New investigational treatment interrupts disease at its source, offering hope for millions with life-threatening liver condition linked to obesity and diabetes.
Scientists at UC San Diego have identified a new drug, ION224, that could transform the treatment of MASH, a dangerous form of fatty liver disease tied to obesity and diabetes. By blocking a key liver enzyme, the drug reduces fat and inflammation, halting the root causes of liver damage. In a year-long clinical trial, patients showed major improvements without serious side effects, offering hope to millions affected worldwide.
Researchers at University of California San Diego School of Medicine have identified a new investigational drug that shows promise in treating metabolic dysfunction-associated steatohepatitis (MASH), a serious form of fatty liver disease linked to obesity and type 2 diabetes that can lead to cirrhosis, liver failure, and even liver cancer.
They found that the medication, ION224, targets a liver enzyme called DGAT2, which plays a key role in how the liver produces and stores fat. By blocking this enzyme, the drug helps reduce fat buildup and inflammation, two major drivers of liver damage in MASH.
MASH, formally known as nonalcoholic steatohepatitis (NASH), affects people with metabolic conditions like obesity and type 2 diabetes. It is often called a "silent" disease because it can progress for years without symptoms.
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Ionis Pharmaceuticals, Inc. announced positive results from a Phase 2 study of ION224, an investigational DGAT2 antisense inhibitor in development for the treatment of metabolic dysfunction-associated steatohepatitis (MASH), previously referred to as nonalcoholic steatohepatitis (NASH). The study met its primary endpoint at both doses (120 mg and 90 mg), achieving liver histologic improvement, and also met the important secondary endpoint of MASH resolution.
Reducing the production of DGAT2 enzyme decreases the overproduction of triglycerides that contribute to excess liver fat, which can result in liver damage and inflammation. We are encouraged by these ION224 data, showing that a monthly subcutaneous medicine targeting DGAT2 has the potential to improve MASH and prevent its progression to more severe stages, including advanced liver fibrosis and cirrhosis.
ION224 is an investigational LIgand-Conjugated Antisense (LICA) medicine designed to reduce the production of diacylglycerol acyltransferase 2 (DGAT2) to treat patients with MASH. DGAT2 is an enzyme that catalyzes the final step in triglyceride synthesis in the liver. Reducing the production of DGAT2 should therefore decrease triglyceride synthesis in the liver. Additionally, there is evidence of an increase in both fatty acid oxidation and oxidative gene expression associated with antisense inhibition of DGAT2. ION224 offers a unique approach, which is potentially complementary to other therapies currently in clinical development.
NOTE: In 2023, several multinational liver societies made the recommendation to update NAFLD to metabolic dysfunction-associated steatotic liver disease (MASLD) and to update non-alcoholic steatohepatitis (NASH) to metabolic dysfunction-associated steatohepatitis (MASH).
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