Hepatology Month in Review: January 2025

January was a busy month for the field of hepatology, which started 2025 with a series of breakthroughs signaling a year of innovation, expanding treatment options, and a deeper understanding of liver disease.

Among these breakthroughs was the US Food and Drug Administration granting Orphan Drug Designation to CNP-104 for primary biliary cholangitis (PBC) and Breakthrough Therapy Designation to brelovitug for chronic hepatitis delta. In the clinical trial space, findings from the phase 2b SYMMETRY study highlight efruxifermin’s ability to reverse compensated cirrhosis in patients with metabolic dysfunction-associated steatohepatitis (MASH). Paired with the plethora of research about metabolic dysfunction-associated steatotic liver disease (MASLD), liver transplantation, and alcohol in liver disease, January represented a strong start to 2025 for the field of hepatology.

Check out this January 2025 hepatology month in review for a recap of HCPLive’s coverage of the top hepatic news and research from the first few weeks of the year:

Hepatic Pipeline Updates

FDA Grants Orphan Drug Designation to CNP-104 for Primary Biliary Cholangitis

Starting the month off strong, in January 8, 2025, the FDA granted Orphan Drug Designation to CNP-104 for the treatment of PBC following the presentation of positive topline data from the phase 2a clinical trial of CNP-104 in PBC at the American Association for the Study of Liver Diseases’ The Liver Meeting in 2024.

“Receiving Orphan Drug Designation for CNP-104 underscores its potential to become the first disease-modifying treatment for individuals with PBC,” said Dannielle Appelhans, president and chief executive officer of COUR, in a press release. “Notably, in addition to demonstrating favorable T cell responses among treated participants, CNP-104 slowed disease progression, as evidenced by a statistically significant reduction in liver stiffness measured by FibroScan by day 120 of the study period.”

Brelovitug Receives FDA Breakthrough Therapy Designation for Chronic Hepatitis Delta

Later in the month, the FDA granted Breakthrough Therapy designation to Bluejay Therapeutics’ brelovitug (BJT-778) for the treatment of chronic hepatitis delta. The designation came just 2 months after phase 2 data demonstrating 100% virologic response and up to 78% combined virologic response and alanine aminotransferase normalization with brelovitug monotherapy were presented at The Liver Meeting 2024 of the American Association for the Study of Liver Diseases.

What’s New in MASLD

Efruxifermin Reverses MASH Compensated Cirrhosis in Phase 2b SYMMETRY Study

Preliminary topline week 96 results from the phase 2b SYMMETRY study of efruxifermin in patients with biopsy-confirmed compensated cirrhosis (F4), Child-Pugh Class A, due to MASH showed 39% of patients with baseline and week 96 biopsies who were treated with efruxifermin 50 mg experienced reversal of cirrhosis with no worsening of MASH, compared to 15% for placebo (P = .009). In the intent-to-treat population, 29% of patients in the efruxifermin 50 mg group experienced reversal of cirrhosis with no worsening of MASH, compared to approximately 12% in the placebo group (P = .031).

MASLD Prevalence Projected to Exceed 40% by 2050, Increasing Health System Burden

In this decision analytical modeling study, the prevalence of MASLD was estimated to reach 41.4%, equating to approximately 122 million people in the US, in 2050. Findings also called attention to stark increases in prevalent cases of decompensated cirrhosis, incident cases of liver cancer, and the need for liver transplantation.

Liver Transplant Research

Normothermic Machine Perfusion Improves Liver Transplant Outcomes, Lowers Resource Use

Findings from this study suggest normothermic machine perfusion (NMP) in liver transplantation is associated with better graft and patient survival, reduced early allograft dysfunction, and lower hospital resource use.

1/4 of Pediatric Liver Transplant Recipients Experience Early Hospital Readmission

Leveraging data from the Society of Pediatric Liver Transplantation (SPLIT) database, findings from this study suggest 23% of first-time pediatric liver transplant recipients are readmitted to the hospital within 30 days of discharge after transplant hospitalization. Shorter hospital stays following transplantation, insurance status, and the recipient's history of diabetes or malignancy were identified as factors associated with early hospital readmission.

Alcohol and Liver Disease

Alcohol-Associated Liver Disease, MASLD Drive Growing Liver Cancer Burden in the US

This study highlights a 142% increase in prevalence, a 141% increase in incident cases, and a 136% increase in deaths attributable to primary liver cancer in the US between 2000 and 2021. While hepatitis C virus-associated primary liver cancer accounted for the greatest prevalence, alcohol-associated liver disease and MASLD were the fastest-growing etiologies.

Young Women Face Worse Long-Term Alcohol-Associated Hepatitis Outcomes Than Men

Findings from this population-based study of more than 3000 patients 13-39 years of age in Canada suggest rapid increases in the rates of alcohol-associated hepatitis (AH) in this patient population over the past 2 decades, especially among women. Despite numerically more males being affected by AH, females had greater rates of liver-related mortality and were at an approximately 50% increased risk of developing cirrhosis.

Related: Jennifer Flemming, MD: Understanding the Shifting Burden of Alcohol-Associated Hepatitis