Imara to Present Preclinical Effectiveness and Safety Data on IMR-687 as Sickle Cell Disease Treatment

Margarida Azevedo, MSc avatar

by Margarida Azevedo, MSc |

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Imara is scheduled to present preclinical efficacy and safety data on IMR-687, its lead product candidate for the treatment of sickle cell disease (SCD), at the American Society of Hematology (ASH) 58th Annual Meeting and Exposition in San Diego, Calif., Dec. 3-6.

The oral presentation, titled “A Novel, Highly Potent and Selective PDE9 Inhibitor for the Treatment of Sickle Cell Disease,” will be presented by James McArthur, PhD, founder, president and chief executive officer of Imara.

“There is a tremendous need for safe, effective, and easy-to-administer, disease-modifying treatments for people living with sickle cell disease,” McArthur said in a press release. “Imara looks forward to sharing the compelling data supporting the development of IMR-687 for prevention of the pathologies of severe hemoglobinopathies at the ASH annual meeting as we continue working to advance new therapeutic options aimed at reducing both the sickling of red blood cells and blood vessel occlusion,” he said.

Sickle cell disease is an inherited blood disorder caused by flawed hemoglobin, the protein in red blood cells that carries oxygen to the tissues of the body.

Cells with sickle cell hemoglobin are stiff and sticky, and when they lose their oxygen, they form into the shape of a sickle or crescent. These cells stick together and can’t easily move through the blood vessels, which can block small blood vessels and the movement of healthy, normal, oxygen-carrying blood. IMR-687 both reportedly reduces red blood cell sickling and blockage of blood vessels, the underlying causes of SCD. The drug is a highly potent, selective inhibitor of phosphodiesterase-9 (PDE9) in blood cells.

In preclinical animal studies, IMR-687 has been shown to increase fetal hemoglobin, the major genetic modulator of the hematologic and clinical features of SCD, which prevents the polymerization of the sickled hemoglobin.

By increasing fetal globin, IMR-687 is able to reduce red blood cell sickling, reduce red blood cell death, and reduce occlusion of blood vessels. The inhibition of the PDE9 enzyme also reduces white blood cell “stickiness,” which also contributes to the reduction of the blockage of blood vessels. Imara is moving IMR-687 into human clinical trials.

During the ASH presentation, the company also will provide an update of the clinical development of IMR-687.