Novo Nordisk signs deal for Prothena's heart drug worth up to $1.2bn

Danish pharma company Novo Nordisk has agreed to purchase Prothena’s clinical stage antibody and broader clinical programme for the rare heart disease ATTR amyloidosis for up to $1.2bn.

Under the terms of the agreement, Novo Nordisk will gain full worldwide rights to the intellectual property and related rights to Prothena’s ATTR amyloidosis business and pipeline.

In return, Prothena will be eligible to receive development and sales milestone payments totalling up to $1.2bn, which includes $100m in upfront and near-term clinical milestone payments.

Initially, Novo will focus on the clinical development of Prothena’s antibody PRX004 in ATTR cardiomyopathy, an underdiagnosed and potentially fatal form of ATTR amyloidosis.

Cardiomyopathy is the most serious of the symptoms seen in patients with hereditary or wild-type ATTR, a condition that means a type of amyloid protein known as transthyretin gets deposited in organs, stopping them from working correctly. In the heart, it causes stiffness in the muscle walls that impairs pumping and can lead to heart failure.

A phase 1 study of PRX004 in patients with hereditary forms of ATTR has already been completed, with the antibody found to be safe and well tolerated in this trial.

“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, 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 that are the world’s leading cause of death,” he added.

With PRX004, Novo will enter the increasingly competitive ATTR amyloidosis market. Elsewhere in the therapy area is Alnylam’s Onpattro (patisiran), which received approval in hereditary ATTR amyloidosis (hATTR) in 2018.

Onpattro is also currently being evaluated in a phase 3 study in ATTR amyloidosis with cardiomyopathy, with results expected in mid-2022 – making it a direct rival to PRX004.