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

11th October 2018

LONDON, 11 October 2018:

Arix Bioscience plc (“Arix”) (LSE: ARIX), a global healthcare and life science company supporting medical innovation, is pleased to note that its Group Business, Pharmaxis Ltd (ASX: PXS), an Australian pharmaceutical research company focused on inflammation and fibrosis, today announced positive results from the Phase 1 clinical trial for the first 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 announcement can be accessed on Pharmaxis’ website at http://www.pharmaxis.com.au/investor-centre/news/ and full text of the announcement from Pharmaxis is contained below.

ENDS

Media Release : 11 October 2018

PHARMAXIS RELEASES POSITIVE RESULTS OF PHASE 1 CLINICAL TRIAL FOR LOXL2 INHIBITOR COMPOUND

Pharmaceutical company Pharmaxis (ASX: PXS) today announced positive results from the Phase 1 clinical trial for the first 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 10mg to 400mg 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 ranging from 100mg to 400mg or placebo for 14 days.

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.

In addition to studying the safety and pharmacokinetic profile, the clinical trial also investigated the degree to which the drug can inhibit the target enzyme LOXL2 which is implicated in several different fibrotic diseases. Importantly, Pharmaxis has been able to demonstrate a large and highly significant inhibition of this enzyme in blood serum for a full 24 hours from a single dose and that daily dosing over a 14‐day period now meets our targeted effect of greater than 80% inhibition at the 400mg dose.



"I’m delighted that the excellent pharmacokinetic parameters and the significant and long lasting inhibition of the target LOXL2 enzyme demonstrated in the single dose stage of the study earlier this year completely translated into the profile we have seen in the multiple dosing study. This drug profile has led to increased interest from major pharmaceutical companies looking for good anti fibrotic programs to acquire. Today’s announcement that enzyme inhibition is further enhanced after daily dosing over 14 days goes a long way to completing the data package on which we will base continuing scientific and commercial discussions with potential partners during the current quarter.”

Gary Phillips, Pharmaxis CEO

The Phase 1 trial for a second Pharmaxis LOXL2 compound being studied has recently completed dosing and will report in the current quarter.

The company’s LOXL2 program compounds are highly selective small molecule inhibitors of LOXL2 that can be administered orally. The ongoing pre‐clinical development program supports the potential of both compounds to treat fibrotic disease in several organs. This support has been enhanced by recent breakthroughs in Pharmaxis proprietary assay technology that have demonstrated target engagement in animal tissue from the pre‐clinical studies as well as serum. 28‐day animal toxicity studies have been completed in two species for both compounds and the remaining 3 month studies are due to be completed later this quarter.

Pharmaxis has previously announced its intention to partner the LOXL2 program after phase 1 studies are complete. Mr Phillips said, “We believe that the best in class LOXL2 inhibition and the availability of two compounds with differentiated pharmacokinetic profiles make this program very attractive and we look forward to concluding a licensing deal with a partner committed to develop the compounds in indications where there remain a lack of treatment options and significant commercial opportunities.

SOURCE: Pharmaxis Ltd, Sydney, Australia

CONTACT: Felicity Moffatt, phone +61 418 677 701 or email felicity.moffatt@pharmaxis.com.au