What is the mechanism of action of this treatment?

Common treatments for Nonalcoholic Steatohepatitis (NASH) target key pathways involved in the disease, such as lipid metabolism, inflammation, and fibrosis. Efruxifermin (EFX), an FGF21 agonist, stimulates fatty acid oxidation, reduces lipogenesis, and improves insulin sensitivity, thereby decreasing hepatic fat and inflammation. Other treatments, like GLP-1 receptor agonists, enhance glucose and lipid metabolism, while antioxidants reduce oxidative stress. These mechanisms are vital for NASH patients as they address the root metabolic dysfunctions and liver damage, potentially slowing disease progression and improving liver health.

What side effects are associated with this type of intervention?

Common treatments for Nonalcoholic Steatohepatitis (NASH) include lifestyle modifications, pharmacologic agents like pioglitazone, vitamin E, and newer agents such as FGF21 agonists. Efruxifermin (an FGF21 agonist) and similar treatments have shown promise in reducing hepatic fat and fibrosis. Known side effects of FGF21 agonists can include gastrointestinal issues such as nausea and diarrhea, as well as potential injection site reactions. Other pharmacologic treatments like pioglitazone may cause weight gain, fluid retention, and an increased risk of bone fractures. Vitamin E is generally well-tolerated but can increase the risk of hemorrhagic stroke in high doses.

What prior FDA approvals exist?

As of now, there are no FDA-approved treatments specifically for Nonalcoholic Steatohepatitis (NASH). Efruxifermin (EFX), an FGF21 agonist, is currently being studied for its potential benefits in treating NASH. Other investigational therapies for NASH include GLP-1 receptor agonists, which have shown promise in improving glucose and lipid metabolism, reducing hepatic fat content, and improving liver enzymes. However, these treatments are still under investigation and have not yet received FDA approval for NASH.

What other types of research exist?

Promising alternatives to Efruxifermin for NASH patients include NGM282, an engineered fibroblast growth factor 19 analogue, which has shown significant reduction in liver fat content in clinical trials. Additionally, other therapies being studied in clinical trials include peroxisome proliferator-activated receptor agonists, cyclin-dependent kinase inhibitors, and mitogen-activated protein kinase inhibitors. These alternatives are in various stages of research and may offer new treatment options in the near future.

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Growth Factor

Efruxifermin for NASH

Verified Trial

Phase 3

Recruiting

Research Sponsored by Akero Therapeutics, Inc

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Previous history or presence of 2 out of 4 components of metabolic syndrome (obesity, dyslipidemia, elevated blood pressure, elevated fasting glucose) or type 2 diabetes

Biopsy-proven NASH. Must have had a liver biopsy obtained ≤ 180 days prior to screening with fibrosis stage 2 or 3 and a non-alcoholic fatty liver disease (NAFLD) activity score (NAS) of ≥ 4 with at least a score of 1 in each of the following NAS components: Steatosis (scored 0 to 3), Ballooning degeneration (scored 0 to 2), and Lobular inflammation (scored 0 to 3)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 52 weeks, 96 weeks

Awards & highlights

Pivotal Trial

Summary

This trial is testing a medication called efruxifermin (EFX) to see if it can help people with a specific liver condition called non-cirrhotic NASH/MASH. The patients have significant liver damage but not cirrhosis. EFX aims to improve liver health by reducing swelling and scarring in the liver.

Who is the study for?

This trial is for people with a liver condition called non-cirrhotic NASH or MASH, which involves fat buildup and fibrosis (scarring) at stage 2 or 3. Participants should not have cirrhosis, a more advanced liver scarring.

What is being tested?

The study tests Efruxifermin (EFX), comparing it to a placebo in a controlled setting. Participants are randomly assigned to either the drug group or the placebo group without knowing which one they receive.

What are the potential side effects?

Potential side effects of Efruxifermin may include gastrointestinal issues like nausea or diarrhea, reactions at the injection site, and possible changes in blood sugar levels.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have at least two conditions like obesity, high cholesterol, high blood pressure, or high blood sugar.

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I have NASH confirmed by a recent biopsy showing specific liver damage.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 52 weeks, 96 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~52 weeks, 96 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 52 weeks, 96 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Secondary study objectives

Change from baseline in liver fibrosis and no worsening of steatohepatitis

Change from baseline of body weight (kg)

Change from baseline of lipoproteins

+9 more

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

3Treatment groups

Experimental Treatment

Placebo Group

Group I: EFX 50 mgExperimental Treatment1 Intervention

Group II: EFX 28 mgExperimental Treatment1 Intervention

Group III: PlaceboPlacebo Group1 Intervention

0 / 2Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for Nonalcoholic Steatohepatitis (NASH) target key pathways involved in the disease, such as lipid metabolism, inflammation, and fibrosis. Efruxifermin (EFX), an FGF21 agonist, stimulates fatty acid oxidation, reduces lipogenesis, and improves insulin sensitivity, thereby decreasing hepatic fat and inflammation. Other treatments, like GLP-1 receptor agonists, enhance glucose and lipid metabolism, while antioxidants reduce oxidative stress. These mechanisms are vital for NASH patients as they address the root metabolic dysfunctions and liver damage, potentially slowing disease progression and improving liver health.

Current and Emerging Therapies for Non-alcoholic Fatty Liver Disease.