from Days 6 through 22 in fasted state.
Part D (Optional): VX-407 With Drospirenone/Ethinyl Estradiol (DRSP/EE)EXPERIMENTALParticipants will receive a single dose of DRSP/EE on Days 1 and 21 in fasted state. Participants will also receive VX-407 q12h from Days 8 through 26 in fasted state.
Part A: Single Ascending DoseEXPERIMENTALParticipants will be randomized to receive a single dose of VX-407.
Placebo Part APLACEBO_COMPARATORParticipants will be randomized to receive placebo matched to VX-407.
Part B: Multiple Ascending DoseEXPERIMENTALParticipants will be randomized to receive multiple doses of VX-407.
Placebo Part BPLACEBO_COMPARATORParticipants will be randomized to receive placebo matched to VX-407.
Part A: Single Ascending Dose (SAD)EXPERIMENTALParticipants will be randomized to receive a single dose of different dose levels of VX-407.
Part A: PlaceboPLACEBO_COMPARATORParticipants will be randomized to receive placebo matched to VX-407.
Part B: Multiple Ascending Dose (MAD)EXPERIMENTALParticipants will be randomized to receive multiple doses of different dose levels of VX-407. The dose levels will be determined based on the data from Part A.
Part B: PlaceboPLACEBO_COMPARATORParticipants will be randomized to receive multiple doses of placebo matched to VX-407.
Part C: Drug-Drug InteractionEXPERIMENTALParticipants will be administered Midazolam (MDZ) in the presence or absence of VX-407. The dose levels will be determined based on the data from Part B.
Part DEXPERIMENTALParticipants will be randomized to receive VX-407 in 1 of 3 treatment sequences with 3 dosing periods to assess the relative bioavailability of VX-407 formulations and the effect of food on the pharmacokinetics of VX-407.
Interventions
NameTypeDescription
VX-407DRUGTablets for oral administration.
LNG/EEDRUGCombination Tablets for Oral Administration.
NGM/EEDRUGCombination Tablets for Oral Administration.
NET/EEDRUGCombination Tablets for Oral Administration.
DRSP/EEDRUGCombination Tablets for Oral Administration.
PlaceboDRUGSuspension for oral administration.
MidazolamDRUGSyrup for oral administration.
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Eligibility Criteria
Age Range18 Years — 65 Years
SexALL
Healthy VolunteersNo
Study Sites42

Key Inclusion Criteria: * A pre-existing diagnosis of ADPKD as defined in the protocol * Willing and able to comply with scheduled visits and other study procedures * Participants with ADPKD with Mayo imaging classification (MIC) status of 1B (with htTKV ≥250 mL/m), 1C, 1D, or 1E confirmed by abdom...

Countries:United StatesBelgiumCanadaFranceGermanyNetherlandsSpainUnited Kingdom
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Recent Changes (Last 90 Days)
MEDIUMMay 26, 2026NCT07074327TRIAL_REMOVED: changed
MEDIUMMay 26, 2026NCT07022119TRIAL_REMOVED: changed
LOWMay 26, 2026NCT07161037primaryCompletionDate: changed
LOWMay 24, 2026NCT07161037studyFirstPostDate: changed
LOWMay 24, 2026NCT07074327studyFirstPostDate: changed
LOWMay 24, 2026NCT07022119studyFirstPostDate: changed