First-line clinical trial marks a turning point in drug development 

Genoscience Pharma, a clinical-stage biotech company developing lysosomotropic drug candidates for the treatment of cancer, fibrosis and auto-immune diseases through autophagy modulation has launched the ABE-LIVER study, along with trial sponsor Grenoble University Hospital. 

The trial  

This Phase IIb clinical trial will focus on first-line treatment of HepatoCellular Carcinoma (HCC) patients with Genoscience Pharma’s drug candidate ezurpimtrostat (GNS561).  

This is administered in conjunction with standard atezolizumab/bevacizumab treatment. The first patient received the initial treatment on January 2, 2023 and Genoscience Pharma is providing ezurpimtrostat and operational support. 

ABE-LIVER is a multicenter, prospective, comparative, randomised and open Phase IIb trial. The principal investigator is Dr Gaël Roth, digestive oncologist and expert in primitive hepatic tumours at the Grenoble Alpes University Hospital (CHUGA).  

The main objective of this trial is to evaluate the efficacy of ezurpimtrostat in conjunction with standard treatment (atezolizumab-bevacizumab) compared to standard treatment alone, as the first-line treatment in patients fighting advanced HCC. The primary criteria is PFS. Between 187 and 196 patients will be enrolled in this trial, which will take place in two stages: a preliminary safety phase involving three to 12 patients, followed by an expansion phase. 

The trial is set to take place over three years, with intermediate results expected in 2024.


Although standard treatment with atezolizumab (an anti-PDL1) / bevacizumab (an antiangiogenic agent) does produce some encouraging results, a tumour response is observed in only 30% of patients and the Progression-Free Survival (PFS) period remains short. Not only does ezurpimtrostat demonstrate intrinsic anti-tumour activity, but its addition to standard treatment also allows regulation of autophagy, a key mechanism involved in immune evasion in the case of immunotherapy.

“This trial is an important step both for Genoscience and for our product ezurpimtrostat, giving us an opportunity to demonstrate the efficacy of autophagy inhibition in oncology,” said Professor Philippe Halfon, CEO of Genoscience Pharma. “We have high expectations for this trial, and we are delighted to be collaborating with Grenoble University Hospital, an institution involved in a number of major clinical trials in oncology, specifically promoting ABE-LIVER, thanks to its active network in the area of liver cancer.” 

Official comments 

“Ezurpimtrostat could become a new weapon in our arsenal of treatments for a form of cancer that is still extremely aggressive and for which current treatments are only able to achieve a low five-year survival rate,” said Professor Eric Raymond, medical director of Genoscience Pharma and head of medical oncology at the St-Joseph Hospital in Paris.

“By targeting PPT-1, ezurpimtrostat exerts an anti-tumour effect by reducing the nutrient intake of tumour cells and sensitises the tumour to immunotherapy by enhancing the expression of the Major Histocompatibility Complex (MHC-I) on the surface of the tumour cells. It also makes it possible to modulate the immune response through recolonisation and activation of the CD8+ cytotoxic T-cells, rendering it a highly promising treatment strategy,” added principal investigator Dr Roth. “We are thrilled to have been able to start this trial, which involves most of the specialist liver cancer centers in France. We are looking forward to evaluating the potential clinical impact of ezurpimtrostat in combination with atezolizumab-bevacizumab in HCC patients, whose treatment options are limited and whose prognosis remains very bleak at present.” 

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