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VX-809

Phase 3

Cystic Fibrosis | Small molecule | Respiratory |Vertex Pharmaceuticals Incorporated|Last Updated: Oct 23, 2017

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_CONTROLLEDDMCBiomarker
Total Trials4
Total Enrollment371
FDA Designations
No designations recorded
Clinical Trials (4)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02514473A Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects With CF, Homozygous for the F508del-CFTR MutationPHASE3 COMPLETED 206Jul 1, 2015Sep 1, 2016Oct 23, 201753 United States, Australia +7
NCT00865904Study of VX-809 in Cystic Fibrosis Subjects With the ∆F508-CFTR Gene MutationPHASE2 COMPLETED 93Mar 1, 2009Dec 1, 2009Aug 28, 201525 United States, Belgium +3
NCT01216046Drug-Drug Interaction Study of VX-770 and VX-809 in Healthy SubjectsPHASE1 COMPLETED 48Oct 1, 2010May 1, 2011Jan 18, 20121 United States
NCT00966602Drug-Drug Interaction Study of VX-809 and VX-770 in Healthy SubjectsPHASE1 COMPLETED 24Sep 1, 2009Dec 1, 2009Jan 5, 20101 Netherlands
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Study Endpoints
Primary Endpoints
Absolute Change From Baseline in Lung Clearance Index 2.5 (LCI2.5) Through Week 24
Baseline, Through Week 24

Lung clearance index (LCI) is a measure of ventilation inhomogeneity that is derived from a multiple breath washout test using Nitrogen (N2). LCI2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value.

Safety and Tolerability Based on Adverse Events (AEs)
Up to 14 days after last dose (last dose = Day 28)

AE: any untoward medical occurrence in a participant during the study; the event does not necessarily have a causal relationship with the treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after the informed consent form is signed. AE includes serious as well as Non-serious AEs. Serious adverse event (SAE) (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, in-patient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. Number of participants with AEs and SAEs are reported. An AE that started at or after initial dosing of study drug, or increased in severity after initial dosing of study drug visit is considered treatment-emergent.

PK parameters (including concentration, exposure and half-life) of VX-809 and its metabolite in plasma in the presence and absence of VX 770
70 days

Blood samples drawn during the study will be analyzed to measure the PK parameters, such as concentration, exposure and half-life of VX-809 and its metabolite.

PK parameters (including concentration, exposure and half-life) of VX-770 and its metabolites in plasma in the presence and absence of VX 809
70 days

Blood samples drawn during the study will be analyzed to measure the PK parameters, such as concentration, exposure and half-life of VX-770 and its metabolites.

Pharmacokinetic (PK) parameters of VX-770 and its metabolites in plasma in the presence and absence of VX-809
70 days
PK parameters of VX-809 in plasma in the presence and absence of VX-770
70 days
Secondary Endpoints
Average Absolute Change From Baseline in Sweat Chloride at Day 15 and Week 4
Baseline, Day 15 and Week 4
Absolute Change From Baseline in Body Mass Index (BMI) at Week 24
Baseline, Week 24
Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 24
Baseline, Through Week 24
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
LUM/IVAEXPERIMENTALFixed-dose combination with lumacaftor (LUM) 200 mg every 12 hours (q12h)/ ivacaftor (IVA) 250 mg q12h
PlaceboPLACEBO_COMPARATORMatching placebo q12h
VX-809, 25 mgEXPERIMENTALVX-809, 25 milligram (mg) capsule orally once daily for 28 days.
VX-809, 50 mgEXPERIMENTALVX-809, 50 mg capsule orally once daily for 28 days.
VX-809, 100 mgEXPERIMENTALVX-809, 100 mg capsule orally once daily for 28 days.
VX-809, 200 mgEXPERIMENTALVX-809, 200 mg capsule orally once daily for 28 days.
Treatment ArmEXPERIMENTALSubjects randomized to study drug will take VX-809 for 14 days followed by a 14 day washout. Next subjects will take VX-770 for 14 days followed by a 14 day washout. Lastly, subjects will take both VX-809 and VX-770 for 14 days.
Placebo ArmPLACEBO_COMPARATORSubjects randomized to placebo will take VX-809 placebo for 14 days followed by a 14 day washout. Next subjects will take VX-770 placebo for 14 days followed by a 14 day washout. Lastly, subjects will take both VX-809 and VX-770 placebo for 14 days.
Treatment Period 1EXPERIMENTAL -
Treatment Period 2EXPERIMENTAL -
Treatment Period 3EXPERIMENTAL -
Interventions
NameTypeDescription
VX-809DRUG -
PlaceboDRUG -
VX-770DRUG -
VX-809 placeboDRUGcapsule, taken once daily
VX-770 placeboDRUGtablet, taken once every 12 hours
VX-809 & VX-770DRUGVX-809 capsule, once daily, and VX-770 tablets, once every 12 hours, for 14 days
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Eligibility Criteria
Age Range6 Years — 11 Years
SexALL
Healthy VolunteersNo
Study Sites53

Inclusion Criteria: * Subjects who weigh ≥15 kg without shoes a the Screening Visit * Subjects with confirmed diagnosis of CF at the Screening Visit. * Subjects who are homozygous for the F508del CFTR mutation * Subjects with ppFEV1 of ≥70 percentage points adjusted for age, sex, and height * Subje...

Countries:United StatesAustraliaBelgiumCanadaDenmarkFranceGermanySwedenUnited KingdomNetherlands
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