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Title: A randomized, double-blind, placebo-controlled trial of Aldafermin in patients with nonalcoholic steatohepatitis and compensated cirrhosis.
Austin Authors: Rinella, Mary E;Lieu, Hsiao D;Kowdley, Kris V;Goodman, Zachary D;Alkhouri, Naim;Lawitz, Eric;Ratziu, Vlad;Abdelmalek, Manal F;Wong, Vincent Wai-Sun;Younes, Ziad H;Sheikh, Aasim M;Brannan, Donald;Freilich, Bradley;Membreno, Fernando;Sinclair, Marie ;Melchor-Khan, Liza;Sanyal, Arun J;Ling, Lei;Harrison, Stephen A
Affiliation: University of Chicago Pritzker School of Medicine, Chicago, IL, United States.
NGM Biopharmaceuticals, South San Francisco, CA, United States.
Washington State University, Spokane, WA, United States.;Liver Institute Northwest, Seattle, WA, United States.
Inova Health System, Falls Church, VA, United States.
Arizona Liver Health, Tucson, AZ, United States.
Texas Liver Institute, University of Texas Health San Antonio, San Antonio, TX, United States.
INSERM UMRS 1138 CRC, Paris, France.
Mayo Clinic, Rochester, MN, United States.
The Chinese University of Hong Kong, Hong Kong, China.
Gastro One, Germantown, TN, United States.
GI Specialists of Georgia, Atlanta, GA, United States.
Gastrointestinal Associates, Flowood, MS, United States.
Kansas City Research Institute, Kansas City, MO, United States.
DHR Health Transplant Institute, McAllen, TX, United States.
Austin Health
NGM Biopharmaceuticals, South San Francisco, CA, United States.
Virginia Commonwealth University, Richmond, VA, United States.
NGM Biopharmaceuticals, South San Francisco, CA, United States.
Radcliffe Department of Medicine, University of Oxford, Oxford, United Kingdom.;Pinnacle Clinical Research, San Antonio, TX, United States.
Issue Date: 21-Sep-2023
Date: 2023
Publication information: Hepatology (Baltimore, Md.) 2023-09-21
Abstract: Aldafermin, an engineered analog of the human hormone FGF19, improves liver histology in patients with non-cirrhotic nonalcoholic steatohepatitis (NASH), however, its efficacy and safety in compensated cirrhosis is unknown. No drug has yet to demonstrate benefit in the compensated NASH population. In this multicenter, double-blind, placebo-controlled, phase 2b trial, 160 patients with compensated NASH cirrhosis were randomized to aldafermin 0.3 mg (n=7), 1 mg (n=42), 3 mg (n=55), or placebo (n=56) for 48 weeks. The 0.3 mg group was discontinued to limit exposure to suboptimal dose. The primary end point was a change in ELF from baseline to week 48. The analyses were performed in the intention-to-treat population. At week 48, the least squares mean difference in the change in ELF was ̶ 0.5 (95% confidence interval [CI], ̶ 0.7 to ̶ 0.2; p=0.0003) between the 3 mg group and the placebo group. 15%, 21% and 23% of patients in the placebo, 1 mg and 3 mg group, respectively, achieved fibrosis improvement ≥1-stage; and 13%, 16% and 20% achieved fibrosis improvement ≥1-stage without NASH worsening. Improvement in ALT, AST, Pro-C3 and liver stiffness favored aldefermin groups over placebo. Diarrhea was the most frequent adverse event, occurring 26% and 40% in the 1 mg and 3 mg groups, respectively, compared to 18% in the placebo group. 0%, 2% and 9% of patients in the placebo, 1 mg and 3 mg group, respectively, discontinued due to treatment-related adverse events. Aldafermin 3 mg resulted in significant reduction in ELF in patients with compensated NASH cirrhosis.
DOI: 10.1097/HEP.0000000000000607
Journal: Hepatology (Baltimore, Md.)
PubMed URL: 37732990
ISSN: 1527-3350
Type: Journal Article
Appears in Collections:Journal articles

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