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VX-264

Phase 1

Type 1 Diabetes | Small molecule | Metabolic |Vertex Pharmaceuticals Incorporated|Last Updated: Jun 4, 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
UNCONTROLLED
Total Trials1
Total Enrollment7
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05791201A Safety, Tolerability, and Efficacy Study of VX-264 in Participants With Type 1 DiabetesPHASE1 ACTIVE NOT_RECRUITING 7May 16, 2023Mar 12, 2027Jun 4, 202616 United States, Canada +5
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Study Endpoints
Primary Endpoints
Part A and 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 24 months
Part B: Change in Peak C-peptide during Mixed-Meal Tolerance Test (MMTT)
From Baseline and at Day 90
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
VX-264EXPERIMENTAL -
Interventions
NameTypeDescription
VX-264DRUGAllogeneic human stem cell-derived islets.
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Eligibility Criteria
Age Range18 Years — 65 Years
SexALL
Healthy VolunteersNo
Study Sites16

Key Inclusion Criteria: Clinical history of T1D with greater than or equal to (\>=) 5 years duration Participant is on a stable diabetic treatment Consistent use of continuous glucose monitoring (CGM) for at least 4 weeks before Screening and willingness to use CGM for the duration of the study Ke...

Countries:United StatesCanadaGermanyItalyNetherlandsSwitzerlandUnited Kingdom
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Recent Changes (Last 90 Days)
MEDIUMJun 4, 2026NCT05791201primaryCompletionDate: changed
MEDIUMJun 4, 2026NCT05791201primaryCompletionDate: changed
MEDIUMJun 4, 2026NCT05791201primaryCompletionDate: changed
MEDIUMJun 4, 2026NCT05791201primaryCompletionDate: changed
LOWMay 26, 2026NCT05791201primaryCompletionDate: changed
LOWMay 24, 2026NCT05791201studyFirstPostDate: changed