CAMBRIDGE, Mass.--(BUSINESS WIRE)--Imara Inc., a biotechnology company dedicated to developing novel therapeutics for patients with sickle cell disease and other hemoglobinopathies, today announced the first healthy volunteer has been dosed in a phase 1 clinical study evaluating the safety and tolerability of the company’s lead product candidate, IMR-687.
“More than 160,000 individuals are living with sickle cell disease in the United States and Europe with many more in Africa and Asia; yet, there remains a serious medical need facing those whose lives are burdened by this devastating disease,” said James McArthur, Ph.D., founder, president, and chief executive officer of Imara. “Initiating this first study in humans is a critically important milestone as we work to advance new therapies for people living with sickle cell disease.”
IMR-687 was specifically designed to address the underlying pathology of sickle cell disease. An orally-administered, highly potent and selective phosphodiesterase 9 (PDE9) inhibitor, IMR-687 is a potentially disease-modifying therapeutic for sickle cell disease as well as other hemoglobinopathies. Pre-clinical data demonstrate IMR-687 reduces both the sickling of red blood cells and blood vessel occlusion that cause debilitating pain, organ damage, and early mortality in affected patients.
About Sickle Cell Disease
Sickle cell disease is a rare, genetically inherited condition that alters hemoglobin, the protein in red blood cells that transports oxygen throughout the body. The altered hemoglobin distorts red blood cells into a sickle, or crescent, shape. Painful episodes can occur when sickled red blood cells, which are stiff and inflexible, get stuck in small blood vessels. These episodes deprive tissues and organs of oxygen-rich blood and can lead to vaso-occlusive crisis (VOC), acute chest syndrome (ACS), and permanent damage to organs including the liver, spleen, kidney and brain.
Imara Inc., a Cydan Development company, is dedicated to developing novel therapeutics for patients with sickle cell disease. Imara is developing IMR-687, a highly selective, potent small molecule inhibitor of PDE9, to treat patients with sickle cell disease. The company was launched following an 18-month diligence and de-risking scientific collaboration between orphan drug accelerator Cydan Development and H. Lundbeck A/S with initial funding from life science investors NEA, Pfizer Venture Investments, Lundbeckfond Ventures, Bay City Capital and Alexandria Venture Investments.