Alexion will make an upfront payment of $30m with the potential for additional contingent milestone payments of up to $730m 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.
AstraZeneca says its ambition is to be the leading company in heart failure treatments, expanding from Forxiga today in heart failure with reduced ejection fraction (HFrEF), to the full spectrum including cardiomyopathies.
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.
Improving cardiac function
AstraZeneca’s rare disease group Alexion was created last year with the $39bn acquisition of Alexion Pharmaceuticals.
Commenting on the new agreement with Neurimmune, Marc Dunoyer, CEO, Alexion, 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 underdiagnosed. 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 underserved by existing treatment options.”
Cardiomyopathy due to ATTR is caused by aging or genetic mutations resulting in misfolded TTR protein and accumulation as amyloid fibrils in the cardiac myocardium. In patients with ATTR-CM, both the mutant and wild type TTR protein builds up as fibrils in tissues, including the heart. The presence of TTR fibrils interferes with the normal functions of these tissues. As the TTR protein fibrils enlarge, more tissue damage occurs and the disease worsens, resulting in poor quality of life and eventually death.
Worldwide, there are an estimated 300,000-500,000 patients with ATTR-CM; however, many of those patients remain undiagnosed.
NI006 is an investigational human monoclonal antibody that 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.
The company identifies '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 its pipeline of investigational therapies focused on amyloidosis.
Neurimmune will continue to be responsible for completion of the current Phase Ib clinical trial on behalf of Alexion, and Alexion will pay certain trial costs. Past this, Alexion will be responsible for further clinical development, manufacturing and commercialisation.
The transaction is expected to close following satisfaction of customary closing conditions and regulatory clearances.
