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IONIS-ENaCRx

Phase 1

Healthy Subjects | Small molecule | Respiratory |Ionis Pharmaceuticals, Inc.|Last Updated: Feb 4, 2021

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 Enrollment98
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03647228A Phase 1/2a Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Doses of IONIS-ENaCRx in Healthy Volunteers and Patients With Cystic FibrosisPHASE1 COMPLETED 98Dec 13, 2018Oct 13, 2020Feb 4, 20218 Germany, United Kingdom
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Study Endpoints
Primary Endpoints
Safety and Tolerability as Measured by the Number of Participants with at least one Treatment-Emergent Adverse Event
Up to 113 Days
Secondary Endpoints
Cmax: maximum observed drug concentration in plasma of IONIS-ENaCRx
Up to 113 Days
Tmax: time taken to reach maximal concentration in plasma of IONIS-ENaCRx
Up to 113 Days
AUCt: area under the plasma concentration-time curve from time zero to time t for IONIS-ENaCRx
Up to 113 Days
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Study Design & Arms
AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
IONIS-ENaCRxEXPERIMENTALAscending single and multiple doses of IONIS-ENaCRx inhaled or nebulized.
PlaceboPLACEBO_COMPARATORPlacebo comparator calculated volume to match active comparator inhaled or nebulized.
Interventions
NameTypeDescription
IONIS-ENaCRxDRUGAscending single and multiple doses of IONIS-ENaCRx inhaled or nebulized.
PlaceboDRUGPlacebo comparator calculated volume to match active comparator inhaled or nebulized.
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Eligibility Criteria
Age Range18 Years — 50 Years
SexALL
Healthy VolunteersYes
Study Sites8

Inclusion Criteria (Healthy Volunteers) 1. Females must be non-pregnant and non-lactating, and either surgically sterile or post-menopausal. 2. Males must be surgically sterile or, abstinent or, if engaged in sexual relations with a woman of child-bearing potential, the subject or the subject's non...

Countries:GermanyUnited Kingdom
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