Pharmaxis Ltd (ASX:PXS) and its collaborator UK biotechnology company Synairgen plc (AIM: SNG) have completed the pre-clinical development stage of their antifibrotic Lysyl Oxidase type 2 (LOXL2) inhibitor program.
This allows the first compound to commence human clinical phase 1 studies in the December quarter 2017.
As part of the collaboration, Synairgen is funding preclinical toxicology work and phase 1, and utilising its BioBank human tissue models technology platform.
Gary Phillips, chief executive officer for Pharmaxis, commented:
"The extensive pre-clinical program performed on our program compounds has confirmed that they have all the characteristics of a successful once a day, oral drug.
"They have shown excellent efficacy in several different in vivo fibrosis models including fibrosis of the liver, lung, kidney and heart.
"These findings have been the subject of presentations at a number of international scientific conferences and more data will be presented at similar upcoming events as the phase 1 studies proceed.
"In regulatory toxicity studies, our compounds have been well tolerated and shown a good safety profile."
Pharmaxis has therefore consolidated its position as a significant competitor in the NASH market as the LOXL2 inhibitor joins its SSAO inhibitor in the clinic.
What is NASH
NASH is a major cause of fibrosis and cirrhosis of the liver and is an area of high unmet medical need with no treatments currently available.
Current research has reported the prevalence of NASH to range from 1.5% to 6.45%, a number twice as high as 20 years ago and the market has been forecast by Deutsche Bank to be worth in excess of $35 billion by 2025.
The potential for the LOXL2 program to be developed as a therapeutic for NASH positions Pharmaxis as a key player in this important and growing disease.
In addition to its anti-fibrotic LOXL2 program, Pharmaxis also has an ongoing interest in the anti-inflammatory SSAO/VAP-1 inhibitor PXS-4728A with which its partner Boehringer has just commenced a NASH phase 2 study.
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Phillips added: "NASH is clearly a growing and valuable market for the future.
"It’s a disease that is attracting a variety of well-funded research strategies and it is anticipated that a combination approach that tackles the metabolic, inflammatory and fibrotic drivers of NASH, will be necessary in order to reduce the long term effects of this disease.
"As lysyl oxidase type 2 is the key enzyme in the fibrotic cascade we anticipate that our LOXL2 program will be attractive to many of the multinational pharmaceutical companies that are building a portfolio approach to treating NASH and some other fibrotic diseases such as IPF."