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IONIS-STAT3Rx

Phase 1

Advanced Cancers | Small molecule | Oncology |Ionis Pharmaceuticals, Inc.|Last Updated: Jun 25, 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
UNCONTROLLED
Total Trials1
Total Enrollment64
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01563302Phase 1/2, Open-label, Dose-escalation Study of IONIS-STAT3Rx, Administered to Patients With Advanced CancersPHASE1 COMPLETED 64Feb 27, 2012Mar 23, 2016Jun 25, 20189 United States
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Study Endpoints
Primary Endpoints
Safety of IONIS-STAT3Rx in patients with Advanced Cancers
Approximately 28 days after last dose of IONIS -STAT3Rx
Maximum-tolerated dose (MTD) of IONIS-STAT3Rx in patients with Advanced Cancers.
Approximately 28 Days

Highest dose level at which no more than 1 out of 6 patients develops a DLT

Secondary Endpoints
Clinical activity of IONIS-STAT3Rx
Approximately 28 Days after last dose of IONIS-STAT3Rx
Pharmacokinetics-Cmax
Approximately 28 days after last dose of IONIS-STAT3Rx
STAT3 and other biomarkers
Approximately 28 days after last dose of IONIS-STAT3Rx
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Group 1EXPERIMENTALIONIS-STAT3Rx
Interventions
NameTypeDescription
IONIS-STAT3RxDRUGThree-hour IV infusions on Cycle 0 Days 1, 3, 5, and weekly three-hour IV infusions in Cycles 1 and beyond, on Days 1, 8, and 15 of each cycle.
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites9

Inclusion Criteria: * Aged 18 years or older * Tumors that are relapsed or refractory to at least 1 prior anti-cancer systemic therapy and for which no standard therapy exists * Expansion cohort only: Advanced lymphoma confirmed by histopathology * Measurable or evaluable disease according to RECIS...

Countries:United States
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