#1 Clinical Trials Search in Europe

Efruxifermin

Efruxifermin (EFX) is an innovative drug currently undergoing clinical trials for the treatment of nonalcoholic steatohepatitis (NASH) and metabolic dysfunction-associated steatohepatitis (MASH). These conditions are serious liver diseases associated with fat accumulation, inflammation, and fibrosis. The ongoing studies aim to evaluate the safety and efficacy of EFX in patients with various stages of liver disease, from non-cirrhotic to compensated cirrhosis. The trials are designed to assess improvements in liver health, metabolic parameters, and overall patient outcomes.

Table of Contents

What is Efruxifermin?

Efruxifermin, also known as EFX, is a new drug being studied for the treatment of various liver diseases[1]. It is currently undergoing clinical trials to evaluate its safety and effectiveness in patients with different types of liver conditions.

What Conditions Does Efruxifermin Treat?

Efruxifermin is being investigated for the treatment of several liver-related conditions, including:

  • Nonalcoholic Steatohepatitis (NASH): Also known as Metabolic Dysfunction-Associated Steatohepatitis (MASH), this is a severe form of fatty liver disease not caused by alcohol consumption[1][2].
  • Nonalcoholic Fatty Liver Disease (NAFLD): Also referred to as Metabolic Dysfunction-Associated Steatotic Liver Disease (MASLD), this is a broader category of liver conditions involving fat accumulation in the liver[3].
  • Liver Fibrosis: This refers to scarring of the liver tissue, which can occur in various stages (F2, F3, or F4/cirrhosis)[2].

How is Efruxifermin Administered?

Efruxifermin is administered through subcutaneous injection. This means the medication is injected just under the skin, rather than into a vein or muscle[1][2][3].

Current Clinical Trials

Efruxifermin is currently being studied in several Phase 3 clinical trials. These are large-scale studies designed to confirm the drug’s effectiveness and safety. The ongoing trials include:

  1. A study for patients with compensated cirrhosis due to NASH/MASH[1].
  2. A study for patients with non-cirrhotic NASH/MASH and liver fibrosis[2].
  3. A study for patients with non-invasively diagnosed NASH/MASH and NAFLD/MASLD[3].

These trials are randomized, double-blind, and placebo-controlled, which means that participants are randomly assigned to receive either Efruxifermin or a placebo (a substance with no active ingredients). Neither the participants nor the researchers know who is receiving which treatment until the study is completed. This design helps ensure that the results are as unbiased as possible.

Potential Benefits of Efruxifermin

Based on the clinical trial designs, Efruxifermin is being evaluated for several potential benefits:

  • Improvement in liver fibrosis: The drug may help reduce scarring in the liver[1][2].
  • Resolution of NASH/MASH: It may help reduce inflammation and fat accumulation in the liver[2].
  • Improvement in liver function markers: The trials are measuring changes in various blood tests that indicate liver health, such as ALT and AST[1][2][3].
  • Effects on lipid levels: The drug may have positive effects on cholesterol and triglyceride levels[1][2][3].
  • Improvement in insulin sensitivity and blood sugar control: This could be particularly beneficial for patients with diabetes or pre-diabetes[1][2].
  • Potential weight loss: Changes in body weight are being monitored in the trials[1][2][3].

Safety and Side Effects

As with any new medication, understanding the safety profile and potential side effects of Efruxifermin is a crucial part of the clinical trials. The studies are closely monitoring:

  • Adverse events (any unfavorable or unintended sign, symptom, or disease that occurs during the study)[1][2][3].
  • The severity of any side effects[1][2][3].
  • Changes in laboratory test results, including liver function tests, blood counts, and other indicators of overall health[1][2][3].
  • Changes in vital signs, electrocardiograms (ECGs), and ultrasound results[1][2][3].

It’s important to note that the full safety profile of Efruxifermin will not be known until these clinical trials are completed and the results are analyzed. Patients considering participation in a clinical trial should discuss the potential risks and benefits with their healthcare provider.

Aspect Details
Drug Name Efruxifermin (EFX)
Administration Subcutaneous injection
Target Conditions NASH, MASH, NAFLD, MASLD with various stages of liver fibrosis
Study Design Randomized, double-blind, placebo-controlled
Primary Outcomes Improvement in liver fibrosis, resolution of NASH/MASH, event-free survival
Secondary Outcomes Changes in non-invasive liver markers, lipid profiles, glycemic control, body weight
Safety Assessments Adverse events, laboratory tests, ECGs, ultrasounds, vital signs
Study Duration Up to 240 weeks for long-term follow-up

FAQ

  • What is Efruxifermin (EFX)? Efruxifermin (EFX) is a drug being studied for the treatment of nonalcoholic steatohepatitis (NASH) and metabolic dysfunction-associated steatohepatitis (MASH). It is administered by subcutaneous injection and is being tested in various clinical trials to evaluate its safety and effectiveness in improving liver health.
  • What conditions is Efruxifermin being studied for? Efruxifermin is being studied for NASH (Nonalcoholic Steatohepatitis), MASH (Metabolic Dysfunction-Associated Steatohepatitis), NAFLD (Nonalcoholic Fatty Liver Disease), and MASLD (Metabolic Dysfunction-Associated Steatotic Liver Disease). These conditions involve fat accumulation in the liver, inflammation, and varying degrees of liver damage.
  • How is Efruxifermin administered in the clinical trials? In the clinical trials, Efruxifermin is administered by subcutaneous injection. This means the drug is injected just under the skin, allowing for gradual absorption into the bloodstream.
  • What are the main outcomes being measured in the Efruxifermin trials? The main outcomes being measured include improvements in liver fibrosis, resolution of NASH/MASH, changes in non-invasive markers of liver health (such as ELF score and liver stiffness), changes in lipid profiles, markers of insulin sensitivity and glycemic control, body weight changes, and overall safety and tolerability of the drug.
  • How long do the Efruxifermin clinical trials last? The duration of the clinical trials varies, but some studies extend up to 240 weeks (about 4.6 years) for long-term follow-up. Shorter-term outcomes are often assessed at 52 or 96 weeks. The extended duration allows researchers to evaluate both the short-term and long-term effects of the drug.

Ongoing Clinical Trials on Efruxifermin

  • Study on Efruxifermin for Patients with Non-Cirrhotic Nonalcoholic Steatohepatitis (NASH) and Fibrosis

    Recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    France Germany Italy Poland Spain

  • Study on Efruxifermin for Patients with Compensated Cirrhosis Due to Nonalcoholic Steatohepatitis (NASH) or Metabolic Dysfunction-Associated Steatohepatitis (MASH)

    Recruiting

    1 1
    Investigated drugs:
    France Germany Italy Poland Spain

Glossary

  • NASH: Nonalcoholic Steatohepatitis: A type of liver disease characterized by fat accumulation, inflammation, and damage in people who drink little or no alcohol.
  • MASH: Metabolic Dysfunction-Associated Steatohepatitis: A term similar to NASH, emphasizing the metabolic factors contributing to liver inflammation and damage.
  • Fibrosis: The formation of excess fibrous connective tissue in the liver, which can occur as a result of inflammation or injury.
  • Cirrhosis: Advanced scarring of the liver caused by long-term liver damage, where healthy liver tissue is replaced by scar tissue.
  • ELF Score: Enhanced Liver Fibrosis score: A blood test that measures markers of liver fibrosis, with scores ranging from 6.7 to 11.3, where higher scores indicate increased fibrosis.
  • FibroScan: A non-invasive test that uses ultrasound technology to measure liver stiffness, which can indicate the presence and severity of liver fibrosis.
  • FAST Score: FibroScan-AST Score: A measure that combines liver stiffness measurement and blood test results to assess the likelihood of significant liver inflammation and fibrosis.
  • ALT and AST: Alanine aminotransferase and Aspartate aminotransferase: Enzymes found primarily in the liver that can indicate liver damage when levels are elevated in the blood.
  • HDL-C: High-Density Lipoprotein Cholesterol: Often referred to as 'good' cholesterol, it helps remove other forms of cholesterol from the bloodstream.
  • LDL-C: Low-Density Lipoprotein Cholesterol: Often called 'bad' cholesterol, high levels can contribute to the buildup of plaque in arteries.
  • HOMA-IR: Homeostatic Model Assessment of Insulin Resistance: A method used to quantify insulin resistance and beta-cell function, with values ≥ 2.5 indicating insulin resistance.
  • Subcutaneous Injection: A method of administering medication by injecting it into the layer of fat just beneath the skin.
  • Placebo: An inactive substance that looks like the drug being tested, used in clinical trials to help determine the effectiveness of the actual drug.

References

  1. https://clinicaltrials.gov/study/NCT06528314
  2. https://clinicaltrials.gov/study/NCT06215716
  3. https://clinicaltrials.gov/study/NCT06161571