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VX-840

Phase 1

APOL1-mediated Kidney Disease | Small molecule | Nephrology |Vertex Pharmaceuticals Incorporated|Last Updated: Dec 8, 2022

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 Enrollment88
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05324410A Phase 1 Dose Escalation Study to Evaluate Safety and Pharmacokinetics (PK) of VX-840 in Healthy ParticipantsPHASE1 COMPLETED 88Apr 7, 2022Nov 7, 2022Dec 8, 20221 United States
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Study Endpoints
Primary Endpoints
Parts A and 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 25
Secondary Endpoints
Parts A and B: Plasma Concentration of VX-840 Over Time
From Day 1 up to Day 14
Part A: Area Under the Concentration Versus Time Curve From the Time of Dosing Extrapolated to Infinity [AUC(0 - inf)]
From Day 1 up to Day 14
Part B: Area Under the Concentration Versus Time Curve From the Time of Dosing to the Last Measurable Concentration [AUC(0-tlast)]
From Day 1 up to Day 14
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Part A: VX-840EXPERIMENTALParticipants will be randomized to receive single dose of one of different dose levels of VX-840.
Part A: PlaceboPLACEBO_COMPARATORParticipants will receive placebo matched to VX-840.
Part B: VX-840EXPERIMENTALParticipants will be randomized to receive multiple doses of one of different dose levels of VX-840. The dose levels will be determined based on the data from Part A.
Part B: PlaceboPLACEBO_COMPARATORParticipants will receive placebo matched to VX-840.
Interventions
NameTypeDescription
VX-840DRUGSolution for oral administration.
PlaceboDRUGPlacebo matched to VX-840 for oral administration.
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Eligibility Criteria
Age Range18 Years — 55 Years
SexALL
Healthy VolunteersYes
Study Sites1

Key Inclusion Criteria: * Female participants must be of non-childbearing potential * Body mass index (BMI) of 18.0 to 32.0 kilograms per meter square (kg/m\^2) * A total body weight greater than (\>) 50 kg Key Exclusion Criteria: * History of febrile illness or other acute illness within 14 days...

Countries:United States
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