Alexion will obtain an exclusive licence to develop, manufacture and market NI006, and will pay $30m upfront, and up to $730m in milestone payments

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AstraZeneca’s Discovery Centre overhead view. (Credit: AstraZeneca)

AstraZeneca, through its rare disease division Alexion, has signed an exclusive global collaboration and licence deal with Swiss firm Neurimmune to develop the latter’s potential treatment NI006.

Under the terms of the agreement, Alexion will obtain an exclusive licence to develop, manufacture and commercialise NI006, and will make an upfront payment of $30m.

Neurimmune is eligible to receive additional milestone payments of up to $730m subject to achieving certain development, regulatory and commercial milestones.

The Swiss biotechnology company will also receive low-to-mid teen royalties on net sales of any drug developed under the collaboration.

In addition, Neurimmune is responsible for completing the currently ongoing Phase 1b clinical trial, while Alexion will fund certain trial costs.

AstraZeneca’s rare disease-focused subsidiary will conduct further clinical development, manufacturing and commercialisation activities after the Phase 1b trial.

NI006 is an investigational human monoclonal antibody, currently in Phase 1b trials for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM).

ATTR-CM is an underdiagnosed, systemic condition that may lead to progressive heart failure and elevated fatality rate within four years from diagnosis.

It is caused due to ageing or genetic mutations that result in misfolded TTR protein and accumulation as amyloid fibrils in the cardiac myocardium.

Patients with ATTR-CM will experience a build-up of TTR protein as fibrils in tissues, including the heart, which interferes with the normal functions of the tissues.

NI006 is an ATTR depleter that specifically targets tissue-deposited, misfolded transthyretin and removes amyloid fibril deposits in the heart of ATTR-CM patients.

AstraZeneca said that the drug brings a novel approach to its pipeline of amyloidosis-focused therapies and strengthens its commitment to addressing cardiomyopathies.

Alexion, AstraZeneca Rare Disease is a business division within AstraZeneca, focused on rare diseases. It was created last year, after the acquisition of Alexion Pharmaceuticals.

Last year, AstraZeneca has partnered with US-based biotech company Ionis Pharmaceuticals to develop and commercialise the latter’s eplontersen.

Previously called IONIS-TTR-LRX, eplontersen is an investigational ligand-conjugated antisense (LICA) medicine, designed to treat TTR amyloidosis (ATTR).