«Our cardiomyopathy program relies heavily on our experience around the biology of amyloid removal in the central nervous system»

Christoph Hock, CMO

ATTR Cardiomyopathy

Amyloid transthyretin (ATTR)-cardiomyopathy is an age-associated disease of the heart characterized by intramyocardial deposition of misfolded and aggregated transthyretin. The accumulation of amyloid results in increased ventricular wall thickness and severely impacts heart function and survival. The disease manifests predominantly in men ≥ 60 years of age and can be diagnosed using echocardiography, scintigraphy and biopsies. The prevalence of the disease is currently unknown despite the fact that diagnosis tools are widely accessible. It is estimated that today only 1% or less of the patients are being diagnosed.

Using Neurimmune’s Reverse Translational Medicine technology, we performed high-throughput immune repertoire analyses of healthy elderly subjects for ATTR-reactive B-cell memory. We selected NI006, a recombinant human monoclonal IgG1 antibody that exclusively targets with high affinity the disease-associated amyloid conformation but not physiological forms of transthyretin. NI006 targets both wild-type ATTR as well as ATTR mutants that are linked to hereditary forms of ATTR cardiomyopathy and ATTR polyneuropathy. NI006 induces the clearance of pathological ATTR in preclinical models.