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VX-272

Phase 1

Cystic Fibrosis | Small molecule | Respiratory |Vertex Pharmaceuticals Incorporated|Trials Updated: Sep 23, 2026

VX-272 development status

Highest phase Phase 1
Registered trials 1 across 1 sponsor since Feb 2026

VX-272 target and mechanism

Molecular targetCFTR
Target classProtein
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials1
Total Enrollment160

FDA Designations

No designations recorded

VX-272 clinical trials

VX-272 · 1 trial · 1 indication

Phase 1 1
NCT07437105Dose Escalation Study Evaluating the Safety and Pharmacokinetics of VX-272 in Healthy ParticipantsCystic Fibrosis
RECRUITING160 Analytics
PHASE1RECRUITING
Dose Escalation Study Evaluating the Safety and Pharmacokinetics of VX-272 in Healthy Participants
Cystic FibrosisUnlock trial analytics

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 Signing of Informed Consent Form (ICF) up to Safety Follow Up (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 Signing of Informed Consent Form (ICF) up to Safety Follow Up (Up to Day 66)
Part C: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
From Signing of Informed Consent Form (ICF) up to Safety Follow Up (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.
Part C: VX-272 in Combination With Tezacaftor (TEZ)/ Deutivacaftor (D-IVA) or D-IVAEXPERIMENTALParticipants will be randomized to receive VX-272 in combination with TEZ/D-IVA or D-IVA.
Placebo: Part CPLACEBO_COMPARATORParticipants will be randomized to receive placebo matched to VX-272 in combination with TEZ/D-IVA or D-IVA.

Interventions

NameTypeDescription
VX-272DRUGSuspension for Oral Administration.
PlaceboDRUGSuspension for Oral Administration.
TezacaftorDRUGTablet for oral administration.
DeutivacaftorDRUGTablet for oral administration.
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Eligibility Criteria

Age Range18 Years to 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 20 trials

Recent Changes (Last 90 Days)

MEDIUMSep 23, 2026NCT07437105Enrollment: 128 → 160
MEDIUMSep 23, 2026NCT07437105Enrollment: 128 → 160

Frequently asked questions about VX-272

What is VX-272?

VX-272 is an investigational small molecule being developed by Vertex Pharmaceuticals for cystic fibrosis. It is currently in Phase 1 clinical development and is designed to target the CFTR protein. It has not been approved by the FDA and remains an experimental therapy under study.

What is VX-272 used for in cystic fibrosis?

VX-272 is being studied for the treatment of cystic fibrosis, a respiratory condition. Development is at the Phase 1 stage, so its use remains investigational. The clinical program is evaluating the drug in a dose escalation study rather than in an approved treatment setting.

What does VX-272 target?

VX-272 targets CFTR, the cystic fibrosis transmembrane conductance regulator protein. CFTR is the protein implicated in cystic fibrosis. By acting on this protein target, VX-272 is being developed as a small molecule therapeutic for the disease, though it remains in early clinical testing.

Who makes VX-272?

VX-272 is being developed by Vertex Pharmaceuticals Incorporated, which trades on the Nasdaq under the ticker VRTX. Vertex is the sponsor of the clinical program evaluating the drug in cystic fibrosis.

What phase is VX-272 in?

VX-272 is in Phase 1 clinical development. It is an investigational drug and has not been approved by the FDA. The Phase 1 program is ongoing, with one active trial and no completed trials reported for this asset.

What clinical trials is VX-272 in?

VX-272 is being evaluated in one active clinical trial, NCT07437105, titled a Dose Escalation Study Evaluating the Safety and Pharmacokinetics of VX-272 in Healthy Participants. The Phase 1 study is recruiting, is placebo controlled and double blind, and is expected to enroll 160 participants in the United States.