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VX-440

Phase 1

Cystic Fibrosis | Small molecule | Respiratory |Vertex Pharmaceuticals Incorporated|Last Updated: Apr 3, 2018

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
RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials1
Total Enrollment16
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03486236A Study Evaluating Safety and Pharmacokinetics of VX-440 in Combination With Tezacaftor/Ivacaftor in Healthy Adult SubjectsPHASE1 COMPLETED 16Jul 20, 2016Sep 14, 2016Apr 3, 20181 United Kingdom
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Study Endpoints
Primary Endpoints
Safety and tolerability were based on the number and assessment of adverse events (AEs) and serious adverse events (SAEs)
from baseline through safety follow-up visit (up to 29 days)
Secondary Endpoints
Maximum observed concentration (Cmax) of VX-440, TEZ and its metabolites (M1-TEZ and M2-TEZ), and IVA and its metabolites (M1-IVA and M6-IVA)
from Day 1 through Day 18
Area under the concentration versus time curve during a dosing interval (AUCtau) of VX-440, TEZ and its metabolites (M1-TEZ and M2-TEZ), and IVA and its metabolites (M1-IVA and M6-IVA)
from Day 1 through Day 18
Observed pre-dose concentration (Ctrough) of VX-440, TEZ and its metabolites (M1-TEZ and M2-TEZ), and IVA and its metabolites (M1-IVA and M6-IVA)
from Day 1 through Day 18
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Cohort C1EXPERIMENTAL -
Cohort C1: Triple PlaceboPLACEBO_COMPARATOR -
Cohort C2EXPERIMENTAL -
Cohort C2: Triple PlaceboPLACEBO_COMPARATOR -
Interventions
NameTypeDescription
VX-440DRUGVX-440 was administered in TC with TEZ and IVA.
TEZDRUGTEZ was administered as part of a fixed-dose combination (FDC) tablet (TEZ/IVA)
IVADRUGIVA was administered as part of a FDC tablet (TEZ/IVA) and as a mono tablet
Matched PlacebosDRUGPlacebos matched to VX-440, TEZ, and IVA.
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Eligibility Criteria
Age Range18 Years — 55 Years
SexALL
Healthy VolunteersYes
Study Sites1

Inclusion Criteria: * Female subjects of non-childbearing potential only. * Body mass index (BMI) of 18.0 to 31.0 kg/m2, inclusive, and a total body weight \>50 kg. * Normal pulmonary function measurements, defined as forced expiratory volume in 1 second (FEV1) and forced vital capacity (FVC) both ...

Countries:United Kingdom
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