A major boost for Sydney-based Pharmaxis after Boehringer Ingelheim exercised its option and acquired global ownership of the company’s investigational anti-inflammatory drug candidate PXS4728A.
The company's share price jumped over 50 per cent on Monday in response to the news, and has now almost tripled since early March.
Pharmaxis will shortly receive an upfront payment of $39 million, with the potential for a further $560 million in development milestone payments, plus sales milestones and royalties.
PXS4728A is directed at the liver related disease Non-Alcoholic Steatohepatitis (NASH), but could also have application in respiratory diseases such as chronic obstructive pulmonary disease.
NASH is the progressive form of non-alcoholic fatty liver disease which is the most common liver disorder in Western industrialised countries.
NASH is regarded as a major cause of cirrhosis of the liver and is an area of high unmet clinical need. The high prevalence of type 2 diabetes and obesity, which can lead to NASH and other non-alcoholic fatty liver diseases, could make NASH the most common cause of advanced liver disease in coming decades and the global market has been estimated to exceed US$3.5 billion by 2025.
PXS4728A is a Semicarbazide-Sensitive Amine Oxidase/Vascular Adhesion Protein-1 (SSAO/VAP-1) Inhibitor, discovered by Pharmaxis and developed successfully into Phase 1 clinical trials that have demonstrated the drug has oral bioavailability, long-lasting target inhibition and good tolerability and safety.
“This is a transformational event for Pharmaxis," said CEO Gary Phillips. "The agreement with Boehringer is an endorsement of our capability to identify and develop breakthrough drug opportunities that are globally competitive. We have also demonstrated that we can execute a business deal that is globally significant and generates value for shareholders.”
Pharmaxis employs 10 researchers in its drug discovery program based at laboratories at its Frenchs Forest headquarters in Sydney. Some funding for the PXS4728A development program has been sourced from the Australian government’s R&D tax incentive.
Pharmaxis said the agreement with Boehringer Ingelheim will also provide the necessary resources to fast-track other projects in its pipeline, including a LOXL2 inhibitor program.
LOXL2 is implicated in several fibrotic diseases including pulmonary fibrosis, liver fibrosis and some cancers.
According to Glyn Parkin, Corporate SVP and Metabolism Head at Boehringer Ingelheim, “We have ambitious strategic goals in diabetes and metabolism and this Phase 1 asset acquisition fits well into our development portfolio. We are pleased to have achieved access to Pharmaxis’ research excellence and innovative approach to treatments for NASH. We will continue to build our portfolio through both internal and external innovation so that we are able to bring much needed medications to the patients we serve.”