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Pharmaxis receives positive results from clinical trial of treatment for fibrotic diseases

Published: 23:30 14 Nov 2018 GMT

Scientists in a Pharmaxis laboratory
Several large pharmaceutical companies are showing interest in the program

Pharmaxis Ltd (ASX:PXS) has received positive results from a phase I clinical trial for the second of its LOXL2 inhibitor compounds which are being developed to treat fibrotic diseases.

These include the liver disease known as non-alcoholic steatohepatitis (NASH) and the lung disease idiopathic pulmonary fibrosis (IPF).

READ: Pharmaxis receives positive results from LOXL2 clinical trial

Repeating the positive results seen in the phase I trial of the first inhibitor compound, released last month, the excellent drug-like properties demonstrated in earlier pre-clinical testing were confirmed.

There were no adverse safety findings in the first or second stages of the study and the pharmacokinetic profile showed the expected dose-related increases in exposure.

Both inhibitors complete phase I studies

Pharmaxis CEO Gary Phillips said: “Several large pharma companies are interested in the program where both of our LOXL2 inhibitors have now successfully completed phase I 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.”

Two-stage study

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.

Significant target engagement of the LOXL2 enzyme by both compounds has been demonstrated in blood serum for a full 24 hours from a single dose over a 14-day period.

The second compound achieved more than 85% inhibition over 24 hours from a 100mg daily dose, thus achieving the target for this program.

READ: Pharmaxis gains Arix Bioscience Plc as a new substantial shareholder

Pharmaxis’ LOXL2 program compounds are highly selective small molecule inhibitors of LOXL2 that can be administered orally.

The soon to be completed pre-clinical development program supports the potential of both compounds to treat fibrotic disease in one or more organs.

Remaining elements

Phillips said: “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,” he added.

Portfolio of products

Pharmaxis is an Australian pharmaceutical research company focused on inflammation and fibrosis with a portfolio of products at various stages of development and approval.

Its product Bronchitol® for cystic fibrosis is marketed in Europe, Russia and Australia while Aridol®, for the assessment of asthma, is sold in Europe, Australia and Asia.

The company’s development pipeline is centred on its expertise in amine oxidase chemistry and includes a series of Lysyl Oxidase Inhibitors under clinical development targeting fibrotic diseases of the heart, kidney, liver and lung.

Pharmaxis Ltd expects review of Bronchitol NDA to be completed by mid-2020

Pharmaxis Ltd (ASX:PXS) (OTCMKTS:PXSLY) (FRA:UUD) CEO Gary Phillips speaks to Proactive's Andrew Scott after it was announced its US licensee Chiesi Farmaceutici SpA filed a resubmission to the FDA for Bronchitol® . The move addresses issues raised by the US Food and Drug Administration (FDA)...

on 6/5/20