According to a story from BioPortfolio, Alexion Pharmaceuticals, Inc. and Eidos Therapeutics, Inc., have recently struck an agreement related to the development and marketing of an experimental treatment for hATTR amyloidosis in the country of Japan. This therapy is currently known as AG10 and it is undergoing evaluation for various complications caused by this form of amyloidosis.
About hATTR Amyloidosis
HATTR Amyloidosis is a disease which is characterized by the build up amyloid fibrils in body tissue. Amyloid fibrils are a type of abnormal protein. The disease is caused by a heritable genetic mutation. Symptoms of the illness include swelling, heart failure, irregular heartbeat, shortness of breath, fatigue, weight loss, easy bruising and bleeding, stroke, lung problems, enlarged liver, and changes in skin color. Treatment approaches for hATTR amyloidosis includes liver transplant; This can actually cure the disease, but the procedure carries substantial risks and side effects. This form of amyloidosis has a better prognosis than some of the more common types with some patients surviving for over a decade. It is estimated to affect around 50,000 people worldwide. To learn more about amyloidosis, click here.
AG10 is a small molecule drug that is precisely tailored to address the underlying disease mechanism in hATTR amyloidosis: the misfolded transthyretin (TTR) protein. AG10 is designed to treat the disease by binding to the misfolded proteins and stabilizing them. The drug is currently undergoing phase 3 clinical trials for ATTR cardiomyopathy. This is a lethal heart disease which occurs as the result of excess buildup of transthyretin in the heart. There are also plans for an additional phase 3 study that will test the drug against ATTR polyneuropathy (peripheral nervous system damage caused by protein buildup).
Prior study has indicated that AG10 has substantial potential in treating this disease. In a phase 2 study of amyloidosis patients, the drug brought down TTR levels in the blood (a critical indicator of disease progression and survival) and achieved almost complete stabilization of the protein.
With Alexion’s prior experience with commercialization in Japan, the deal will allow AG10 to impact a greater number of patients if the experimental medication is ultimately approved.