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11 January 2022BiotechnologyMuireann Bolger

AstraZeneca inks $730m licensing deal for rare heart disease treatment

Alexion, AstraZeneca’s rare disease group, has entered into an exclusive licensing agreement with Neurimmune AG for NI006, an antibody in development for the treatment of the heart condition, transthyretin amyloid cardiomyopathy (ATTR-CM).

Under the agreement announced by AstraZeneca on January 7, Alexion will be granted an exclusive worldwide licence to develop, manufacture and commercialise NI006.

ATTR-CM is a systemic, progressive and fatal condition that leads to progressive heart failure and a high rate of fatality within four years from diagnosis.

It remains underdiagnosed and its prevalence is thought to be underestimated due to a lack of disease awareness and the heterogeneity of symptoms.

NI006 is an investigational human monoclonal antibody designed to directly address the pathology of ATTR-CM by enabling removal of amyloid fibril deposits in the heart.

Alexion will pay Neurimmune an upfront payment of $30 million with the potential for additional contingent 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 clinical trial on behalf of Alexion, and Alexion will pay certain trial costs. Alexion will be responsible for further clinical development, manufacturing and commercialisation.

Marc Dunoyer, chief executive officer of 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-CM3, however, many of those patients remain undiagnosed.

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