The chronic respiratory disorders pharmaceutical specialist Pharmaxis completed the development of its experimental drug, the Semicarbazide-Sensitive Amine Oxidase/Vascular Adhesion Protein-1 (SSAO/VAP-1) Inhibitor (PXS4728A), which is designed to treat several forms of chronic inflammation in humans. The novel therapy will be part of human clinical phase I studies during the first quarter of 2015.
Pharmaxis’ goal is to develop a treatment able to target inflammatory diseases with high unmet clinical need, including Chronic Obstructive Pulmonary Disease (COPD) and Non-Alcoholic Steatohepatitis (NASH), since the SSAO/VAP-1 enzyme is one of the causes for various forms of chronic inflammation in humans, as well as a marker for disease severity in conditions such as atherosclerosis, liver, and kidney inflammation.
“The extensive pre-clinical program performed on PXS4728A has confirmed that it has all characteristics of a successful once a day, oral drug,” explained Pharmaxis CEO Gary Phillips. “It has shown excellent efficacy in several in vivo inflammation models including liver diseases such as NASH and the findings will soon be presented at a scientific conference. In regulatory toxicity studies, PXS4728A has been well tolerated and shown a very good safety profile.”
Since the disease is afflicting more and more people, the company predicts that the drug will enter a market that is estimated to be worth in excess of $35 billion and, if proven effective, it will be the first one to treat NASH. However, other companies are already competing for market share as well, with Genfit’s PPAR agonist, Intercept’s FXR activator, and Gilead’s LOXL2 antibody all due to report on large proof of concept studies in coming months.
“NASH is clearly a growing and valuable market for the future. It’s a disease with several different metabolic manifestations and is attracting a variety of well-funded research strategies,” stated Phillips. “In addition to our anti-inflammatory SSAO/VAP-1 inhibitor program Pharmaxis also has a small molecule anti-fibrotic lysyl oxidase inhibitor (LOXL2) program in pre-clinical development. As lysyl oxidase is the key enzyme in the fibrotic cascade this program has the potential to emerge as a competitor in the fibrosis treatment market, including NASH.”
NASH is a progressive inflammatory disease and can lead to fibrosis and cirrhosis of the liver that already affects about 13 million people in the US and Europe, which researchers think is related to increased rates of obesity and diabetes.
The company is currently looking for development partners for its SSAO/VAP-1 program, at the same time that they are preparing the first clinical trials in humans, scheduled for the beginning of the next year.