AstraZeneca and Neurimmune Collaborate on Treatment for Transthyretin Amyloid Cardiomyopathy

Alexion, AstraZeneca’s Rare Disease group, has closed an exclusive global collaboration and license agreement with Neuroimmune for NI006, the company announced on March 1, 2022. NI006 is an investigational treatment, currently in development to treat transthyretin amyloid cardiomyopathy (ATTR-CM), an underdiagnosed, systemic condition that leads to progressive heart failure and high rate of fatality within four years from diagnosis.

NI006 specifically targets tissue-deposited, misfolded transthyretin, potentially being able to treat patients with advanced ATTR-CM. Heart failure due to ATTR is caused by aging or genetic mutations resulting in misfolded TTR protein and accumulation as amyloid fibrils in the cardiac myocardium. According to the press release, there are an estimated 300,000­–500,000 patients with ATTR-CM worldwide; however, many of those patients remain undiagnosed.

Alexion has been granted an exclusive worldwide license to develop, manufacture, and commercialize NI006. AstraZeneca and Alexion are also investing in multiple other investigational therapies to address cardiomyopathies that can lead to heart failure.