Galmed's Aramchol Shows Promise in Hepatocellular Carcinoma Combination Therapy, Prompting Phase 1/2 Trial
Key Insights
Galmed Pharmaceuticals announced acceptance of a late-breaking abstract at HEP-DART 2025 demonstrating that Aramchol enhances regorafenib activity in hepatocellular carcinoma (search) models.
The research shows SCD1 (search) inhibition by Aramchol augments regorafenib activity through ATM (search)-AMPK (search)-autophagy signaling pathways in gastrointestinal cancer (search) models.
VCU Massey Comprehensive Cancer Center (search) will initiate an investigator-initiated Phase 1/2 clinical trial of the Aramchol-regorafenib combination in advanced GI cancers starting in 2026.
Galmed Pharmaceuticals Ltd. (NASDAQ: GLMD) announced that its lead drug candidate Aramchol has demonstrated significant potential in enhancing the efficacy of standard-of-care regorafenib in hepatocellular carcinoma (search) (HCC) models, leading to the acceptance of a late-breaking abstract at the prestigious HEP-DART 2025 Meeting.
Preclinical Data Supports Combination Approach
The company's research revealed that Aramchol significantly enhances Bayer's regorafenib effect in gastrointestinal cancer (search) models to kill GI tumor cells. The mechanism involves SCD1 (search) inhibition, which augments regorafenib (Stivarga®) activity through ATM (search)-AMPK (search)-autophagy signaling pathways. These findings provide the scientific and translational rationale for advancing the combination therapy into clinical development.
Clinical Trial Development Moves Forward
Based on these promising preclinical results, VCU Massey Comprehensive Cancer Center (search) has committed to initiating an investigator-initiated Phase 1/2 clinical trial of Aramchol combined with regorafenib in advanced gastrointestinal cancers, including HCC. Patient enrollment is planned to begin in 2026.
The clinical development strategy includes a dose-escalation phase to establish the recommended Phase 2 dose, followed by a dose expansion cohort that will incorporate Metformin to evaluate the efficacy of the three-drug combination.
Strategic Implications for Galmed
"The acceptance of our late-breaking abstract to the HEP-DART prestigious scientific meeting underscores the significance of our data," said Allen Baharaff, CEO of Galmed Pharmaceuticals. "The research work presented has directly informed VCU Massey Comprehensive Cancer's decision to initiate an investigator-initiated Phase 1/2 clinical trial of Aramchol and regorafenib in advanced GI cancers, including HCC, with planned enrollment starting in 2026."
Baharaff emphasized that positive findings from the upcoming trial could lay the groundwork for accelerated clinical development of Aramchol in three key GI cancers and potentially expand Galmed's oncology pipeline.
Company Focus and Pipeline Expansion
Galmed Pharmaceuticals is a clinical-stage biopharmaceutical company that has focused almost exclusively on developing Aramchol for liver diseases, particularly continuing to advance Aramchol for combination therapy in NASH (search) (non-alcoholic steatohepatitis (search)). The company is now seeking to develop Aramchol for certain oncological indications outside of NASH and fibrosis, representing a strategic expansion of its therapeutic focus.
As part of its growth strategy, Galmed is actively pursuing opportunities to expand and diversify its product pipeline, specifically targeting cardiometabolic indications and other innovative product candidates that align with its core expertise in drug development.
The late-breaking abstract, titled "Targeting SCD1 (search) enhances activity of standard of care regorafenib in hepatocellular carcinoma (search): translational rationale for a phase 1/2 study of the combination of Aramchol and regorafenib," will be presented as a poster on December 9 at 3pm (HST) at the HEP-DART 2025 Meeting.