Boehringer Ingelheim and pharmaceutical company Pharmaxis have announced that Boehringer Ingelheim has exercised its option and acquired the investigational drug PXS4728A, to develop it for the treatment of the liver-related condition NASH and to prevent its consequences.
PXS4728A is a Semicarbazide-Sensitive Amine Oxidase/Vascular Adhesion Protein-1 (SSAO/VAP-1) Inhibitor discovered by Pharmaxis that 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.
NASH is the progressive form of non-alcoholic fatty liver disease (NAFLD), the most common liver disorder in Western industrialized nations. It is regarded as a major cause of fibrosis and 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 its long term consequences, is considered to make NASH one of the most common causes of advanced liver disorders in coming decades.
Pharmaxis CEO Mr Gary Phillips said, “This is a transformational event for Pharmaxis. With a total potential value in excess of $A750 million, it is a globally competitive deal and significant for the Australian biotech sector. We are delighted that Boehringer Ingelheim, a leader in cardiometabolic research and development, has acquired PXS4728A. Boehringer Ingelheim’s clinical expertise will now be applied to the development of this drug which has the potential to make a real difference in the treatment of diseases with high unmet clinical need.”
Glyn Parkin, Corporate SVP and Metabolism Head at Boehringer Ingelheim commented, “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.”
Pharmaxis will receive an upfront payment of €27.5 million (approximately A$39m) and, subject to the continuing successful development and commercialization of the PXS4728A program, the following payments:
• up to a total of €55 million in development milestone payments tied to the commencement of phase 2 and 3 clinical trials
• up to a total of €140 million in regulatory milestone payments upon filing of applications for marketing approval and receipt of regulatory and pricing approvals for a PXS4728A program product in the major pharmaceutical markets (i.e., USA, EU, and China or Japan) for the first indication
• additional milestone payments similar in total to those set forth above upon achievement of the same development and regulatory milestone events by a PXS4728A program product for a second indication
• earn-out payments on annual net sales of PXS4728A program products at tiered percentages starting in the high single digits
• commercialization milestone payments upon achievement of specified levels of annual net sales of PXS4728A program products
Boehringer will be responsible for all development, regulatory, manufacturing and commercialization activities. Under the agreement, Boehringer has also acquired other SSAO/VAP-1 inhibitor molecules related to PXS4728A and associated patents.