Neurimmune ATTR-CM Asset Nets Potential $700M Plus From AstraZeneca

AstraZeneca rare disease arm Alexion and Neurimmune AG have signed an exclusive global collaboration and licensing deal to develop, manufacture and commercialize NI006 for transthyretin amyloid cardiomyopathy (ATTR-CM). 

NI006 is an investigational human monoclonal antibody that works as an ATTR depleter targeting misfolded transthyretin in tissues. The goal is to treat patients with advanced ATTR-CM, a largely undiagnosed systemic disease leading to progressive heart failure. It also has a high fatality rate of four years from the time of diagnosis. 

Cardiomyopathy from ATTR is due to genetic or aging mutations resulting in misfolded TTR protein and accumulation as amyloid fibrils in the myocardium. Patients with both mutant and wild type TTR protein see a buildup in tissues, including the heart. As the fibrils enlarge, more tissue damage occurs, resulting in poor quality of life and death. There are around 300,000 to 500,000 patients with ATTR-CM worldwide, many of which are undiagnosed. 

“ATTR-CM is characterized by the deposition of misfolded and aggregated transthyretin in the heart. Using our Reverse Translational Medicine technology, we developed a human monoclonal antibody that exclusively targets ATTR to remove amyloid from hearts,” commented Jan Grimm, chief scientific officer of Neurimmune. 

RTM is Neurimmune's proprietary technology platform based on the scientific understanding of high-throughput analyses of human immune responses to disease-related proteins.

NI006 is currently being investigated in a Phase IB clinical trial in ATTR-CM patients in four European countries. Under terms of the partnership, Neurimmune will be responsible for completing the current Phase IB clinical trial on behalf of Alexion, and the latter will take care of certain trial expenses. Alexion will be responsible for further clinical development, manufacturing and commercialization following the study. 

"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," said Marc Dunoyer, chief executive officer of Alexion, said in an earlier statement. 

Alexion will pay $30 million to Neurimmune and make contingent milestone payments of as much as $730 million when certain development, regulatory and commercial milestones are achieved down the line. It will pay low-to-mid teen royalties on net sales from the collaboration. The two companies first announced this collaboration in January 2002. 

News of this investigational ATTR-CM treatment coincides with Intellia Therapeutics and Regeneron's announcement of preliminary data from the Phase I trial of their candidate for ATTR amyloidosis. NTLA-2001 is a CRISPR/Cas9-based therapy candidate that inactivates the TTR gene in liver cells to reduce misfolded TTR protein production. Their initial findings after evaluating different dosage levels showed potentially long-term effectiveness.