Clinical Trials Overview
We are currently enrolling three clinical trials as part of a Phase 3 SYNCHRONY program evaluating EFX for the treatment of pre-cirrhotic MASH (F2-F3) and compensated cirrhosis (F4) due to MASH.
The SYNCHRONY studies build on a robust Phase 2 program and have been designed in consultation with global regulatory authorities.
SYNCHRONY
STATUS: ongoing
The Phase 3 SYNCHRONY clinical trial program consists of three randomized, placebo-controlled trials: (1) SYNCHRONY Histology, which is evaluating the efficacy and safety of EFX in patients with biopsy-confirmed pre-cirrhotic MASH (F2-F3); (2) SYNCHRONY Real-World, which is assessing the safety and tolerability of EFX in patients with non-invasively diagnosed MASH or metabolic dysfunction-associated steatotic liver disease (MASLD) (F1-F4); and (3) SYNCHRONY Outcomes, which is evaluating EFX for the treatment of compensated cirrhosis due to MASH (F4). The SYNCHRONY Histology and SYNCHRONY Real-World studies began enrolling patients in the fourth quarter of 2023, while SYNCHRONY Outcomes began enrolling patients in the third quarter of 2024.
In all EFX Phase 3 studies, patients will self-administer EFX using the LyoJect 3S dual chamber syringe, a pre-filled device intended for commercial use in the event EFX is approved for marketing. This optimized formulation delivers blood levels of EFX comparable to those of the liquid formulation used in prior clinical studies.
Read more about study details at Clinicaltrials.gov: SYNCHRONY Histology, SYNCHRONY Real-World, and SYNCHRONY Outcomes.
Learn more about enrolling in the SYNCHRONY Histology clinical trial.
SYMMETRY
STATUS: completed
The 96-week Phase 2b SYMMETRY study was a multicenter, randomized, double-blind, placebo-controlled, clinical trial that enrolled 182 patients with biopsy-confirmed compensated cirrhosis (F4, Child-Pugh class A) due to MASH who received once-weekly subcutaneous dosing of 28mg or 50mg EFX, or placebo.
Preliminary topline results of the study demonstrated statistically significant reversal of fibrosis following 96 weeks of treatment with EFX. Among patients with baseline and week 96 biopsies (N=134), 39% of patients treated with 50mg EFX (p<0.01) experienced reversal of cirrhosis with no worsening of MASH, compared with 15% for placebo. By ITT analysis (N=181), with all missing week 96 biopsies treated as failures and without imputation, 29% of the 50mg EFX group (p<0.05) experienced reversal of cirrhosis with no worsening of MASH, compared with 12% for placebo. Histologically confirmed cirrhosis reversal was corroborated by improvements in key noninvasive measures of liver fibrosis and injury.
Read more about the topline results of the study.
