Boehringer Ingelheim (BI) has exercised its option and acquired the Phase I investigational drug, PXS4728A, from Pharmaxis, an Australian specialty pharmaceutical company, as a treatment for the liver-related condition, non-alcoholic steatohepatitis (NASH).

Under the deal, Pharmaxis will receive an upfront payment of EUR 27.5 million ($31.1 million) and, subject to the continuing successful development and commercialization of the PXS4728A program, the following payments: up to a total of EU 55 million ($62 million) in development milestone payments tied to the commencement of Phase II and III clinical trials; up to a total of EUR 140 million ($158 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., US, EU, 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; and 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. 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, according to BI. 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 is a specialist pharmaceutical company with a portfolio of products at various stages of development and approval. Its product Bronchitol for cystic fibrosis is marketed in Europe and Australia, and a Phase III trial to enable completion of a new drug application for the US market is underway. Its product Aridol for the assessment of asthma is sold in Europe, Australia, and Asia. The company's development pipeline is centred around its expertise in amine oxidase chemistry and includes semicarbazide-sensitive amine oxidase inhibitors NASH and inflammatory disease. The company's head office, research, and manufacturing facilities arein Sydney, Australia.

Source: Boehringer Ingelheim