Announced in January, Alexion has been granted an exclusive worldwide licence to develop, manufacture and commercialise NI006: an ATTR depleter that specifically targets tissue-deposited, misfolded transthyretin, with the potential to treat patients with advanced ATTR-CM.
NI006 is currently in Phase Ib development.
Clinical development
NI006 adds a ‘novel and complementary approach’ to AstraZeneca and Alexion’s pipeline of investigational therapies focused on amyloidosis and strengthens its broader focus on cardiomyopathies that can lead to heart failure, according to the company.
Alexion is making an upfront payment to Neurimmune of $30m; and will pay additional contingent milestone payments of up to $730m upon achievement of certain development, regulatory and commercial milestones. It will also pay low-to-mid teen royalties on net sales of any approved medicine resulting from the collaboration.
Neurimmune will continue to be responsible for the current Phase Ib clinical trial on behalf of Alexion, while Alexion will pay certain trial costs.
Alexion will take on responsibility for further clinical development, manufacturing and commercialisation following the Phase Ib trial.
ATTR-CM is a systemic condition that leads to progressive heart failure and high rate of fatality within four years from diagnosis. There are an estimated 300,000 – 500,000 patients globally, although many patients remain undiagnosed.
Cardiomyopathy due to ATTR is caused by aging or genetic mutations resulting in misfolded TTR protein and accumulation as amyloid fibrils in the cardiac myocardium. In patients with ATTR-CM, both the mutant and wild type TTR protein builds up as fibrils in tissues, including the heart. The presence of TTR fibrils interferes with the normal functions of these tissues.
NI006 is an investigational human monoclonal antibody that specifically targets misfolded transthyretin and is designed to directly address the pathology of ATTR-CM by enabling removal of amyloid fibril deposits in the heart, with the potential to treat patients with advanced ATTR-CM.