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VX-272

Phase 1

Cystic Fibrosis | Small molecule | Respiratory |Vertex Pharmaceuticals Incorporated|Last Updated: Mar 31, 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 Enrollment128
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT07437105Dose Escalation Study Evaluating the Safety and Pharmacokinetics of VX-272 in Healthy ParticipantsPHASE1 RECRUITING 128 — — Feb 25, 2026Mar 23, 2027Mar 31, 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 34
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 66
Secondary Endpoints
Part A: Maximum Observed Plasma Concentration (Cmax) of VX-272
From Day 1 up to Day 34
Part A: Area Under the Concentration Versus Time Curve From the Time of Dosing Up to 24 Hours (AUC0-24h) of VX-272
From Day 1 up to Day 34
Part B: Maximum Observed Plasma Concentration (Cmax) of VX-272
From Day 1 up to Day 66
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Part A: Single Ascending DoseEXPERIMENTALParticipants will be randomized to receive a single dose of VX-272.
Placebo: Part APLACEBO_COMPARATORParticipants will be randomized to receive a single dose of placebo matched to VX-272.
Part B: Multiple Ascending DoseEXPERIMENTALParticipants will be randomized to receive multiple doses of VX-272.
Placebo: Part BPLACEBO_COMPARATORParticipants will be randomized to receive multiple doses of placebo matched to VX-272.
Interventions
NameTypeDescription
VX-272DRUGSuspension for Oral Administration.
PlaceboDRUGSuspension 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 32.0 kilogram per meter square (kg/m\^2) * A total body weight of more than (\>) 50 kg * Male and Female participants of non-childbearing potential Key Exclusion Criteria: * History of febrile illness or other acute illness that has not f...

Countries:United States
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Competitive Landscape -Cystic Fibrosis 18 trials
CompanyTickerTrialsLead PhaseDrugs
Vertex Pharmaceuticals IncorporatedVRTX9PHASE3VX-121/TEZ/D-IVA, ELX/TEZ/IVA, IVA, VNZ/TEZ/D-IVA, VX-522 mRNA therapy
Sionna Therapeutics, Inc.SION2PHASE2SION-719, SION-451, SION-2222, SION-109
BiomX LtdPHGE1PHASE2BX004
4D Molecular Therapeutics, Inc.FDMT1PHASE24D-710
Arcturus Therapeutics Holdings, Inc.ARCT1PHASE2ARCT-032
Krystal Biotech, Inc.KRYS1PHASE1KB407
Illumina, Inc.ILMN1 — Undisclosed
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT07437105Status: NOT_YET_RECRUITING → RECRUITING
LOWMay 24, 2026NCT07437105studyFirstPostDate: changed