Phase 1 Trial of Antibody NI006 for Depletion of Cardiac Transthyretin Amyloid

Transthyretin amyloid (ATTR) cardiomyopathy is a fatal disease caused by misfolded transthyretin. There is currently no treatment available to remove ATTR from the heart and improve cardiac function. A phase 1 trial was conducted to evaluate the safety and effectiveness of NI006, a recombinant human antibody designed to remove ATTR using immune cells. In the trial, 40 patients with ATTR cardiomyopathy and heart failure were randomly assigned to receive either NI006 or a placebo. The patients received intravenous infusions every 4 weeks for 4 months, with increasing doses of NI006. After four infusions, patients entered an open-label extension phase and received additional infusions with gradually increasing doses of NI006. The safety, pharmacokinetics, and cardiac imaging studies were assessed. The use of NI006 was found to be safe, with no serious adverse events related to the drug. The pharmacokinetic profile of NI006 was consistent with that of an IgG antibody, and no antibodies against the drug were detected. Cardiac imaging markers of amyloid load, such as tracer uptake and extracellular volume, appeared to decrease over a period of 12 months with doses of at least 10 mg per kilogram of body weight. Additionally, levels of N-terminal pro–B-type natriuretic peptide and troponin T, which are indicators of heart failure, showed a potential reduction. In conclusion, the phase 1 trial of NI006 for the treatment of ATTR cardiomyopathy and heart failure demonstrated its safety