Date: 2015-09-22
Type of information: Results
phase: 1
Announcement: results
Company: Pharmaxis (Australia)
Product: PXS4728A
Action
mechanism: protein inhibitor/vascular adhesion protein-1 (VAP-1) inhibitor. PXS4728A is a highly selective oral small molecule inhibitor of vascular adhesion protein-1 that has shown activity in preclinical investigation in non-alcoholic steatohepatitis (NASH). This inhibitor works by blocking leucocyte adhesion and tissue infiltration in inflammatory processes. Pharmaxis has developed it through to phase 1 clinical studies, demonstrating oral bioavailability, long-lasting target inhibition and good tolerability and safety. Boehringer Ingelheim has signed an Option and Asset Purchase agreement for PXS4728A that expires on May 15, 2015.
Disease: NASH (non-alcoholic steatohepatitis)
Therapeutic area: Liver diseases - Hepatic diseases
Country: Australia
Trial details:
Latest
news: * On September 22, 2016, Pharmaxis announced positive results for all primary and secondary endpoints from the phase 1 clinical trial of PXS4728A, a drug that was recently acquired by Boehringer Ingelheim to develop as a treatment for cardiometabolic diseases such as Non-Alcoholic Steatohepatitis (NASH). 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 PXS4728A were trialled. * On January 21, 2015, Pharmaxis announced that it has commenced a Phase 1 clinical trial of PXS4728A investigating its potential to treat inflammatory diseases. The initial stage of the trial is a single ascending dose study being conducted in 48 subjects at an Australian Phase 1 clinical trial centre. If these results are positive, the company will proceed to a multiple ascending dose study in 24 subjects. The trial is expected to report in the third quarter of 2015 and cost approximately $2 million.
Once daily oral dosing of PXS4728A for 14 days at doses between 3 and 10 mg was found to be safe and well tolerated. The data confirmed the high oral bioavailability of PXS4728A and most importantly, showed these low doses are efficacious in inhibiting the enzyme and cause a long lasting inhibition suggesting PXS4728A can be dosed once a day. These positive phase 1 results enable Boehringer Ingelheim to proceed with further development of the program.
