Pharmaceutical company Pharmaxis (ASX: PXS) has taken another step towards developing a useful treatment for debilitating fibrotic diseases such as Non‐Alcoholic Steatohepatitis (NASH) and Idiopathic Pulmonary Fibrosis (IPF), after announcing positive results from its Phase 1 clinical trial.
The trial investigated the safety profile for the second of its ‘Lysyl Oxidase Like 2’ (LOXL2) inhibitor compounds, via a double‐blind placebo-controlled study consisting of two distinct stages.
The first stage was conducted among 48 healthy subjects divided into six groups with each subject taking a single dose ranging from 5mg to 200mg, or a placebo.
The second stage involved multiple doses, conducted amongst 24 healthy subjects divided into three groups with each receiving a single daily dose of either 50mg, 100mg, 200mg or a placebo for 14 days.
Pharmaxis said there were no adverse safety findings in either the first or second stages of the study and that the pharmacokinetic profile showed the expected dose-related increases in exposure.
Furthermore, the company asserts that its latest results emulated the positive results obtained from a previous Phase 1 trial round which involved its first inhibitor compound, first announced last month.
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Significant target engagement of the LOXL2 enzyme by both compounds has now been demonstrated in blood serum for a full 24 hours from a single dose over a 14‐day period, with the second compound achieving more than 85% inhibition over 24 hours from a 100mg daily dose – thereby achieving the pre-set target set for this program by Pharmaxis.
This morning’s positive clinical trial news means Pharmaxis is now in with a chance with attracting the attention of larger pharma companies in the hope of commercialising its technology via a licensing agreement sometime next year.
“Several large pharma companies are interested in the Pharmaxis program where both of our LOXL2 inhibitors have now successfully completed Phase 1 studies and demonstrated a best in class profile,” said Gary Phillips, CEO of Pharmaxis.
In a further significant scientific advancement, Mr Phillip said that Pharmaxis has managed to underline the relevance of the program to potential partners by using its proprietary research tools to confirm its compounds “directly inhibit the activity of the raised levels of LOXL2 seen in diseased tissue from NASH and IPF animal models.”
“The only remaining elements necessary to finalise the data package that companies are now conducting diligence on, are the 3‐month toxicity studies on both compounds which are due to report later this quarter,” he said.
“Pharmaxis intends to conduct a final series of scientific briefings to potential partners before moving to commercial partnering discussions to secure a comprehensive licensing agreement in 2019,” Mr Phillips said.
As part of its mission to publicise its findings to both market analysts and the wider scientific community, Pharmaxis has scheduled an investor research briefing for 20 November, expected to feature presentations by a Boehringer Ingelheim global project manager and a research leader from the Garvan Institute of Medical Research.
According to the pharmaceutical developer, the event will provide an overview of the Pharmaxis drug discovery pipeline including anti‐inflammatory drug currently being developed by Boehringer Ingelheim, but also, the work currently ongoing in collaboration with the Garvan Institute of Medical Research on an “anti‐fibrotic LOX inhibitor” targeting pancreatic cancer.
The briefing is also expected to feature news regarding the anti‐fibrotic LOXL2 inhibitor program currently completing phase 1 trials and extended toxicity studies.