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IONIS-PKKRx

Phase 2

Chronic Migraine | Small molecule | Neurology |Ionis Pharmaceuticals, Inc.|Last Updated: Feb 12, 2020

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 Enrollment30
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03108469Efficacy and Safety of IONIS-PKKRx for Preventive Treatment of Chronic MigrainePHASE2 COMPLETED 30Aug 1, 2017Apr 26, 2019Feb 12, 20202 United States
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Study Endpoints
Primary Endpoints
Migraine Days
Comparing baseline to treatment period, up to 16 weeks

Compare the efficacy of ISIS 546254 in the preventive treatment of chronic migraine, measured by the number of monthly migraine days comparing baseline to the final month of the 4-month treatment period for subjects treated with ISIS 546254 vs. placebo.

Secondary Endpoints
Headache Severity
Comparing baseline to treatment period, up to 16 weeks
Headache Days
Comparing baseline to treatment period, up to 16 weeks
Number of Subjects Reporting a ≥ 50% Reduction in the Number of Migraine Headaches
Comparing baseline to treatment period, up to 16 weeks
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Study Design & Arms
AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
IONIS-PKKRx (ISIS 546254)ACTIVE_COMPARATORThose randomized to IONIS-PKKRx (ISIS 546254) will receive subcutaneous injections containing 1.00 mL (200mg) weekly for weeks 1-16.
PlaceboPLACEBO_COMPARATORThose randomized to placebo will receive subcutaneous injections containing 1.00 mL (200mg) weekly for weeks 1-16.
Interventions
NameTypeDescription
IONIS-PKKRx (ISIS 546254)DRUGThose randomized to IONIS-PKKRx (ISIS 546254) will receive subcutaneous injections containing 1.00 mL (200mg) of IONIS- PKKRx (ISIS 546254) weekly for weeks 1-16.
PlaceboOTHERThose randomized to placebo will receive subcutaneous injections 1.00 mL (200mg) weekly for weeks 1-16.
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Eligibility Criteria
Age Range18 Years — 65 Years
SexALL
Healthy VolunteersNo
Study Sites2

Inclusion Criteria: Potential subjects must meet the following criteria at the screening visit to enter this study: 1. male or female, in otherwise good health, 18 to 65 years of age. 2. history of chronic migraine meeting the diagnostic criteria listed in the International Classification of Heada...

Countries:United States
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