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ION547

Phase 1

Healthy Volunteers | Small molecule | Other |Ionis Pharmaceuticals, Inc.|Last Updated: Sep 23, 2022

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_CONTROLLEDBiomarker
Total Trials1
Total Enrollment48
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04934891A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Doses of ION547PHASE1 COMPLETED 48 — — Mar 24, 2021Sep 14, 2022Sep 23, 20221 Canada
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Study Endpoints
Primary Endpoints
Number of Participants With at Least One Treatment-emergent Adverse Event (TEAE)
Up to Day 232
Cmax: Maximum Observed Plasma Concentration of ION547
Up to Day 232
Tmax: Time Taken to Reach Maximal Plasma Concentration of ION547
Up to Day 232
AUCt: Area Under the Plasma Concentration-time Curve From Time Zero to Time t for ION547
Up to Day 232
Urine 0-24 Hour (hr) Excretion of ION547
Up to 24 hours
Change from Baseline in FXII Antigen Levels
Up to Day 232
Change from Baseline in FXII Activity Levels
Up to Day 232
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Study Design & Arms
AllocationRANDOMIZED
MaskingDOUBLE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ION547EXPERIMENTALAscending single multiple doses of ION547 will be administered by SC injection.
PlaceboPLACEBO_COMPARATORAscending single multiple doses of ION547-matching placebo will be administered by SC injection.
Interventions
NameTypeDescription
ION547DRUGION547 will be administered by SC injection.
PlaceboDRUGION547-matching placebo will be administered by SC injection.
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Eligibility Criteria
Age Range18 Years — 65 Years
SexALL
Healthy VolunteersYes
Study Sites1

Inclusion Criteria: 1. Must have given written informed consent (signed and dated) and any authorizations required by local law and be able to comply with all study requirements. 2. Healthy males or females 18 to 65, inclusive, at the time of informed consent. 3. Satisfy the following: a. Femal...

Countries:Canada
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