Approval Probability
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Adjusted LOA
ML Risk
ION224 · 1 trial · 1 indication
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).
| Arm | Type | Description |
|---|---|---|
| ION224 | EXPERIMENTAL | Multiple doses of ION224 will be administered by SC injection once every 4 weeks for up to 49 weeks. |
| Placebo | PLACEBO_COMPARATOR | Multiple doses of matching placebo will be administered by SC injection once every 4 weeks for up to 49 weeks. |
| Name | Type | Description |
|---|---|---|
| ION224 | DRUG | ION224 will be administered by SC injection. |
| Placebo | OTHER | ION224-matching placebo solution will be administered by SC injection. |
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...
ION224 is being developed for the treatment of nonalcoholic steatohepatitis, a form of fatty liver disease. It is an investigational medicine and has not been approved by the FDA. Ionis Pharmaceuticals is studying it in a Phase 2 clinical program in adults with this condition.
ION224 targets DGAT2, an enzyme involved in triglyceride synthesis. By acting on DGAT2, the drug is designed to reduce fat accumulation in the liver. DGAT2 is the molecular target of ION224, and the compound is a small molecule.
ION224 is being developed by Ionis Pharmaceuticals, Inc., which trades on the Nasdaq under the ticker IONS. Ionis is the sponsor of the clinical program evaluating ION224 in nonalcoholic steatohepatitis.
ION224 is in Phase 2 clinical development. It is an investigational drug and is not FDA approved. The Phase 2 study has been completed, and no active trials of ION224 are currently listed.
ION224 has been evaluated in the Phase 2 trial NCT04932512, titled A Study to Assess the Safety, Efficacy, and Pharmacokinetics of Multiple Doses of ION224. The trial enrolled 160 adults with nonalcoholic steatohepatitis in the United States and Puerto Rico and has been completed.
The Phase 2 trial NCT04932512 was a randomized, double-blind, placebo-controlled study of multiple doses of ION224. It enrolled 160 participants aged 18 years and older with nonalcoholic steatohepatitis. The study was conducted in the United States and Puerto Rico and has been completed.