Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as Efimosfermin
Efimosfermin alfa · 9 trials · 4 indications
Liver-related outcome comprises all-cause mortality; liver transplantation; occurrence of significant hepatic decompensation events.
Participants experiencing improvement in liver fibrosis of \>=1 stage (based on MASH Clinical Research Network (CRN) fibrosis score) and no worsening of MASH (defined as no increase in nonalcoholic fatty liver disease activity score for ballooning, inflammation, or steatosis). MASH CRN fibrosis score ranges from 0 to 4, higher score indicates greater severity.
Proportion of participants experiencing improvement in fibrosis of greater than or equal to (\>=) 1 stage by MASH clinical research network (CRN) fibrosis scores and no worsening of steatohepatitis (defined as no increase in nonalcoholic fatty liver disease activity score \[NAS\] for ballooning, inflammation, or steatosis) at 52 weeks will be assessed. MASH CRN fibrosis score ranges from 0 to 4, higher score indicates greater severity. NAS score ranges from 0 to 8, higher score indicates worse disease activity.
Resolution of steatohepatitis is defined as absence of fatty liver disease or isolated or simple steatosis without steatohepatitis and a NAS of 0 or 1 for inflammation, 0 for ballooning, and any value for steatosis. NAS score ranges from 0 to 8, higher score indicates worse disease activity. MASH CRN fibrosis score ranges from 0 to 4, higher score indicates greater severity.
VCTE-LSM is a non-invasive test that uses vibration-controlled transient elastography to measure the stiffness of the liver in kilopascals (kPa)
MELD is a scoring system for assessing the severity of chronic liver disease. MELD scores range between 6 and 40, with 40 being the most severe.
To assess the effects of efimosfermin on safety and tolerability.
The effects of Efimosfermin on safety and tolerability will be assessed.
The effects of Efimosfermin on safety and tolerability will be assessed.
The effects of Efimosfermin on safety and tolerability will be assessed.
The effects of Efimosfermin on safety and tolerability will be assessed.
| Arm | Type | Description |
|---|---|---|
| Participants receiving efimosfermin alfa | EXPERIMENTAL | - |
| Participants receiving placebo | PLACEBO_COMPARATOR | - |
| Part A: Participants receiving Efimosfermin alfa | EXPERIMENTAL | Participants will receive efimosfermin alfa in Part A of the study. |
| Part A: Participants receiving Placebo | PLACEBO_COMPARATOR | Participants will receive placebo in Part A of the study. |
| Part B: Participants receiving Efimosfermin alfa | EXPERIMENTAL | All participants will receive efimosfermin alfa in Part B of the study. |
| Efimosfermin Alfa Dose Level 1 | EXPERIMENTAL | Participants randomized to this group will receive Efimosfermin Alfa at dose level 1 |
| Efimosfermin Alfa Dose Level 2 | EXPERIMENTAL | Participants randomized to this group will receive Efimosfermin Alfa at dose level 2 |
| Placebo | PLACEBO_COMPARATOR | Participants randomized to this group will receive Placebo |
| Participants receiving dose level 1 of efimosfermin alfa | EXPERIMENTAL | - |
| Participants receiving dose level 2 of efimosfermin alfa | EXPERIMENTAL | - |
| Efimosfermin alfa Dose Level 3 | EXPERIMENTAL | Participants will receive efimosfermin alfa dose level 3. |
| Efimosfermin (Dose 1) | EXPERIMENTAL | Participants will receive efimosfermin (Dose 1) once every 4 weeks (Q4W). |
| Efimosfermin (Dose 2) | EXPERIMENTAL | Participants will receive efimosfermin (Dose 2) Q4W. |
| Cohort A1: Efimosfermin Dose 1 or placebo (PBO) | EXPERIMENTAL | - |
| Cohort A2: Efimosfermin Dose 2 or PBO | EXPERIMENTAL | - |
| Cohort A3: Efimosfermin Dose 3 or PBO | EXPERIMENTAL | - |
| Cohort A4: Efimosfermin Dose 4 or PBO | EXPERIMENTAL | - |
| Cohort A5: Efimosfermin Dose 5 or PBO | EXPERIMENTAL | - |
| Part B: Efimosfermin Dose 1 or PBO | EXPERIMENTAL | - |
| Part C: Efimosfermin Dose 1 | EXPERIMENTAL | - |
| Part D: Efimosfermin Dose 6 or PBO | EXPERIMENTAL | - |
| Part D: Efimosfermin Dose 1 or PBO | EXPERIMENTAL | - |
| Efimosfermin alfa in participants with moderate hepatic impairment due to MASH without alcohol | EXPERIMENTAL | All participants will receive efimosfermin alfa. Participants will have moderate hepatic impairment (Child-Pugh B) due to Metabolic Dysfunction-Associated Steatohepatitis (MASH) with typical alcohol consumption threshold in the 3 months prior to Screening of less than (\<) 5 standard drinks on any day and \<15 standard drinks per week for men; or \<4 standard drinks on any day and \<8 standard drinks per week for women. |
| Efimosfermin alfa in participants with moderate hepatic impairment due to MASH with alcohol | EXPERIMENTAL | All participants will receive efimosfermin alfa. Participants will have moderate hepatic impairment (Child-Pugh B) due to MASH with typical alcohol consumption threshold in the 3 months prior to Screening of greater than or equal to (\>=) 5 standard drinks per day or \>=15 standard drinks per week for men; or \>= 4 standard drinks per day or \>=8 or more drinks per week for women. |
| Efimosfermin alfa in severe hepatic impairment participants due to MASH regardless of alcohol use | EXPERIMENTAL | All participants will receive efimosfermin alfa. Participants will have severe hepatic impairment (Child-Pugh C) due to MASH with any typical daily alcohol consumption. |
| Efimosfermin alfa in participants of Chinese Ancestry | EXPERIMENTAL | Healthy participants of Chinese ancestry will be randomized to receive efimosfermin alfa. |
| Efimosfermin alfa in participants of Japanese Ancestry | EXPERIMENTAL | Healthy participants of Japanese ancestry will be randomized to receive efimosfermin alfa. |
| Efimosfermin alfa in participants of White/European Ancestry | EXPERIMENTAL | Healthy participants of White/European ancestry will be randomized to receive efimosfermin alfa. |
| Placebo in participants of Chinese Ancestry | PLACEBO_COMPARATOR | Healthy participants of Chinese ancestry will be randomized to receive Placebo. |
| Placebo in participants of Japanese Ancestry | PLACEBO_COMPARATOR | Healthy participants of Japanese ancestry will be randomized to receive Placebo. |
| Placebo in participants of White/European Ancestry | PLACEBO_COMPARATOR | Healthy participants of White/European ancestry will be randomized to receive Placebo. |
| Name | Type | Description |
|---|---|---|
| Efimosfermin alfa | DRUG | Efimosfermin alfa (subcutaneous injection) will be administered. |
| Placebo | DRUG | Placebo (subcutaneous injection) will be administered. |
| Efimosfermin | DRUG | Efimosfermin will be administered as a subcutaneous injection. |
Inclusion Criteria: * Participants aged between 18 and 75 years at enrollment. * Participants with compensated cirrhosis due to MASH, confirmed by non-invasive assessments. * Participants with history or presence of at least two components of metabolic syndrome. Exclusion Criteria: * Participants...
Efimosfermin alfa is an investigational medicine being developed by GSK plc for liver diseases. It is under study in alcohol-related liver disease, metabolic dysfunction-associated steatohepatitis (MASH), and non-alcoholic fatty liver disease. It has not been approved by any regulatory authority and remains in clinical development.
Efimosfermin alfa is being studied for liver conditions including alcohol-related liver disease, metabolic dysfunction-associated steatohepatitis (MASH), and non-alcoholic fatty liver disease. Clinical trials are enrolling adults with these conditions, including those with compensated cirrhosis due to MASH. It is investigational and not approved for any use.
Efimosfermin alfa is developed by GSK plc, which trades under the ticker GSK. GSK is running the clinical development program, which includes studies in alcohol-related liver disease, MASH with compensated cirrhosis, and hepatic impairment.
Efimosfermin alfa is in Phase 2 and Phase 3 clinical development. Two Phase 3 studies are recruiting in adults with compensated cirrhosis due to MASH, and Phase 2 and Phase 1 studies are also underway. The program has not been approved, so it remains investigational.
Efimosfermin alfa is being tested in four registered trials. These include NCT07701993 and NCT07704892, Phase 3 studies in compensated cirrhosis due to MASH, NCT07791043, a Phase 2 study in alcohol-related liver disease, and NCT07358546, a Phase 1 study in hepatic impairment. All four are currently recruiting.
Yes, efimosfermin alfa and efimosfermin refer to the same investigational drug developed by GSK plc. Efimosfermin is the shorter name used in some trial titles, such as the Phase 3 MASH studies, while efimosfermin alfa is the full name used in others.