Rare Disease Report

First Sickle Cell Patient Dosed in Phase 2a of IMR-687

FEBRUARY 13, 2018
Mathew Shanley
This morning, Imara Inc. announced that it has dosed the first patient in its Phase 2a clinical trial to evaluate the safety, pharmacokinetics and pharmacodynamics of escalating doses of IMR-687 in adult patients with sickle cell disease (SCD).

In May, IMR-687 was granted Rare Pediatric Disease designation by the U.S. Food and Drug Administration (FDA), the first SCD product candidate to receive the label. The drug is being developed as a highly-potent oral therapy for once-daily dosing with the intention of addressing both the underlying red and white blood cell pathologies associated with the condition.

SCD is a severe, hereditary variation of anemia in which a mutated form of hemoglobin distorts the red blood cells at low oxygen levels into crescent-shaped cells that resemble a sickle. “IMR-687 is being rapidly advanced, as Imara is dosing the first eligible patient less than 2 years from the launch of the company.

At present, the current standard of care for patients with SCD includes hydroxurea and L-glutamine oral powder. Bone marrow or stem cell transplants also serve as options for younger patients, however, have limited efficacy and can result in life-threatening side effects.

“Based on the data to date, we believe IMR-687 has the potential to dramatically improve the lives of patients with SCD by reducing red blood cell sickling and red blood cell lysis, reducing white blood cell adhesions, thus ultimately reducing vaso-occlusive crisis and hospitalizations,” said James McArthur, Ph.D., Founder, President, and Chief Executive Officer of Imara in a press release.

Pre-clinical data demonstrate that 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. A Phase 1 clinical trial showed the drug to be safe and well-tolerated in healthy volunteers.

The Phase 2a study is a randomized, double-blind, multi-center study of IMR-687 that will expand to include adults on a stable dose of hydroxurea, which up to 25% to 35% of patients with SCD are estimated to have been prescribed. Approximately 50 patients will be enrolled at clinical trial centers in both the United States and United Kingdom.

“The current disease-modifying strategies in sickle cell disease are hydroxycarbamide, blood transfusions and, for a few, haematopoietic stem cell transplant. For the majority of patients, the mainstay of treatment is supportive,” said Dr. Shivan Pancham of Sandwell and West Birmingham Hospitals, an investigator in the study. “Newer agents that have been developed based on the greater understanding of the pathophysiology of sickle cell disease have the potential to become new treatment options needed for this condition.”

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