Alexion, AstraZeneca’s Rare Disease group, has closed an exclusive global collaboration and licence agreement with Neurimmune AG for NI006, an investigational human monoclonal antibody currently in phase Ib development for the treatment of 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. Alexion has been granted an exclusive worldwide licence to develop, manufacture and commercialise NI006.

NI006 is an ATTR depleter that specifically targets tissue-deposited, misfolded transthyretin, with the potential to treat patients with advanced ATTR-CM. NI006 adds a novel and complementary approach to AstraZeneca and Alexion’s pipeline of investigational therapies focused on amyloidosis and strengthens the Company’s broader commitment to addressing cardiomyopathies that can lead to heart failure (HF).

Under the terms of the agreement, the upfront payment from Alexion to Neurimmune is US$ 30 million. Alexion will make additional contingent milestone payments of up to $730 million 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 completion of the current phase Ib clinical trial on behalf of Alexion, and Alexion will pay certain trial costs. Alexion will be responsible for further clinical development, manufacturing and commercialisation following the phase Ib trial.

HF is a life-threatening chronic disease that prevents the heart from pumping sufficient levels of blood around the body. HF affects approximately 64 million people worldwide. HF remains as fatal as some of the most common cancers in both men (prostate and bladder cancers) and women (breast cancer). Chronic HF is the leading cause of hospitalisation for those over the age of 65 and represents a significant clinical and economic burden.

AstraZeneca’s ambition is to be the leading company in HF. AstraZeneca is investing in multiple investigational therapies with diverse mechanisms of action to address the spectrum of patient need in this area.

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. As the TTR protein fibrils enlarge, more tissue damage occurs and the disease worsens, resulting in poor quality of life and eventually death.

Worldwide, there are an estimated 300,000-500,000 patients with ATTR-CM; however, many of those patients remain undiagnosed.

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.

Alexion, AstraZeneca Rare Disease, is the group within AstraZeneca focused on rare diseases, created following the 2021 acquisition of Alexion Pharmaceuticals, Inc. As a leader in rare diseases for nearly 30 years, Alexion is focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and commercialisation of life-changing medicines.