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IONIS TMPRSS6-Lrx

Phase 1

Thalassemia | Small molecule | Hematology |Ionis Pharmaceuticals, Inc.|Last Updated: Jan 22, 2019

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 Enrollment36
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03165864Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 702843 Administered Subcutaneously to Healthy VolunteersPHASE1 COMPLETED 36May 9, 2017Sep 25, 2018Jan 22, 20191 Australia
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Study Endpoints
Primary Endpoints
Incidence and severity of adverse events that are related to treatment with IONIS TMPRSS6-Lrx
Up to 148 Days

The safety and tolerability of single and multiple doses of IONIS TMPRSS6-Lrx will be assessed by determining the incidence, severity, and dose relationship of adverse events that are related to treatment with IONIS TMPRSS6-Lrx

Secondary Endpoints
Pharmacokinetics after single and multiple doses of IONIS TMPRSS6-Lrx (maximum observed drug concentration or Cmax)
Up to 148 Days
Pharmacokinetics after single and multiple doses of IONIS TMPRSS6-Lrx (time taken to reach maximal concentration or Tmax)
Up to 148 Days
Pharmacokinetics after single and multiple doses of IONIS TMPRSS6-Lrx (Plasma terminal elimination half-life (t1/2λz)
Up to 148 Days
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Study Design & Arms
AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
IONIS TMPRSS6-LrxEXPERIMENTALAscending single and multiple doses of IONIS TMPRSS6-Lrx administered subcutaneously
PlaceboPLACEBO_COMPARATORSaline .9%
Interventions
NameTypeDescription
IONIS TMPRSS6-LrxDRUGAscending single and multiple doses of IONIS TMPRSS6-Lrx administered subcutaneously
PlaceboOTHERSaline .9%
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Eligibility Criteria
Age Range18 Years — 65 Years
SexALL
Healthy VolunteersYes
Study Sites1

Inclusion Criteria: * Must have given written informed consent and be able to comply with all study requirements * Healthy males or females aged 18-65 inclusive at the time of Informed Consent * Females must be non-pregnant and non-lactating, and either surgically sterile or post- menopausal * Male...

Countries:Australia
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