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VX-272

Phase 1

Cystic Fibrosis | Small molecule | Respiratory |Vertex Pharmaceuticals Incorporated|Last Updated: Mar 31, 2026

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 Enrollment128

FDA Designations

No designations recorded

Clinical trial landscape

VX-272 · 1 trial · 1 indication

Phase 1 1
NCT07437105Dose Escalation Study Evaluating the Safety and Pharmacokinetics of VX-272 in Healthy ParticipantsCystic Fibrosis
RECRUITING128 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 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 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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Frequently asked questions about VX-272

What is VX-272 used for in Cystic Fibrosis?

VX-272 is an investigational small molecule being developed for the treatment of Cystic Fibrosis. It is currently in Phase 1 clinical development and is not yet approved by regulatory authorities. The drug targets the CFTR protein, which is central to the disease.

What does VX-272 target?

VX-272 targets the CFTR protein, a channel involved in chloride transport across cell membranes. By modulating CFTR, the drug aims to address the underlying defect in Cystic Fibrosis. This target is a protein, and the drug is a small molecule designed to interact with it.

Who is developing VX-272?

VX-272 is being developed by Vertex Pharmaceuticals Incorporated, a biopharmaceutical company. Vertex is listed on the stock exchange under the ticker VRTX. The company is conducting clinical trials to evaluate the safety and pharmacokinetics of VX-272.

What phase is VX-272 in?

VX-272 is in Phase 1 clinical development. It is an investigational drug, meaning it has not been approved by the FDA or other regulatory bodies. The ongoing Phase 1 trial is designed to assess safety and pharmacokinetics in healthy participants.

What clinical trials is VX-272 in?

VX-272 is being studied in a Phase 1 trial with the identifier NCT07437105. This is a dose escalation study evaluating the safety and pharmacokinetics of VX-272 in healthy participants. The trial is recruiting and has an enrollment of 128 participants in the United States.

Is VX-272 the same as any other drug?

VX-272 is a distinct investigational compound developed by Vertex Pharmaceuticals. It is not known to be the same as any other marketed drug. Its unique mechanism targets the CFTR protein for the treatment of Cystic Fibrosis.