Boehringer licenses liver disease candidate

Boehringer Ingelheim (BI) has licensed a drug for non-alcoholic fatty liver diseases from Australia's Pharmaxis, adding another candidate to its portfolio of drugs for fibrotic diseases.

The German company is paying A$750 million (around $600 million) for worldwide rights to PXS4728A, which acts by interrupting an inflammatory process that underpins non-alcoholic steatohepatitis (NASH), a major cause of fibrosis and cirrhosis of the liver.

PXS4728A is an oral inhibitor of vascular adhesion protein-1 (VAP-1) that blocks the infiltration of white blood cells into tissues and has passed early stage (phase I) clinical testing. BI will take over responsibility for all subsequent clinical trials of the candidate.

After the spectacular success of new therapies for viral hepatitis, companies have started to turn their attention to other forms of liver disease, including non-alcoholic fatty liver diseases that are being driven by rising levels of obesity and diabetes around the world.

The American Liver Foundation (ALF) has estimated that NASH and other fatty liver conditions could affect half of all Americans by 2030, and with no therapeutic options other than weight loss the potential market for pharmacological interventions has been estimated at tens of billions of dollars a year.

BI and Pharmaxis have predicted that the market for NASH alone could rise from a standing start to more than $3.5 billion by 2025.

In acquiring PXS4728A, BI joins the race to develop an effective NASH therapy alongside rivals such as Intercept Pharmaceuticals with its obeticholic acid candidate and Gilead Sciences with simtuzumab – both of which are in mid-stage trials – along with Galectin Therapeutics, Genfit and Raptor Pharmaceuticals.

Merck & Co also joined the party recently by taking a stake in NGM Biopharmaceuticals, whose NP201 candidate is also in testing as a NASH therapy.

The drug also has potential beyond fatty liver disease, however, and BI is interested in exploring its potential for chronic obstructive pulmonary disease (COPD).

That means it complements not only BI's metabolic disease and diabetes portfolio but also sits well alongside Ofev (nintedanib), the German company's recently-launched drug for idiopathic pulmonary fibrosis (IPF).

Ofev was approved for marketing last year on the strength of data revealing that it could slow down disease progression in IPF, which is characterised by scarring of the lungs, declining respiratory function and has a median survival of just two-to-three years after diagnosis.

Boehringer has just reported long-term follow-up data from the TOMORROW study, which reveals that Ofev's benefits on disease progression extend out to 76 weeks, while updated results from the INPULSIS trial backs the drug's safety and tolerability out to 33 months of therapy.