April 14, 2016
By Alex Keown, BioSpace.com Breaking News Staff
CAMBRIDGE, Mass. – Orphan drug accelerator Cydan Development, Inc. launched Imara Inc., its second orphan drug company launched in the past year.
Imara launches with $31 million and a ready-to-test treatment for sickle cell disease and other hemoglobinopathies. Imara’s IMR-687 is an orally-administered, selective phosphodiesterase 9 (PDE9) inhibitor that was developed to treat the underlying causes of sickle cell disease. Pre-clinical data shows IMR-687 reduces both the sickling of red blood cells and blood vessel occlusion. IMR-687 also demonstrates a robust pre-clinical safety profile, the company said.
In addition to announcing the launch of the company, Imara said it plans to file an Investigational New Drug application with the U.S. Food and Drug Administration to develop IMR-687 for sickle cell disease in 2016.
IMR-687 was discovered by H. Lundbeck A/S, which granted Imara worldwide license for the drug in exchange for undisclosed milestone payments and a minority stake in the new company.
Sickle Cell Disease describes a group of various blood disorders characterized by sickling of red blood cells and the occlusion or blockage of small blood vessels by the rigid, sickle-shaped red blood cells. If the blood vessels are blocked, the areas of the body not receiving proper blood amounts can be damaged. Currently there is no cure for sickle cell disease. There are about 160,000 people affected by the disease in the United States and Europe.
James McArthur, the new chief executive officer of Imara, said over the past year his team of scientists and researchers has produced pre-clinical data demonstrating that IMR-687 is “a very promising therapeutic approach for the treatment of sickle disease.”
“I am hopeful about IMR-687’s unique potential to help the sickle cell patient community who have limited treatment options,” Julie Kanter, director of Sickle Cell Research at the Medical University of South Carolina in Charleston, said in a statement. “Sickle cell disease causes lifelong chronic organ damage including pain, stroke, acute chest syndrome, kidney failure and heart failure. We need more options now to improve the outcomes of all individuals living with sickle cell disease.”
Prior to the spinout of Imara, Cydan was able to secure $31 million in Series A funding from various backers including , New Enterprise Associates, Pfizer Venture Investments, Lundbeckfond Ventures, Bay City Capital and Alexandria Venture Investments.
Imara faces competition in the sickle cell disease market, as other companies are working on therapies for the disease, including Cambridge, Mass. neighbor bluebird bio , which is developing a gene therapy treatment for the disease. Data released by bluebird showed some of the patients treated with the bluebird’s gene therapy LentiGlobin BB305, have responded well to the treatment. According to the data, a sickle cell patient has been transfusion free with no sickle cell hospitalizations for nearly three months after undergoing the therapy and he is now producing higher levels of hemoglobin. LentiGlobin was granted Breakthrough Therapy Status by the U.S. Food and Drug Administration (FDA) in February. The designation is given to drugs expected to treat life-threatening illnesses in order to expedite the development and review of the drug.
Cydan’s first company, Vtesse Inc., which had a banner first year since its launch in January 2015. The company received orphan drug designation for its lead drug candidate, VTS-270 from both the U.S. and E.U. regulatory agencies. VTS-270, has shown promise in pre-clinical and clinical studies as a potential treatment for Niemann-Pick Type C (NPC). The drug is a mixture of (2-hydroxypropyl)-beta-cyclodextrin and has undergone studies at NCATS, NICHD and the NIH Clinical Center, as well as under individual compassionate use investigational new drug applications (iINDs) and in other academic labs.
