Alcoholic Steatohepatitis (ASH) is the most florid manifestation of ALD, and it is often a precursor to the development of cirrhosis. The most severe form, Acute Alcoholic Hepatitis (AAH), is associated with a high 1-year mortality rate of 40%.

15 million people abuse/overuse alcohol in the USA, and there is a growing trend. It is believed that 90% of this population will eventually develop a fatty liver, which eventually leads to more serious conditions such as the most common manifestations of alcoholic liver disease (ALD):

  • Alcoholic Steatohepatitis (ASH)
  • Acute Alcoholic Hepatitis (AAH)
  • Cirrhosis due to alcohol

While abstinence is the most effective treatment for alcoholic hepatitis, AAH patients need a rescue treatment, capable of stopping the liver damage and inflammation, in the hope of restoring liver functions. Several drugs have been tested for this indication, but very few have shown any promise. In many cases, the patients’ conditions worsened with treatment.

Phase I completed

Verlyx has completed a phase I clinical study evaluating VLX103. This is a randomized, double-blind, placebo-controlled, sequential-group administration of a new oral form of pentamidine to investigate its hepatic uptake, pharmacokinetics, safety and tolerability. Results from this trial allows Verlyx to select specific doses to be used in subsequent Phase II trials.

Primary Objective

Liver concentration of pentamidine after oral administration for three days at different doses, measured in liver biopsies obtained during thermal ablation or partial hepatectomy procedures.

Secondary Objectives

Plasma concentration of pentamidine after oral administration for three days at different doses, safety as assessed by adverse events, vital signs and laboratory parameters. Plasma pharmacodynamic markers of anti-inflammatory/hepatoprotection efficacy (e.g. ALT and AST) will be monitored.

The abstracts detailing these results were presented at the American Association for the Study of Liver Disease (AASLD) in 2016. Abstracts #641 and #642 at the Liver Meeting®, in Boston, in November 2016 are available below.

Phase IB in ASH

Verlyx is currently planning a randomized phase IB in ASH.