AstraZeneca’s rare disease group Alexion targets amyloidosis in deal worth potential $760m with Neurimmune AG

Alexion, AstraZeneca’s rare disease group, will $30million upfront and potential milestone payments of up to $730m to Neurimmune AG after entering into an exclusive global collaboration and licence agreement for NI006.

This investigational human monoclonal antibody is in Phase Ib development for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM), which leads to progressive heart failure and a high rate of fatality within four years of diagnosis.

Alexion will pay low-to-mid teen royalties on net sales of any approved medicine.

CEO Marc Dunoyer 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 under-diagnosed.

“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 under-served by existing treatment options.”

Neurimmune will complete the current Phase Ib clinical trial and Alexion, which will pay certain trial costs, will be responsible for further clinical development, manufacturing and commercialisation.

Alexion said there is a significant unmet medical need for patients with various types and levels of severity of amyloidosis that may require multiple mechanisms of action to address those needs.

NI006 is an ATTR depleter that adds a novel and complementary approach to AstraZeneca and Alexion’s pipeline of investigational therapies focused on amyloidosis.

NI006 specifically targets misfolded transthyretin and is designed to address the pathology of ATTR-CM by enabling removal of amyloid fibril deposits in the heart.

Meanwhile, Cambridge-based AstraZeneca recently agreed a deal worth a potential $3.585bn with Ionis Pharmaceuticals for Eplontersen, which is in Phase III clinical trials for transthyretin amyloidosis.

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