Hepion Begins ASCEND-NASH Trial Closure

Hepion Pharmaceuticals, a biopharmaceutical firm focused on developing treatments for non-alcoholic steatohepatitis (NASH), hepatocellular carcinoma (HCC), and chronic liver diseases, has initiated the shutdown of its Phase 2b ASCEND-NASH Trial due to resource limitations. The trial, which began screening its first patient in August 2022, aimed to assess the safety and efficacy of a 12-month treatment with rencofilstat. By April 2023, enrollment had been paused with 151 subjects randomized into the study. As of the announcement, around 80 participants had completed their Day 365 visits and were assessed for both safety and efficacy, with an additional 40 subjects expected to contribute to the safety data.

The company's executive chairman, John Brancaccio, expressed disappointment over the decision to wind down the Phase 2 NASH trial, which was solely due to resource constraints. Despite this setback, Hepion remains dedicated to pursuing the strategic alternatives it announced in December 2023. Given the current number of NASH patients enrolled and the slim chance of obtaining significant efficacy data with the existing financial resources, the company has chosen to conclude the trial and ensure a safe and compliant exit for the patients. Notably, no safety issues related to rencofilstat were reported during the trial.

Rencofilstat, Hepion's key drug candidate, is a potent cyclophilin inhibitor, which plays a role in various disease processes. It has demonstrated its ability to decrease liver fibrosis and reduce the tumor burden in HCC in experimental models. The drug received Fast Track designation from the U.S. Food and Drug Administration (FDA) for NASH treatment in November 2021 and was granted Orphan Drug status for HCC treatment in June 2022.

Hepion also operates a proprietary AI/ML platform that aims to deepen the understanding of disease mechanisms and identify individuals who are likely to respond to rencofilstat treatment. This technology has the potential to expedite the development process and enhance the distinction between placebo and treatment groups in clinical trials. Moreover, Hepion's AI/ML platform could be instrumental in advancing NASH and HCC clinical development programs and discovering new therapeutic applications for cyclophilin inhibition with rencofilstat.