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ION224

Phase 2

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

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials1
Total Enrollment160

FDA Designations

No designations recorded

Clinical trial landscape

ION224 · 1 trial · 1 indication

Phase 2 1
NCT04932512A Study to Assess the Safety, Efficacy, and Pharmacokinetics of Multiple Doses of ION224Steatohepatitis, Nonalcoholic
COMPLETED160 Analytics
PHASE2COMPLETED
A Study to Assess the Safety, Efficacy, and Pharmacokinetics of Multiple Doses of ION224
Steatohepatitis, NonalcoholicUnlock trial analytics

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 to 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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Frequently asked questions about ION224

What is ION224 used for?

ION224 is an investigational small molecule being developed for the treatment of nonalcoholic steatohepatitis (NASH), a form of fatty liver disease. It is currently in Phase 2 clinical development and is not yet approved by the FDA.

Who makes ION224?

ION224 is being developed by Ionis Pharmaceuticals, Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol IONS. The company is conducting clinical trials to evaluate the safety and efficacy of this investigational drug.

What phase is ION224 in?

ION224 is in Phase 2 clinical development. A Phase 2 study has been completed, but the drug remains investigational and has not received regulatory approval. Further clinical trials may be needed to establish its safety and efficacy.

What clinical trials is ION224 in?

ION224 has been studied in a completed Phase 2 clinical trial identified as NCT04932512. This randomized, double-blind, placebo-controlled study enrolled 160 participants with nonalcoholic steatohepatitis at sites in the United States and Puerto Rico.

Is ION224 FDA approved?

ION224 is not FDA approved. It is an investigational drug currently in Phase 2 clinical development. The completed Phase 2 trial assessed its safety, efficacy, and pharmacokinetics, but the drug has not yet been submitted for regulatory approval.