NI006, a novel monoclonal antibody to treat transthyretin amyloidosis
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Reviewed by Dat Tien Nguyen, B.A, ScM.
Translated by Nhi Phuong Quynh Le, B.A |
Posted on September 04th, 2023
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Transthyretin is used as a transporter to traffick thyroxine and retinol to the liver via the blood and cerebrospinal fluid. However, when misfolded, transthyretin can deposit in the myocardium which causes stiffness. As more transthyretin accumulates, the amyloidosis can impair diastolic function which can lead to arrhythmia and fatal systolic dysfunction. Currently, Tafamidis is the only approved treatment for transthyretin amyloidosis that can effectively reduce the morbidity; however, Tafamidis does not prevent disease progression. A study had been conducted to investigate the possibility of using a novel agent called NI006 to treat transthyretin amyloidosis.
NI006 is a recombinant antibody that can bind to the misfolded transthyretin which can eventually be cleared by phagocytosis. The phase 1 clinical trial included 40 patients who were diagnosed with cardiomyopathy due to transthyretin amyloidosis. To determine the most optimal dose of NI006, the researchers compare the effect of different dose levels of the antibody (0.3, 1, 3, 10, 30, and 60 mg per kilogram of body weight) to placebo. The agents were transfused intravenously and their health was monitored for 12 months. The researchers concluded that NI006 did not result in serious adverse effects; however, larger studies of the subsequent phase needed to be conducted to fully understand the safety profile of NI006. Using MRI and scintigraphy scan, the researchers found that a minimum dose of 10 mg per kilogram of body weight was effective at improving cardiac function.
NI006 is a recombinant antibody that can bind to the misfolded transthyretin which can eventually be cleared by phagocytosis. The phase 1 clinical trial included 40 patients who were diagnosed with cardiomyopathy due to transthyretin amyloidosis. To determine the most optimal dose of NI006, the researchers compare the effect of different dose levels of the antibody (0.3, 1, 3, 10, 30, and 60 mg per kilogram of body weight) to placebo. The agents were transfused intravenously and their health was monitored for 12 months. The researchers concluded that NI006 did not result in serious adverse effects; however, larger studies of the subsequent phase needed to be conducted to fully understand the safety profile of NI006. Using MRI and scintigraphy scan, the researchers found that a minimum dose of 10 mg per kilogram of body weight was effective at improving cardiac function.