Pharmaxis releases positive results of Phase 1 clinical trial for second LOXL2 inhibitor compound

15th November 2018

LONDON, 15 November 2018

Arix Bioscience plc (“Arix”) (LSE: ARIX), a global healthcare and life science company supporting medical innovation, is pleased to note that Pharmaxis Ltd (ASX: PXS), an Australian pharmaceutical research company focused on inflammation and fibrosis, has announced positive results from the Phase 1 clinical trial for the second of its Lysyl Oxidase Like 2 (LOXL2) inhibitor compounds being developed to treat fibrotic diseases such as Non‐Alcoholic Steatohepatitis (NASH) and Idiopathic Pulmonary Fibrosis (IPF).

Arix led the A$24 million (£13.4 million) financing for Pharmaxis in August, acquiring an 11.1% equity stake. Arix’s Ed Rayner joined the Pharmaxis Board of Directors in September 2018.

The announcement can be accessed on Pharmaxis’ website at and full text of the announcement from Pharmaxis is contained below.

Pharmaxis will host an investor research briefing on Tuesday, 20 November from 10am to 12.00pm (AEDT). The event will provide an overview of the Pharmaxis drug discovery pipeline including the anti-inflammatory drug currently being developed by Boehringer Ingelheim, work in collaboration with the Garvan Institute of Medical Research on an anti-fibrotic LOX inhibitor targeting pancreatic cancer and the anti-fibrotic LOXL2 inhibitor program currently completing phase 1 trials and extended toxicity studies.

This is a unique opportunity to hear directly from the Pharmaxis executive research team along with the perspectives of a committed pharmaceutical partner and a leading medical research institute. A live webcast will be accessible to all investors via the homepage of the Pharmaxis website at and will be available for replay after the event.


Pharmaceutical company Pharmaxis (ASX: PXS) today announced positive results from the Phase 1 clinical trial for the second of its Lysyl Oxidase Like 2 (LOXL2) inhibitor compounds being developed to treat fibrotic diseases such as Non‐Alcoholic Steatohepatitis (NASH) and Idiopathic Pulmonary Fibrosis (IPF).

The double-blind placebo controlled study consisted of two stages. The first single ascending dose stage was conducted in 48 healthy subjects divided into six groups with each taking a single dose ranging from 5mg to 200mg or placebo. The second multiple ascending dose stage was conducted in 24 healthy subjects divided into three groups which each received a single daily dose of either 50mg, 100mg, 200mg or placebo for 14 days.

Repeating the positive results seen in the Phase 1 trial of the first inhibitor compound announced on 10 October 2018, the excellent drug like properties demonstrated in earlier pre-clinical testing were confirmed. There were no adverse safety findings in either the first or second stages of the study and the pharmacokinetic profile showed the expected dose related increases in exposure.

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, achieving the target for this program.

“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 with 24-hour inhibition of the target enzyme from a single daily dose. In a further significant scientific advancement we have also managed to underline the relevance of the program to potential partners by using our proprietary research tools to confirm that our 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. This is slightly delayed versus initial expectations due to the availability of time slots at the contract organisations we use but successful 3-month studies will significantly enhance the LOXL2 program. Following the completion of the data package, 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.”

Pharmaxis CEO Gary Phillip

The Company’s LOXL2 program compounds are highly selective small molecule inhibitors of LOXL2 that can be administered orally and the soon to be completed pre-clinical development program supports the potential of both compounds to treat fibrotic disease in one or more organs.

SOURCE: Pharmaxis Ltd, Sydney, Australia


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