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VX-522

Phase 1

Cystic Fibrosis | Small molecule | Respiratory |Vertex Pharmaceuticals Incorporated|Last Updated: Sep 8, 2026

Target and mechanism

ModalitySmall molecule

Also known as VX-522 mRNA therapy

Success Probability

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Market & Valuation

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Trial Design

CONTROLLED
Total Trials1
Total Enrollment26

FDA Designations

No designations recorded

Clinical trial landscape

VX-522 · 1 trial · 1 indication

Phase 1 1
NCT05668741A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF)Cystic Fibrosis
ACTIVE NOT_RECRUITING26 Analytics
PHASE1ACTIVE NOT_RECRUITING
A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF)
Cystic Fibrosis
NCT0566874126
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Study Endpoints

Primary Endpoints

Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
From Day 1 Through Week 8 [SAD and MAD]

Secondary Endpoints

MAD: Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
From Baseline at Day 29
MAD: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
From Day 1 Through Safety Follow-up Visit [up to Week 28]
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Single Ascending Dose (SAD)EXPERIMENTALParticipants grouped into different cohorts will receive a single ascending dose of VX-522.
Multiple Ascending Dose (MAD) Cohort 1: VX-522EXPERIMENTALParticipants will receive multiple ascending doses of VX-522.
MAD Cohort 1: VX-522+ IVAEXPERIMENTALFollowing run-in period with ivacaftor (IVA), participants will receive multiple ascending doses of VX-522 with IVA.
MAD Cohort 2: VX-522+ IVAEXPERIMENTALFollowing run-in period with ivacaftor (IVA), participants will receive multiple ascending doses of VX-522 with IVA.

Interventions

NameTypeDescription
VX-522 mRNA therapyDRUGOral inhalation using nebulizer.
IVADRUGTablet for oral administration.
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Eligibility Criteria

Age Range18 Years to 65 Years
SexALL
Healthy VolunteersNo
Study Sites26

Key Inclusion Criteria: * Body mass index is less than (\<) 30.0 kilograms per meter square (kg/m\^2) * A total body weight greater than (\>) 50 kg * Stable CF disease * CFTR gene mutations on both alleles that are not responsive to CFTR modulator therapy o Example mutations include but are not ...

Countries:United StatesAustraliaBelgiumCanadaGermanySwedenUnited Kingdom
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Competitive Landscape -Cystic Fibrosis 15 trials

CompanyTickerTrialsLead PhaseDrugs
Vertex Pharmaceuticals IncorporatedVRTX8PHASE3VX-121/TEZ/D-IVA
BiomX Inc.PHGE1PHASE2BX004
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)

LOWSep 8, 2026NCT05668741lastUpdatePostDate: changed
LOWSep 8, 2026NCT05668741lastUpdatePostDate: changed