The pharmaceutical research company has expanded the scientific program on LOXL2 inhibitors to maximise its value to potential partners.
Pharmaxis has also substantially increased its interest in the program in return for a payment of £5 million to Synairgen.
Collaboration has progressed pre-clinical stage
The companies have been working on the pre-clinical stage of an antifibrotic Lysyl Oxidase type 2 (LOXL2) inhibitor program.
The LOXL2 enzyme is fundamental to the progression of fibrosis in the liver disease NASH.
This also applies to fibrotic disease in organs such as the heart, kidney, and lung where idiopathic pulmonary fibrosis (IPF) remains a high unmet need.
The move to full control has resulted in Pharmaxis doubling the program scope to encompass two lead candidates with unique properties.
It intends to take these to the end of phase I trials with additional toxicology data delivered in parallel to enable them to be phase II ready by mid‐2018.
Full funding responsibility
Pharmaxis has full control and funding responsibility of the ongoing partnering process and aims to conclude a deal after phase I studies have reported.
The company has significantly increased its share of any partnering deal for the LOXL2 program in fibrotic diseases to over 80%.
Synairgen has retained a reduced but fixed percentage share of all future partnering revenues.
The collaboration began in August 2015 with the aim of developing and partnering a small molecule inhibitor of the LOXL2 enzyme at the end of phase I trials.
Two compounds identified
An extensive pre‐clinical program identified two compounds that have all the characteristics of successful once a day, oral drugs.
These show excellent efficacy in several different in vivo fibrosis models.
In regulatory toxicity studies, the two compounds have been well‐tolerated and shown good safety profiles.
Gary Phillips, chief executive officer, said: “The collaboration with Synairgen in the program’s pre‐clinical stage has served its purpose very well.
“However, the increased interest in NASH together with Pharmaxis’ progress over the last few years, its track record of delivering significant partnering deals and enhanced financial position, means we are now in a position to develop this product.
“This will be done under our control with our own resources so that we can maximise shareholder value.”
Discussions with pharma companies
The LOXL2 inhibitor program has been the subject of discussions with large pharma companies since the beginning of 2016.
Pharmaxis expects the interest to intensify as the two compounds progress through phase I clinical trials.
In parallel with these trials, Pharmaxis will enable scientific due diligence of the program by select large pharma companies interested in subsequent partnering discussions.
Phillips said: “To realise the opportunity in a competitive market we need to deliver convincing proof of concept in multiple disease models.
“This includes a phase I toxicology data package that both clears the phase I hurdle and makes this asset phase II ready.”
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.