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VX-522 mRNA therapy

Phase 1

Cystic Fibrosis | Small molecule | Respiratory |Vertex Pharmaceuticals Incorporated|Last Updated: May 22, 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
CONTROLLEDBiomarker
Total Trials1
Total Enrollment26
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05668741A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF)PHASE1 ACTIVE NOT_RECRUITING 26 — — Feb 27, 2023Sep 9, 2026May 22, 202626 United States, Australia +5
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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 — 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 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)
HIGHMay 26, 2026NCT05668741Status: RECRUITING → ACTIVE_NOT_RECRUITING
LOWMay 24, 2026NCT05668741studyFirstPostDate: changed