Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as EFX
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 by Akero Therapeutics for nonalcoholic steatohepatitis (NASH), also called metabolic dysfunction-associated steatohepatitis (MASH). It is being studied in patients with NASH with fibrosis and in patients with compensated cirrhosis due to NASH/MASH. It has not been approved by the FDA and remains in clinical development.
Efruxifermin is being studied for the treatment of NASH/MASH, including non-cirrhotic NASH with fibrosis and compensated cirrhosis due to NASH/MASH. Clinical trials also include patients with non-invasively diagnosed NASH/MASH and NAFLD/MASLD. It is investigational and has not been approved for any indication.
Efruxifermin is being developed by Akero Therapeutics, Inc., which trades on the Nasdaq under the ticker AKRO. Akero is the sponsor of the Phase 3 and Phase 2 clinical trials evaluating efruxifermin in NASH/MASH and related liver conditions.
Efruxifermin is in Phase 3 clinical development. Akero Therapeutics is running three Phase 3 trials in NASH/MASH, and a Phase 2 trial in non-cirrhotic patients with histologically confirmed NASH has been completed. Efruxifermin is investigational and has not received FDA approval.
Efruxifermin is being evaluated in Phase 3 trials NCT06528314 in compensated cirrhosis due to NASH/MASH, NCT06215716 in non-cirrhotic NASH/MASH with fibrosis, and NCT06161571 in non-invasively diagnosed NASH/MASH and NAFLD/MASLD. A completed Phase 2 trial, NCT04767529, studied efruxifermin in non-cirrhotic patients with histologically confirmed NASH.
The efruxifermin clinical program has enrolled 2,278 patients across its trials. The Phase 3 trial NCT06528314 plans to enroll 2,150 patients, NCT06215716 plans 1,650 patients, and NCT06161571 plans 700 patients, while the completed Phase 2 trial NCT04767529 enrolled 128 patients. All trials are randomized, double-blind, and placebo-controlled.