A groundbreaking new drug has emerged as the first targeted therapy to potentially halt the progression of a deadly liver disease that affects over 100 million Americans.

Story Highlights

• ION224 becomes first drug to successfully target DGAT2 enzyme, interrupting MASH at its root cause
• Phase IIb trial shows 60% improvement in liver health independent of weight loss
• Over 100 million Americans suffer from fatty liver disease with limited treatment options
• No serious adverse events reported, marking significant safety breakthrough

Revolutionary Enzyme-Targeting Breakthrough

UC San Diego researchers have achieved a medical milestone with ION224, the first drug to successfully target the DGAT2 enzyme responsible for liver fat synthesis. Published in The Lancet, the Phase IIb clinical trial demonstrated that this antisense oligonucleotide therapy significantly reduces liver fat, inflammation, and cellular damage in patients with metabolic dysfunction-associated steatohepatitis (MASH). Unlike previous failed attempts, ION224 works independently of weight loss, offering a direct therapeutic approach to combat this silent epidemic.

New drug could be first to stop deadly fatty liver disease https://t.co/Pk7BhPHioc

— alanfromtexas (@AAlanfromtexas) September 18, 2025

Clinical Trial Results Demonstrate Unprecedented Success

The multicenter trial involving 160 adults with MASH and fibrosis across 43 US sites revealed remarkable outcomes. Patients receiving the highest dose of ION224 showed a 60% improvement in liver health markers over one year. Dr. Rohit Loomba, principal investigator and chief of Gastroenterology at UC San Diego, emphasized that blocking DGAT2 interrupts the disease process at its fundamental level, potentially halting or reversing liver damage.

Addressing America’s Hidden Health Crisis

MASH represents a severe form of fatty liver disease closely linked to obesity and type 2 diabetes—conditions that have reached epidemic proportions under policies that ignored preventive healthcare priorities. This progressive disease can advance to cirrhosis, liver failure, and cancer, yet often remains undiagnosed until critical stages. The lack of effective treatments has left millions vulnerable to a condition that significantly impacts productivity and strains healthcare resources through costly emergency interventions.

Path Forward and Regulatory Implications

With Phase IIb trials complete and showing no serious adverse events, ION224 advances to Phase III testing—the final hurdle before potential FDA approval. This development represents validation of private-sector innovation and academic collaboration in addressing real health challenges. The drug’s success could transform standard care for MASH patients while potentially reducing long-term healthcare costs associated with advanced liver disease complications.

The breakthrough offers hope for effective disease management through targeted intervention rather than costly reactive treatments, aligning with principles of efficient healthcare resource allocation and personal health responsibility.

Sources:

GB News – Fatty Liver Disease New Drug
Modern Sciences – ION224 MASH Liver Disease
UC San Diego Health News – First Enzyme-Targeting Drug Reverses Liver Damage in MASH
Tribune India – New Drug Could Be First to Stop Deadly Fatty Liver Disease
Science Daily – New Drug Could Be First to Stop Deadly Fatty Liver Disease