Sickle cell drug showing promise in clinical trial

ScienceDaily | 6/19/2019 | Staff
TimHyuga (Posted by) Level 3
For the rare, painful red blood cell disease, which impacts blood circulation, pre-clinical laboratory data had already illuminated the potential of the experimental drug IMR-687 for reducing both the sickling of red blood cells and blood vessel blockages. The drug was shown to reduce these two major culprits that lead to sickle cell disease's debilitating pain, organ damage, and early mortality of patients -- who have an average life expectancy of 40. That is why the drug has been granted U.S. Orphan Drug Designation, U.S. Rare Pediatric Designation, and Fast Track Designation by the Food and Drug Administration (FDA).

After 13 weeks of testing in its current clinical trial, the orally administered, once-a-day phosphodiesterase 9 (PDE9) inhibitor in adult patients with sickle cell disease is demonstrating tolerability and the ability to impact both red and white blood cell biomarkers of the disease, says Dr. Biree Andemariam, lead investigator for the clinical trial, associate professor of medicine at UConn School of Medicine and director of the New England Sickle Cell Institute at UConn Health.

Phase - Data - Potential - IMR-687 - Biomarkers

"These initial Phase 2a data demonstrate the potential of IMR-687 to significantly impact key biomarkers associated with the pathology of this serious disease," she says.

A biomarker is a measurable indicator of a biological condition, often evaluated to examine normal biological processes, pathogenic processes, or pharmacologic responses to a therapeutic intervention.

Trial - Safety - Tolerability - Pharmacokinetics - Pharmacodynamics

The clinical trial is evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of escalating doses of IMR-687 administered once daily for 16 to 24 weeks in two groups of patients with sickle cell disease.

Patient groups include those receiving the current standard of care dose of hydroxyurea -- a drug that helps...
(Excerpt) Read more at: ScienceDaily
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