Sydney-based Pharmaxis (ASX:PXS) has announced positive results for all primary and
secondary endpoints from the phase 1 clinical trial of PXS‐4728A.
The drug was recently acquired by global pharmaceutical company Boehringer Ingelheim to develop as a treatment for cardiometabolic diseases such as Non-Alcoholic Steatohepatitis (NASH).
In May, Pharmaxis announced the deal with Boehringer Ingelheim, under which it received an upfront payment of $39 million, with the potential for a further $560 million in development milestone payments, plus sales milestones and royalties.
PXS‐4728A is a highly selective inhibitor of an enzyme and adhesion protein (SSAO/VAP‐1 encoded by the AOC3 gene) which reduces inflammation and oxidative stress.
According to the company, further to the phase 1a single ascending dose stage that reported positive results in April 2015, this phase 1b multiple ascending dose stage was conducted in 24 healthy subjects divided into three groups with each taking active or placebo once a day for 14 days.
"Three different dosages of PXS‐4728A were trialled," it said.
The data confirmed the high oral bioavailability of PXS‐4728A, and showed low doses are efficacious in inhibiting the enzyme and cause a long lasting inhibition, suggesting PXS‐4728A can be dosed once a day, Pharmaxis said in a statement.
The company confirmed that the positive results will enable Boehringer Ingelheim to proceed with further development of the program.
“NASH is a disease that is becoming more prevalent and there is a clear need for more effective therapies," said Pharmaxis CEO Mr Gary Phillips. "PXS‐4728A inhibits an enzyme which has been highlighted in independent peer reviewed publications as an excellent target to treat NASH and this first human trial of the drug confirms its strong pre‐clinical profile translates into human studies."
He continued, "It is rare to be able to demonstrate effective target engagement in a phase 1 study so the fact that the long lasting enzyme inhibition seen in the phase 1a study was reinforced when given once a day for 14 days adds to our confidence in PXS‐4728A. We now look forward to the next value appreciating steps as the clinical development program proceeds with Boehringer.”
The company's share price rose 15 per cent in response to the announcement.