Evidence map›Paper›PMID 36644237›Full record

ArticleJHEP reports : innovation in hepatology2023

A randomized, double-blind, placebo-controlled phase IIa trial of efruxifermin for patients with compensated NASH cirrhosis.

Stephen A Harrison, Peter J Ruane, Bradley Freilich, Guy Neff, Rashmee Patil, Cynthia Behling, Chen Hu, Reshma Shringarpure, Brittany de Temple, Erica Fong and 4 more

Registry-linked trialOpen access · goldAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in JHEP reports : innovation in hepatology, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03976401 (A Phase 2a, Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Safety and Efficacy of Efruxifermin in Subjects With Nonalcoholic Steatohepatitis), which is not on this map. Cited by 65 papers, 5 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
65citing papers in PubMed, 5 pooled it
13.6field-weighted citation impact, top 1% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT03976401 phase2completednot on this map

A Phase 2a, Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Safety and Efficacy of Efruxifermin in Subjects With Nonalcoholic Steatohepatitis (NASH)

TypeinterventionalSponsorAkero Therapeutics, IncRan2019 to 2022Enrolled110ConditionsNASH - Nonalcoholic SteatohepatitisArmsEFX, Placebo
3 · Its place in the literature

Who cites it

65 citing papers in PubMed, 5 syntheses or guidelines pooled it, 93 citations in OpenAlex.

  1. Pooled it
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  8. A Phase 2a Trial of Myeloperoxidase Inhibitor Mitiperstat in Non-Cirrhotic Metabolic Dysfunction-Associated Steatohepatitis.Liver international : official journal of the International Association for the Study of the Liver · 2025
    Trial
  9. Review
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  12. Drug Development for MASH-Related Compensated Cirrhosis: Past, Present and Future.Liver international : official journal of the International Association for the Study of the Liver · 2026
    Review
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  14. Article
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5 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

14 authors at 7 institutions in 1 country.

Stephen A HarrisonPinnacle Clinical Research, San Antonio, TX, United States.
Peter J RuaneRuane Clinical Research Group Inc., Los Angeles, CA, United States.
Bradley FreilichKansas City Research Institute, Kansas City, MO, United States.
Guy NeffCovenant Metabolic Specialists, LLC, Sarasota, FL, United States.
Rashmee PatilSouth Texas Research Institute, Edinburg, TX, United States.
Cynthia BehlingUniversity of California San Diego, CA, United States.
Chen HuMedPace, INC, Cincinnati, OH, United States.
Reshma ShringarpureAkero Therapeutics, South San Francisco, CA, United States.
Brittany de TempleAkero Therapeutics, South San Francisco, CA, United States.
Erica FongAkero Therapeutics, South San Francisco, CA, United States.
Erik J TillmanAkero Therapeutics, South San Francisco, CA, United States.
Timothy RolphAkero Therapeutics, South San Francisco, CA, United States.
Andrew ChengAkero Therapeutics, South San Francisco, CA, United States.
Kitty YaleAkero Therapeutics, South San Francisco, CA, United States.
Spero Therapeutics (United States) · USKansas City Research Institute · USMedpace (United States) · USPinnacle Clinical Research · USRuane Medical · USTexas Research Institute · USUniversity of California San Diego · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Background & Aims: Efruxifermin has shown clinical efficacy in patients with non-alcoholic steatohepatitis (NASH) and F1-F3 fibrosis. The primary objective of the BALANCED Cohort C was to assess the safety and tolerability of efruxifermin in patients with compensated NASH cirrhosis. Methods: Patients with NASH and stage 4 fibrosis (n = 30) were randomized 2:1 to receive efruxifermin 50 mg (n = 20) or placebo (n = 10) once-weekly for 16 weeks. The primary endpoint was safety and tolerability of efruxifermin. Secondary and exploratory endpoints included evaluation of non-invasive markers of liver injury and fibrosis, glucose and lipid metabolism, and changes in histology in a subset of patients who consented to end-of-study liver biopsy. Results: Efruxifermin was safe and well-tolerated; most adverse events (AEs) were grade 1 (n = 7, 23.3%) or grade 2 (n = 19, 63.3%). The most frequent AEs were gastrointestinal, including transient, mild to moderate diarrhea, and/or nausea. Significant improvements were noted in key markers of liver injury (alanine aminotransferase) and glucose and lipid metabolism. Sixteen-week treatment with efruxifermin was associated with significant reductions in non-invasive markers of fibrosis including Pro-C3 (least squares mean change from baseline [LSMCFB] -9 μg/L efruxifermin Conclusions: Efruxifermin appeared safe and well-tolerated with encouraging improvements in markers of liver injury, fibrosis, and glucose and lipid metabolism following 16 weeks of treatment, warranting confirmation in larger and longer term studies. Lay summary: Cirrhosis resulting from non-alcoholic steatohepatitis (NASH), the progressive form of non-alcoholic fatty liver disease, represents a major unmet medical need. Currently there are no approved drugs for the treatment of NASH. This proof-of-concept randomized, double-blind clinical trial demonstrated the potential therapeutic benefit of efruxifermin treatment compared to placebo in patients with cirrhosis due to NASH. Clinical Trial Number: NCT03976401.

Indexed as

ADA(s), anti-drug antibody(ies)AE, adverse eventALP, alkaline phosphataseALT, alanine aminotransferaseANCOVA, analysis of covarianceAST, aspartate aminotransferaseCFB, change from baselinecirrhosisclinical trialC–P, Child-PughCTX-1, C-terminal telopeptide of type 1 collagenDXA, dual-energy X-ray absorptiometryefruxiferminELF, enhanced liver fibrosisFGF21FGF21, fibroblast growth factor-21FGFR, fibroblast growth factor receptorGGT, gamma-glutamyltransferaseHbA1c, hemoglobin A1cHDL-C, HDL-cholesterolhistopathologyHOMA-IR, homeostatic model assessment of insulin resistanceHPA, hypothalamic-pituitary-adrenalhs-CRP, high-sensitivity C-reactive proteinINR, international normalized ratioIRT, interactive response technologyLDL-C, LDL-cholesterolliver diseaseLS, least squaresMELD, model for end-stage liver diseaseNAb, neutralizing antibodyNAFLD, non-alcoholic fatty liver diseaseNASH, non-alcoholic steatohepatitisNAS, NAFLD activity scorenonalcoholic fatty liver disease/NAFLDnon-alcoholic steatohepatitis/NASHNon-HDL-C, non-HDL-cholesterolP1NP, procollagen type-I N-terminal propeptideP3NP, procollagen type III N-terminal propeptidePAI-1, plasminogen activator inhibitor-1Pro-C3, N-terminal type III collagen propeptideTEAE, treatment-emergent adverse eventTIMP-1, tissue inhibitor of metalloproteinase-1ULN, upper limit of normal

Identifiers

PMID36644237
PMCPMC9832280
OpenAlexW4292830211

What OpenQuestion holds

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LicenceCC BY
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Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.