AstraZeneca, Alexion, Neurimmune to Develop Rare Disease Antibody

AstraZeneca Rare Disease and Alexion group recently closed a global collaboration and license agreement with Neurimmune AG for the investigational human monoclonal antibody, NI006.

NI006 is a transthyretin amyloidosis (ATTR) depleter targeting tissue-deposited, misfolded transthyretin. The antibody has the potential to treat patients with advanced ATTR-CM. Alexion gained the exclusive global license to develop, manufacture, and commercialize NI006 in January.

Currently, NI006 is in Phase 1b development to treat transthyretin amyloid cardiomyopathy (ATTR-CM). This systemic condition leads to progressive heart failure and a high fatality rate within four years from diagnosis.

The antibody adds a complementary approach to AstraZeneca, and Alexion’sAlexion’s pipeline of investigational therapies focused on amyloidosis and strengthens the broader commitment to address cardiomyopathies that may lead to heart failure.

Under the agreement terms, Neurimmune will receive an upfront payment of $30 million with the potential for additional contingent milestone payments of up to $730 million. The company will also continue to hold responsibility for completing the current Phase 1b clinical trial on behalf of Alexion.

ATTR-CM is caused by the buildup of abnormal deposits of specific proteins known as amyloid in the body’sbody's organs and tissues, interfering with normal functioning. The disease remains underdiagnosed and underestimated due to a lack of disease awareness and the heterogeneity of symptoms.

In May 2019, FDA approved Vyndaqel and Vyndamax capsules as the first treatment for ATTR-CM.

And in December 2021, AstraZeneca entered into a global development and commercialization agreement with Ionis Pharmaceuticals for ATTR medication, eplontersen.

Eplontersen is a ligand-conjugated antisense investigational medicine currently in Phase 3 clinical trials for ATTR-CM and amyloid transthyretin polyneuropathy (ATTR-PN). The drug reduces the production of transthyretin (TTR) protein to treat both hereditary and non-hereditary forms of TTR amyloidosis (ATTR).

The companies expect the transaction to close in the fourth quarter of 2022, subject to customary closing conditions and regulatory clearances.