Novo Nordisk invests in amyloidosis pipeline

Novo Nordisk is known as a major player in diabetes treatment, but now the Danish pharma company seems eager to add to this indication. It announced it had agreed to buy up Prothena’s clinical stage antibody PRX004 and broader ATTR amyloidosis programme – and it is willing to dig into its pocket. In addition to the US$100m upfront payment, Dublin-headquartered Prothena is set to receive up to US$1.1bn in development and sales milestone payments.

ATTR amyloidosis is a rare, progressive and fatal disease characterised by the abnormal build-up of amyloid deposits composed of misfolded transthyretin protein in organs and tissues, most commonly the heart and/or nervous system. PRX004 is a phase II-ready anti-amyloid humanised monoclonal antibody designed to deplete the amyloid deposits that are associated with the disease pathology of ATTR amyloidosis. Prothena has completed a phase 1 study with PRX004 in patients with hereditary forms of ATTR, in which PRX004 was found to be safe and well tolerated.

Novo Nordisk said it will initially focus on the clinical development of PRX004 in ATTR cardiomyopathy – a potentially fatal form of ATTR amyloidosis characterised by build-up of amyloid deposits in cardiac tissue. “With its innovative amyloid-depleting mechanism, PRX004 has the potential to offer a novel treatment option for ATTR cardiomyopathy – an often fatal disease with significant unmet medical need,” said Marcus Schindler, CSO at EVP Research and Early Development at Novo Nordisk. “This acquisition is a testament to Prothena’s pioneering work in ATTR amyloidosis and Novo Nordisk’s dedication to advancing new disease-modifying therapies for the benefit of people with cardiovascular diseases which are the world’s leading cause of death.”