ATTR amyloidosis is a rare, progressive and fatal disease characterized by the abnormal build-up of amyloid deposits and accumulation of proteins in the body’s organs and tissues, most commonly the heart and/or nervous system.

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.

Under the terms of the transaction, Prothena is eligible to receive development and sales milestone payments totaling up to $1.2 billion, including $100 million in upfront and near-term clinical milestone payments.

Initially, Novo Nordisk will focus on the clinical development of PRX004 in ATTR cardiomyopathy - an underdiagnosed and potentially fatal form of ATTR amyloidosis characterized 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, chief scientific officer, European vice president 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."

Hideki Garren, Prothena’s chief medical officer, added that the company “will continue to focus on our mission to advance our robust portfolio designed to address rare peripheral amyloid and neurodegenerative diseases.”

Prothena was established in December 2012 when it was spun out of US drugmaker Elan with a substantial portion of the former parent’s drug discovery business. The late-stage clinical company has a pipeline that includes both wholly-owned and partnered programs being developed for treating diseases including AL amyloidosis, Alzheimer’s disease, Parkinson’s disease and several other neurodegenerative diseases.

Author: Elaine Burridge, Freelance Journalist