Hundreds of thousands of babies are born around the world each year with sickle cell disease, and the numbers are expected to climb. According to a study, the number of sickle cell disease cases is expected to increase by about 30 percent globally by 2050, with sub-Saharan Africa to be hit the hardest. Currently, there is no cure for the disease, but screening newborn babies, followed by vaccines and antibiotics, can prevent deadly complications and many in developed countries live well into adulthood. However, many countries do not have the necessary resources to provide the lifelong care needed, and sadly as many as 90 percent of children die in the first five years of their life.
The startup Imara is dedicated to developing novel therapeutics for people living with sickle cell disease. Their lead program, IMR-687, is an orally administered phosphodiesterase 9 (PDE9) inhibitor developed to treat the underlying cause of sickle cell disease. In preclinical models, the drug demonstrates a robust safety profile, and reduces red blood cell sickling, red blood cell death, as well as occlusion of blood vessels. PDE9 inhibition also reduce the white blood cell “stickiness” which further reduces the blockage of blood vessels. IMR-687 was discovered by Lundbeck, and has granted Imara an exclusive worldwide license, with Imara being responsible for all future development and commercialization costs for the drug.
Imara has a unique potential to help sickle cell patients, a group of severely ill patients who currently have no treatment options. Sickle cell disease causes lifelong chronic organ damage including pain, stroke, acute chest syndrome, kidney failure and heart failure, and IMR-687 could significantly improve quality of life for thousands all over the world. With numbers of sickle cell disease expected to climb, with projections of about 400,000 babies born each year with the disease by 2050, IMR-687 could provide a desperately needed solution.
The startup launched in 2016 from Cydan Development, Inc, an orphan drug accelerator dedicated to creating therapies that improves the lives of people living with rare genetic diseases. Cydan and Imara recently raised $31 million in a Series A round led by New Enterprise Associates, Pfizer Venture Investments, Lundbeckfond Ventures, Bay City Capital, and Alexandria Venture Investments. Imara now plans to file an IND application with the FDA to advance IMR-687 into human clinical studies.