RARE Daily

AstraZeneca and Neurimmune Enter Global Collaboration and License Agreement on ATTR-CM

January 7, 2022

Alexion, AstraZeneca’s Rare Disease group, has entered into an exclusive global collaboration and license agreement with Neurimmune AG for NI006, an experimental human monoclonal antibody in phase 1b development for the treatment of transthyretin amyloid cardiomyopathy.

Photo: Marc Dunoyer, CEO of Alexion

Photo: Marc Dunoyer, CEO of Alexion

Transthyretin amyloid cardiomyopathy ATTR-CM is a systemic, progressive, and fatal condition that leads to progressive heart failure and 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 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.

Under the terms of the agreement, 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 phase 1b clinical trial on behalf of Alexion, and Alexion will pay certain trial costs. Alexion will be responsible for further clinical development, manufacturing, and commercialization.

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

“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,” said Marc Dunoyer, CEO of Alexion.

The company 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, an ATTR depleter, adds a novel and complementary approach to AstraZeneca and Alexion’s pipeline of investigational therapies focused on amyloidosis and strengthens its broader commitment to addressing cardiomyopathies that can lead to heart failure.

Author: Rare Daily Staff

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