Pharmaxis Phase 1 clinical trial proves safety of new LOXL2 drug in fibrotic disease treatment

Pharmaceutical company Pharmaxis (ASX: PXS) has returned positive results from a 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 and idiopathic pulmonary fibrosis.

The double-blind placebo trial confirmed the “excellent drug like properties” demonstrated in earlier pre‐clinical testing, and showed the compound can be well-tolerated by subjects with no adverse safety findings reported.

The trial was split into two ascending dose stages, both of which confirmed the ability for the compound to safely inhibit the target enzyme LOXL2 in blood serum for up to 24 hours from a single dose.

LOXL2 compounds are highly-selective small molecule inhibitors of the LOXL2 enzyme which is implicated in fibrotic diseases.

The compounds have been developed to be administered orally as part of treatment of the disease in a number of organs including lungs, kidneys and heart.

Daily usage of the compound over a 14‐day period was able to meet Pharmaxis’ targeted effect of greater than 80% inhibition at the 400mg dose.

Safety first

Under the parameters of the Phase 1 trial’s first stage conducted earlier this year, 48 healthy subjects were divided into six groups, with each taking a single dose of LOXL2 inhibitor ranging from 10mg to 400mg, or a placebo drug.

The second stage was conducted in 24 healthy subjects divided into three groups, with each receiving a daily dose ranging from 100mg to 400mg, or placebo, for 14 days.

Pharmaxis said the Phase 1 trial demonstrated that the compound can be well-tolerated and the pharmacokinetic profile showed the expected dose-related increases in exposure.

“This drug profile has led to increased interest from major pharmaceutical companies looking to acquire good anti-fibrotic programs,” said chief executive officer Gary Phillips.

“[The results of Phase 1] complete the data package for us to continue scientific and commercial discussions with our potential partners.”

Mr Phillips said the Phase 1 trial for a second Pharmaxis LOXL2 compound being studied has recently completed dosing and results will be reported by year end.

Animal studies

Support for the development of LOXL2 compound has been enhanced by recent breakthroughs in Pharmaxis proprietary assay technology which have demonstrated target engagement in animal tissue from pre‐clinical studies as well as serum.

Animal toxicity studies over 28 days have been completed in two species for both compounds and the remaining three-month studies are due to be completed by year end.

Capital Raising

Earlier this year, Pharmaxis conducted a two-tranche placement with investors to raise $24 million to assist with LOXL2 partnering negotiations.

The funds have also been earmarked for further investment in pre-clinical programs, and to cover general working capital and capital raising costs.

At midday, shares in Pharmaxis were trading 3.45% higher at $0.300.