Editor’s Note: This interview was conducted at #Bio2018. Please pardon the background noise.

James McArthur, PhD, Founder of Imara and President, R&D of Imara discusses the development of IMR-687 for the treatment or rare hemoglobinopathies.

Dr. McArthur was a founder of Cydan’s spinout developing therapies for Niemann Pick type C, Vtesse, which was acquired by Sucampo, and serves as a member of the Board of Directors of Nightstar Therapeutics, a public company focused on developing gene therapies for rare retinal diseases. Imara Inc., is dedicated to developing novel therapeutics for people living with sickle cell disease and other hemoglobinopathies. Sickle cell disease is a rare, genetic blood disease that causes red blood cells to sickle and become damaged, activating immune cells and blocking blood flow in capillaries, injuring many organs and causing daily pain. Imara is developing IMR-687, a highly selective, potent small molecule inhibitor of phosphodiesterase-9 (PDE9i), to treat patients with sickle cell disease.

IMR-687 was identified and selected specifically to treat patients with sickle cell disease by both reducing red blood cell sickling and blockage of blood vessels that are underlying causes of the pathology of sickle cell disease. IMR-687 is a highly potent, selective inhibitor of phosphodiesterase-9 (PDE9i) in blood cells. IMR-687 was developed to target the same biochemical pathway as hydroxyurea, a chemotherapeutic agent, but without its safety issues.