Imara Announces FDA Fast Track Designation for IMR-687 for the Treatment of Sickle Cell Disease
May 29, 2019
Cambridge, Mass., May 29, 2019 – Imara, Inc., a clinical-stage biopharmaceutical company focused on sickle cell disease and other hemoglobinopathies, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to IMR-687, the company’s lead product candidate for the treatment of sickle cell disease. IMR-687 is currently being evaluated in a multi-national Phase 2a clinical trial in adult patients.
“We believe that IMR-687 has the potential to transform outcomes for people living with sickle cell disease. It has been designed as an oral, once-daily therapy with a dual mechanism of action to address both red blood cell and white blood cell pathologies associated with the disease,” said Rahul D. Ballal, Chief Executive Officer of Imara. “The Fast Track designation granted by the FDA for IMR-687 is designed to allow us to advance this novel, investigative therapy more rapidly through increased opportunities for dialogue and collaboration with the Agency.”
Fast Track designation aims to facilitate the development and expedite the review of drugs to treat serious conditions where an unmet medical need exists, with the goal of getting important new therapeutics to patients earlier.
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.
Sickle cell disease represents a critical unmet medical need globally, as a rare disease in many parts of the world including in the United States and as an endemic condition in certain African countries.
IMR-687 has been designed to address the underlying pathology of sickle cell disease. An orally-administered, highly potent and selective phosphodiesterase 9 (PDE9) inhibitor, IMR-687 has the potential to be a 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. In a Phase 1 clinical trial in healthy volunteers, IMR-687 was demonstrated to be well-tolerated. IMR-687 has been granted U.S. Orphan Drug Designation, U.S. Rare Pediatric Designation and Fast Track Designation by the Food and Drug Administration (FDA).
Imara, Inc. is dedicated to developing novel therapeutics for patients with sickle cell disease and other hemoglobinopathies. Imara is currently developing IMR-687, a highly selective, potent small molecule inhibitor of PDE9, to treat patients with sickle cell disease. IMR-687 has been specifically designed to treat patients with sickle cell disease by both reducing red blood cell sickling and adherence of white blood cells to the blood vessel wall. These combined effects may lead to a lower occurrence of blockage of blood vessels that are underlying causes of the pathology of sickle cell disease. IMR-687 successfully completed a Phase 1 study in healthy volunteers and is currently being evaluated in a multi-national Phase 2a study in adult patients with sickle cell disease. Imara expects to broaden its development of IMR-687, with clinical trials of IMR-687 planned in pediatric patients with sickle cell disease as well as in patients with beta thalassemia, which is a group of genetic blood disorders that often leads to chronic anemia and other serious complications. For more information about Imara, visit www.imaratx.com.