Rare Daily Staff

Branching out from its traditional focus in diabetes and obesity, Danish biopharmaceutical Novo Nordisk is moving into rare disease with the acquisition of Prothena’s clinical stage antibody PRX004 and broader ATTR amyloidosis program for up to $1.2 billion.

ATTR amyloidosis is a rare, progressive, and fatal disease in which mutations in the TTR gene cause the liver to produce misshapen transthyretin protein that misfold and build up as amyloid in the body, causing serious complications in multiple tissues, including the heart, nerves and digestive system.

PRX004 is a phase 2 ready anti-amyloid immunotherapy 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.

“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 and executive vice president, Research and Early Development at Novo Nordisk.

Novo Nordisk will initially focus on the clinical development of PRX004 in ATTR cardiomyopathy, an underdiagnosed and potentially fatal form of ATTR amyloidosis characterized by the build-up of amyloid deposits in cardiac tissue.

Under the terms of the purchase agreement, Novo Nordisk acquires Prothena’s wholly owned subsidiary and gains full worldwide rights to the intellectual property and related rights of Prothena’s ATTR amyloidosis business and pipeline. Prothena is eligible to receive development and sales milestone payments totaling up to $1.2 billion, including 100 million dollars in upfront and near-term clinical milestone payments.

“We are confident that Novo Nordisk will leverage its extensive expertise in developing treatments for those affected by cardiovascular diseases to advance this promising potential treatment to patients on an expedited timeline,” said Hideki Garren, chief medical officer of Prothena. “With Novo Nordisk’s commitment to further develop PRX004 in ATTR cardiomyopathy, Prothena will continue to focus on our mission to advance our robust portfolio designed to address rare peripheral amyloid and neurodegenerative diseases.”

Photo: Hideki Garren, chief medical officer of Prothena

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