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ION224

Phase 2

Steatohepatitis, Nonalcoholic | Small molecule | Infectious Disease |Ionis Pharmaceuticals, Inc.|Last Updated: Nov 27, 2024

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 Enrollment160
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04932512A Study to Assess the Safety, Efficacy, and Pharmacokinetics of Multiple Doses of ION224PHASE2 COMPLETED 160 — — Jun 17, 2021Feb 28, 2024Nov 27, 202451 United States, Puerto Rico
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Study Endpoints
Primary Endpoints
Percentage of Participants With at Least 2-point Reduction in Non-alcoholic Fatty Liver Disease Activity Score (NAS) With at least 1-point Improvement in Hepatocellular Ballooning or Lobular Inflammation, and Without Worsening in Fibrosis Stage at EOT
Up to Week 49

The NAS is a histology grading score composed on the assessment of steatosis (scale 0-3), hepatocellular ballooning (scale 0-2), and lobular inflammation (scale 0-3), with higher scores indicating more severe hepatitis. Worsening of fibrosis is defined as an increase in fibrosis of at least one stage on the Kleiner fibrosis classification: fibrosis stages range from 0-4, with higher scores indicating greater fibrosis (0=None, 4=Cirrhosis).

Secondary Endpoints
Change From Baseline in Hepatic Fat Content Measurement as Evaluated by MRI-PDFF and Calculated by an Independent, Blinded-To-Treatment, Central Reader
Baseline up to Week 15, Week 29 and Week 49
Percentage of Participants Achieving Non-alcoholic Steatohepatitis (NASH) Resolution, as Defined by Scores of 0 for Ballooning and 0 or 1 for Inflammation by the NAS, and Without Worsening of Fibrosis, Assessed Through Liver Biopsy at the EOT
Up to Week 49
Percentage of Participants Achieving Reduction of at Least 1 Stage in the Fibrosis Score, and Without Worsening of Steatohepatitis by the NAS, Assessed Through Liver Biopsy at the EOT
Up to Week 49
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Study Design & Arms
AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ION224EXPERIMENTALMultiple doses of ION224 will be administered by SC injection once every 4 weeks for up to 49 weeks.
PlaceboPLACEBO_COMPARATORMultiple doses of matching placebo will be administered by SC injection once every 4 weeks for up to 49 weeks.
Interventions
NameTypeDescription
ION224DRUGION224 will be administered by SC injection.
PlaceboOTHERION224-matching placebo solution will be administered by SC injection.
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Eligibility Criteria
Age Range18 Years — 75 Years
SexALL
Healthy VolunteersNo
Study Sites51

Inclusion Criteria: * Males or females greater than or equal to (≥) 18 and less than or equal to (≤) 75 years old at the time of informed consent * Body mass index ≥ 25 kg/m\^2 and ≥ 22 kg/m\^2 for participants of Asian race, as assessed during screening * Liver fat ≥ 10% as assessed by MRI-PDFF be...

Countries:United StatesPuerto Rico
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