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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.
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This platform is made possible through a partnership with the Sickle Cell Disease Association of America, Inc. (SCDAA) and its member organizations. SCDAA's mission is to advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure.