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VX-509

Phase 2

Rheumatoid Arthritis | Small molecule | Immunology |Vertex Pharmaceuticals Incorporated|Last Updated: Nov 20, 2015

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 Enrollment647
FDA Designations
No designations recorded
Clinical Trials (4)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01830985A Phase 2/3 Open-label Extension Study to Evaluate Long-Term Safety and Efficacy With VX-509 in Subjects With Rheumatoid ArthritisPHASE2 COMPLETED 39 — — Apr 1, 2013Jul 1, 2014Nov 20, 201522 United States, Estonia +2
NCT01754935A Magnetic Resonance Imaging Study and Arthroscopic Biopsy Substudy in Subjects With Active Rheumatoid Arthritis Receiving VX-509, an Oral JAK3 InhibitorPHASE2 COMPLETED 43 — — Jan 1, 2013Feb 1, 2014May 19, 201530 United States, Denmark +4
NCT0159045924-week Study With Open Label Extension of VX-509, an Oral JAK3 Inhibitor, in Subjects Taking MethotrexatePHASE2 COMPLETED 359 — — Apr 1, 2012Jul 1, 2014Nov 20, 201594 United States, Argentina +12
NCT01052194A 12-week Study of 4 Doses of VX-509 in Subjects With Active Rheumatoid ArthritisPHASE2 COMPLETED 206 — — Feb 1, 2010Jul 1, 2011Dec 13, 201248 United States, Belgium +8
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Study Endpoints
Primary Endpoints
Long-term safety and tolerability of VX-509 treatment
Baseline through 104 weeks

Measured by clinical laboratory tests

Proportion of subjects achieving a ≥20% improvement in disease severity according to the American College of Rheumatology criteria (ACR20), using C reactive protein (CRP) (ACR20 CRP)
Week 12
Change from baseline in Disease Activity Score 28 using CRP (4-component) (DAS28-4[CRP])
Week 12
Change from baseline in OMERACT RAMRIS synovitis score in designated hand wrist
Week 12
Change from baseline in OMERACT RAMRIS bone marrow edema (osteitis) in designated hand wrist
Week 12
Change from baseline in OMERACT RAMRIS erosion score in designated hand wrist
Week 12
Proportion of subjects who achieve a 20% improvement in disease severity according to the American College of Rheumatology criteria, assessed using the C-reactive protein level (ACR20-CRP) response
Week 12
Change from baseline in Disease Activity Score 28 using C-reactive protein (DAS28- CRP)
Week 12
Safety and tolerability
Week 12

Measured by incidence of treatment-emergent adverse events

Proportion of subjects who achieve an ACR20 response
Week 12
Change from baseline in DAS28
Week 12
Secondary Endpoints
Proportion of subjects who achieve CDAI LDA (≤10) or CDAI remission (≤2.8)
Baseline through 104 weeks
Proportion of subjects who achieve ≥20% (50%, 70%) improvement in disease severity according to the ACR criteria, using CRP (ACR20 CRP, ACR50 CRP, ACR70 CRP)
Baseline through 104 weeks
Change from baseline in DAS28 using CRP (4-component) (DAS28 4[CRP])
Baseline through 104 weeks
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Single Arm VX-509EXPERIMENTAL -
VX-509 100 mg qd ArmEXPERIMENTAL -
VX-509 200 mg qd ArmEXPERIMENTAL -
VX-509 300 mg qd ArmEXPERIMENTAL -
Placebo ArmPLACEBO_COMPARATOR -
VX-509 150 mg qd ArmEXPERIMENTAL -
VX-509 100 mg bid ArmEXPERIMENTAL -
25 mg b.i.d. VX-509EXPERIMENTAL -
50 mg b.i.d. VX-509EXPERIMENTAL -
100 mg b.i.d. VX-509EXPERIMENTAL -
150 mg b.i.d. VX-509EXPERIMENTAL -
PlaceboPLACEBO_COMPARATOR -
Interventions
NameTypeDescription
VX-509DRUGVX-509 dose may be increased every 8 weeks in a stepwise fashion from 100 to 150 mg and from 150 to 200 mg, as needed (determined by ongoing disease activity by CDAI)
VX-509 matching placeboDRUG0 mg oral tablet
PlaceboDRUGtablet, placebo b.i.d. for 12 weeks
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Eligibility Criteria
Age Range18 Years — 65 Years
SexALL
Healthy VolunteersNo
Study Sites22

Inclusion Criteria: * Subjects must have completed the assigned study drug treatment phase of a previous VX-509 study (e.g., Study 103). * Subjects must voluntarily sign and date the Study 104 informed consent document. * Subject must be willing and able to comply with the scheduled visits, treatme...

Countries:United StatesEstoniaLithuaniaSouth AfricaDenmarkNetherlandsArgentinaBulgariaCzechiaGermanyHungaryMexicoPolandRomaniaRussiaSerbiaSlovakiaUkraineBelgiumCroatiaPuerto Rico
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Competitive Landscape -Rheumatoid Arthritis 97 trials
CompanyTickerTrialsLead PhaseDrugs
AbbVie, Inc.ABBV6PHASE3Upadacitinib, Adalimumab, Tocilizumab, Lutikizumab, Ravagalimab
Eli Lilly and CompanyLLY5PHASE3Baricitinib, Tocilizumab, LY3541860, LY4213663, LY4298445
Bristol-Myers Squibb CompanyBMY6PHASE3Abatacept, Adalimumab, Methotrexate, BMS-986528, CC-97540
Johnson & JohnsonJNJ2PHASE3Ustekinumab, Guselkumab
AstraZeneca PLCAZN4PHASE2AZD1163, AZD0120, AZD5492, Surovatamig
Novartis AG Sponsored ADRNVS4PHASE2VAY736 PFS, VAY736 AI, PIT565, Secukinumab
Merck & Co., Inc.MRK2PHASE2Tulisokibart, Methotrexate, MK-1045
Sanofi SA Sponsored ADRSNY3PHASE2Sarilumab SAR153191, SAR448501, Leflunomide
AnaptysBio, Inc.ANAB1PHASE2Rosnilimab
Spyre Therapeutics, IncSYRE1PHASE2SPY002-072
Immunovant IncIMVT1PHASE2IMVT-1402
XBiotech, Inc.XBIT1PHASE2Natrunix, Methotrexate
Amgen Inc.AMGN1PHASE2Inebilizumab, Blinatumomab
Artiva Biotherapeutics, Inc.ARTV2PHASE2Allogeneic NK Cells, AB-101, Rituximab, Cyclophosphamide, Fludarabine
Gilead Sciences, Inc.GILD1PHASE1GS-0151
Zenas BioPharma, Inc.ZBIO1PHASE1ZB002
Stryker CorporationSYK5NAUndisclosed
Sonoma Pharmaceuticals, Inc.SNOA2PHASE1SBT777101
Zimmer Biomet Holdings, Inc.ZBH29NAUndisclosed
Adicet Bio IncACET1PHASE1ADI-001, Fludarabine, Cyclophosphamide
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