The company is now ready to enter phase 2 clinical studies for fibrotic diseases such as non‐alcoholic steatohepatitis (NASH), cardiac fibrosis and idiopathic pulmonary fibrosis (IPF).
Adequate safety margin to start phase 2 studies
Both the drug compounds were tested at a range of doses in two species over a 13‐week period to establish the No Observed Adverse Effect Level (NOAEL).
For both compounds, doses that resulted in 85% or greater inhibition of the target enzyme in the phase 1 studies were below the human equivalent NOAEL doses in all toxicity studies and therefore an adequate safety margin to start phase 2 studies of up to three months in length.
With the data package complete, Pharmaxis is now conducting a final series of scientific briefings to potential partners.
Pharmaxis chief executive officer Gary Phillips said: “The results of the toxicity studies complete our scientific package of data for the LOXL2 program.
“It is a high quality and comprehensive data package that is a testament to the expertise and experience of the Drug Discovery and Clinical Development teams at Pharmaxis.
“We have provided the large pharma companies who have been closely monitoring our progress with the latest study results and are now in the process of supporting them to complete their scientific due diligence.
“We are keen to answer remaining scientific questions of potential partners, discuss their proposed clinical development strategies should they acquire or license the program and progress discussions concerning appropriate commercial terms.”
Pharmaxis’ LOXL2 program compounds are highly selective small molecule inhibitors of LOXL2 that can be administered orally and the completed pre‐clinical development program supports the potential of both compounds to treat fibrotic disease in one or more organs.