Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Efruxifermin · 4 trials · 6 indications
Measured by composite of protocol-specified clinical events
Based on NASH CRN fibrosis score (scored by a fibrosis score of 0-4, where 0 = no fibrosis, 1 = centrilobular pericellular fibrosis, 2 = centrilobular and periportal fibrosis, 3 = bridging fibrosis, 4 = cirrhosis)
Based on NAS (scored by 0-3 for steatosis, 0-3 for inflammation, and 0-2 for ballooning) and NASH CRN fibrosis score (scored by a fibrosis score of 0-4, where 0 = no fibrosis, 1 = perisinusoidal or periportal fibrosis, 2 = perisinusoidal and portal/periportal fibrosis, 3 = bridging fibrosis, 4 = cirrhosis)
Based on time from randomization to the first clinical event including evidence of disease progression, liver decompensation events, liver transplantation or eligibility for liver transplantation, and all-cause mortality.
A participant's extent of exposure to study drug (weeks) will be generated from the data recorded in the study drug administration eCRF.
An adverse event (AE) is any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a study drug, whether or not related to the study drug.
All AEs, both serious and non-serious, will be assessed for severity using the Common Terminology Criteria for Adverse Events v5.0.
Clinical assessments include clinical laboratory tests, electrocardiogram, ultrasounds, vital sign assessments, and concomitant medication usage.
Proportions of subjects in EFX vs placebo groups with improvement in liver fibrosis, defined as ≥ 1 stage NASH Clinical Research Network \[CRN\] fibrosis score (score ranges from 0 to 4, increasing with fibrosis severity), and no worsening of steatohepatitis (no increase in NASH Activity Score \[NAS\], which ranges from 0 to 8 and is the sum of scores of steatosis, lobular inflammation, and hepatocyte ballooning), at Week 24
| Arm | Type | Description |
|---|---|---|
| EFX 50 mg | EXPERIMENTAL | - |
| Placebo | PLACEBO_COMPARATOR | - |
| EFX 28 mg | EXPERIMENTAL | - |
| EFX 50 mg (Open-Label Rollover) | EXPERIMENTAL | - |
| Efruxifermin 28 mg | EXPERIMENTAL | double-blind, once-weekly subcutaneous injection |
| Efruxifermin 50 mg | PLACEBO_COMPARATOR | double-blind, once-weekly subcutaneous injection |
| Name | Type | Description |
|---|---|---|
| Efruxifermin | DRUG | Administered by subcutaneous injection |
| Placebo | DRUG | Administered by subcutaneous injection |
Inclusion Criteria: * Cohort 1: Biopsy proven compensated cirrhosis (fibrosis stage 4) due to NASH/MASH and NAS score of \>=3 (at least 1 in each category) or evidence of steatosis and 2 current features of metabolic comorbidities * Cohort 2: Biopsy proven or non-invasively diagnosed compensated ci...
Efruxifermin is an investigational small molecule being developed for nonalcoholic steatohepatitis (NASH), also known as metabolic dysfunction-associated steatohepatitis (MASH), including NASH with fibrosis, non-cirrhotic NASH, and compensated cirrhosis due to NASH/MASH. It is also being studied in nonalcoholic fatty liver disease (NAFLD) and metabolic dysfunction-associated steatotic liver disease (MASLD).
Efruxifermin is being developed by Akero Therapeutics, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol AKRO. The company is conducting clinical trials of Efruxifermin in multiple countries for the treatment of NASH and related metabolic liver diseases.
Efruxifermin is currently in Phase 3 clinical development. It has completed a Phase 2 trial and is being evaluated in multiple Phase 3 trials for NASH/MASH, including studies in non-cirrhotic NASH with fibrosis and compensated cirrhosis due to NASH/MASH. Efruxifermin is investigational and not yet approved by regulatory authorities.
Efruxifermin is being studied in several Phase 3 trials. NCT06161571 evaluates Efruxifermin in subjects with non-invasively diagnosed NASH/MASH and NAFLD/MASLD. NCT06215716 studies Efruxifermin in non-cirrhotic NASH/MASH with fibrosis. NCT06528314 assesses Efruxifermin in compensated cirrhosis due to NASH/MASH. A Phase 2 trial, NCT04767529, has been completed.
Efruxifermin is not FDA approved. It is an investigational drug currently in Phase 3 clinical trials for NASH/MASH and related conditions. The drug has not completed the regulatory approval process, and its safety and efficacy have not been established for commercial use.
The Efruxifermin clinical development program includes trials with substantial enrollment. The completed Phase 2 trial enrolled 128 participants. The Phase 3 trials have planned enrollments of 700, 1650, and 2150 participants, respectively, for a total planned enrollment of approximately 4500 across the Phase 3 program.