26 June 2019

A drug for sickle cell disease is showing great promise in the early stages of its clinical trial, a conference has heard.

The first findings from the trial of the drug IMR-687 were reported to the European Haematology Association Congress in Amsterdam last week. Pre-clinical testing in mice has suggested the drug could reduce the sickling of cells and reduce blood vessel blockages.

The early findings of the trial in Connecticut, USA, suggest that the drug is tolerable to the patients and is having an impact on red and white blood cells biomarkers of the disease, the conference heard.

The trial involves adult patients with sickle cell disease, including those receiving hydroxyurea and those not on hydroxyurea.

The interim analysis at 13 weeks shows an increase in F-cells, the red blood cells containing foetal haemoglobin. There was also a concurrent decrease in absolute reticulocyte count together with a decrease in the percentage of reticulocytes.

IMR-687 is a PDE9 inhibitor, a class of drugs initially developed for the treatment of Alzheimer's disease.

Researcher Dr Biree Andemariam, Director of the New England Sickle Cell Institute at UConn Health, said: “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 added: “The interim Phase 2a data reflect trends that could be indicative of meaningful clinical translation of these important measures in sickle cell disease.”

More results from the trial are expected to be shared later in 2019.

Source: EHA Congress Abstract: https://library.ehaweb.org/eha/2019/24th/267437/biree.andemariam.imr-687.a.highly.selective.phosphodiesterase.9.inhibitor.html

University of Connecticut: https://today.uconn.edu/2019/06/sickle-cell-drug-showing-promise-clinical-trial/