Imara, a pharmaceutical company developing new therapies for sickle cell disease (SCD), has successfully dosed the first patient enrolled in its clinical trial evaluating escalating doses of IMR-687 in adult patients with SCD.
The Phase 2 trial (NCT03401112) is currently recruiting participants and expects to enroll about 50 patients at several clinical trial centers in the United Kingdom and the United States. For more information, visit the clinical trial webpage here. The U.S. trial sites had not yet been listed at publication time.
The primary objective is to assess the safety and tolerability of IMR-687 as well as its effects inside the body, a collection of parameters known as pharmacokinetics and pharmacodynamics, compared to a placebo control group.
Researchers will expand the trial to include a group of adults on a stable dose of hydroxyurea, currently the treatment prescribed in up to 35% of patients with sickle cell disease.
IMR-687 is a potent and selective inhibitor of the enzyme called phosphodiesterase 9 (PDE9) in red blood cells. Previous results from preclinical studies showed that treatment with IMR-687 increased fetal globin and reduced red cell sickling and death. The therapy also improved occlusion of blood vessels.
Last year, the U.S. Food and Drug Administration (FDA) granted orphan drug status and rare pediatric disease designation to IMR-687.
“As part of our commitment to bringing a transformative new treatment to patients in need, we are rapidly advancing IMR-687 – we are dosing our first sickle-cell patient less than two years from launching Imara,” said James McArthur, PhD, founder, president, and CEO of Imara, in a press release.
“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,” McArthur added.
Study investigator Dr. Shivan Pancham of Sandwell and West Birmingham Hospitals in the U.K. said the current disease-modifying strategies for SCD “are hydroxycarbamide, blood transfusions and, for a few, hematopoietic stem cell transplant. For the majority of patients, the mainstay of treatment is supportive.
“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,” Pancham added.