Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as Efruxifermin
EFX · 6 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.
Proportion of subjects who achieve ≥ 1 stage improvement in fibrosis (based on NASH CRN fibrosis score) and no worsening of steatohepatitis at Week 36. No worsening of steatohepatitis was defined as no increase in score for any of the 3 components of NAS. The evaluation of the NAS endpoint was calculated using the following 3 categorical features: steatosis \[0-3\], lobular inflammation \[0-3\], and hepatocellular ballooning \[0-2\]. NAS was derived as the unweighted sum of steatosis, lobular inflammation, and hepatocellular ballooning scores.
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
Main study. ANCOVA multiple imputation with treatment group and F1 fibrosis score (F1 vs F2-3) as factors and baseline hepatic fat fraction measured by MRI-PDFF as a covariate.
| Arm | Type | Description |
|---|---|---|
| EFX 50 mg | EXPERIMENTAL | - |
| Placebo | PLACEBO_COMPARATOR | - |
| EFX 28 mg | EXPERIMENTAL | - |
| EFX 50 mg (Open-Label Rollover) | EXPERIMENTAL | - |
| EFX 28 mg (Main Study) | EXPERIMENTAL | - |
| EFX 50 mg (Main Study) | EXPERIMENTAL | - |
| Placebo (Main Study) | PLACEBO_COMPARATOR | - |
| EFX 50 mg (Cohort D) | EXPERIMENTAL | - |
| Placebo (Cohort D) | PLACEBO_COMPARATOR | - |
| Efruxifermin 28 mg | EXPERIMENTAL | double-blind, once-weekly subcutaneous injection |
| Efruxifermin 50 mg | PLACEBO_COMPARATOR | double-blind, once-weekly subcutaneous injection |
| EFX Dose 1 | EXPERIMENTAL | Main Study |
| EFX Dose 2 | EXPERIMENTAL | Main Study |
| EFX Dose 3 | EXPERIMENTAL | Main Study |
| EFX Dose (Cohort C) | EXPERIMENTAL | - |
| Placebo (Cohort C) | PLACEBO_COMPARATOR | - |
| Name | Type | Description |
|---|---|---|
| Efruxifermin | DRUG | Administered by subcutaneous injection |
| Placebo | DRUG | Administered by subcutaneous injection |
| EFX | DRUG | Investigational drug, Efruxifermin |
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...
EFX is an investigational small molecule being developed for the treatment of nonalcoholic steatohepatitis (NASH), a form of fatty liver disease. It is currently in Phase 2 clinical development and has not been approved by the FDA.
EFX 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 to evaluate the drug's safety and efficacy in patients with NASH.
EFX is in Phase 2 clinical development. Two Phase 2 trials have been completed, one involving 110 participants and another involving 213 participants, both in patients with NASH. The drug remains investigational and is not yet approved.
EFX has completed two Phase 2 clinical trials. The first, NCT03976401, enrolled 110 participants with histologically confirmed NASH in the United States and Puerto Rico. The second, NCT05039450, enrolled 213 participants with compensated cirrhosis due to NASH in the United States, Mexico, and Puerto Rico.
Yes, EFX is also known as efruxifermin. Both names refer to the same investigational drug being developed by Akero Therapeutics for the treatment of nonalcoholic steatohepatitis (NASH).