AstraZeneca's Alexion Secures Global Rights to NI006 for ATTR-CM with Up to $730M Deal

On January 7, AstraZeneca announced that its rare disease company, Alexion, had entered into a collaboration with Neurimmune. With a $30 million upfront payment and up to $730 million in milestone payments, Alexion obtained the global exclusive rights to NI006, a monoclonal antibody drug for treating transthyretin amyloid cardiomyopathy (ATTR-CM), which is currently in Phase Ib clinical trials.

NI006 is a recombinant human monoclonal IgG1 antibody specifically targeting misfolded transthyretin (TTR) without affecting the physiological form of TTR. It aims to address the pathology of ATTR-CM by removing amyloid fibril deposits (ATTR) in the heart and has the potential to treat patients with advanced ATTR-CM. In preclinical models, NI006 induces the clearance of pathological ATTR.

ATTR Amyloidosis is a systemic, progressive, and fatal disease where patients suffer from the impairment of normal function in various tissues and organs such as nerves, heart, intestines, eyes, kidneys, and the central nervous system due to the inappropriate formation and aggregation of TTR amyloid deposits. This leads to a variety of clinical symptoms. Among these, ATTR-CM can result in progressive heart failure and death within four years after diagnosis. Globally, it is estimated that there are 300,000-500,000 patients with ATTR-CM; however, the disease remains underdiagnosed, with many patients still undiagnosed and its incidence underestimated.

For patients with various types and severities of amyloidosis, there is a significant unmet medical need that may require multiple mechanisms of action to address. In December 2020, AstraZeneca acquired Alexion, a company specializing in rare diseases, for $39 billion, gaining access to Alexion's complement technology R&D platform and its capabilities in rare disease research. Now, the company is exploring new therapies for rare diseases, including ATTR amyloidosis, through various approaches.

In December 2021, AstraZeneca and Ionis also announced a new strategic collaboration to develop and commercialize the antisense oligonucleotide (ASO) drug eplontersen (IONIS-TTR-LRX), with a total deal value exceeding $3.5 billion. This drug also targets ATTR amyloidosis-related diseases. Eplontersen is currently in Phase III clinical trials for transthyretin amyloid cardiomyopathy (ATTR-CM) and transthyretin amyloid polyneuropathy (ATTR-PN).

Neurimmune is a biopharmaceutical company established in 2006, focusing on developing drug candidates for CNS and protein aggregation-related diseases, including Alzheimer's disease, Parkinson's disease, amyotrophic lateral sclerosis, dementia, and ATTR cardiomyopathy, with the Reverse Translational Medicine™ technology platform.

The Alzheimer's drug Aducanumab, also discovered by this company, was licensed to Biogen in 2007. In 2010, Neurimmune further expanded its collaboration with Biogen to include antibody drugs targeting α-synuclein, tau, and TDP-43. These proteins are believed to play a central role in Parkinson’s disease, macular degeneration, amyotrophic lateral sclerosis, and frontotemporal dementia.

Neurimmune R&D Pipeline

Source: Corporate Official Website