AstraZeneca’s rare disease business, Swiss company strike deal on heart failure drug

AstraZeneca photo

Alexion, AstraZeneca’s newly acquired rare disease business, entered into an exclusive global collaboration and license agreement with Swiss-based Neurimmune AG for NI006, an investigational human monoclonal antibody heart failure drug.

The drug is Phase Ib development for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM).

Under the agreement, Alexion will be granted an exclusive worldwide license to develop, manufacture and commercialize the drug.

ATTR-CM is a condition that leads to progressive heart failure and high death rate within four years from diagnosis. Its prevalence is thought to be underestimated due to a lack of disease awareness and the nature of symptoms.

Marc Dunoyer, CEO, Alexion, said: “With 30 years of experience in developing medicines for people with rare diseases, Alexion is uniquely positioned to advance innovative science for small patient populations who are frequently underdiagnosed. We look forward to applying this expertise to the development of NI006, which is designed to clear cardiac amyloid fibril deposits with the potential to improve cardiac function for patients living with advanced ATTR-CM, who are currently underserved by existing treatment options.”


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

Alexion will pay Neurimmune an upfront payment of $30 million with the potential for additional milestone payments of up to $730 million upon achievement of certain development, regulatory and commercial milestones, as well as 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.

The transaction is expected to close following completion of customary closing conditions and regulatory clearances.

AstraZeneca’s ambition is to be the leading company in the heart failure space, expanding from its blockbuster drug Forxiga. AstraZeneca is investing in multiple investigational therapies to address the spectrum of patient need in this area.

Worldwide, there are an estimated 300,000-500,000 patients with ATTR-CM3,4; however, many of those patients remain undiagnosed.

Alexion is based in Boston. AstraZeneca has 1,500 employees in Delaware, including a headquarters site near Wilmington. AstraZeneca also has manufacturing and distribution operations near Newark. AstraZeneca is headquartered in Cambridge, UK.