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.Read full media release - pdf
Pharmaxis 2018 Directors' Report and Annual Financial StatementsRead report – pdf