Home > Haematology > EHA 2019 > Benign Haematology > Positive initial data evaluating the safety and efficacy of IMR-687 for treatment of sickle cell disease

Positive initial data evaluating the safety and efficacy of IMR-687 for treatment of sickle cell disease

Presented By
Dr Biree Andemariam, UConn Health, USA
Conference
EHA 2019
Promising 13-week interim phase 2a data on IMR-687 in patients with sickle cell disease (SCD) were presented by Dr Biree Andemariam (UConn Health, USA). Treatment with IMR-687 in adult patients was generally well tolerated. The data also support the dual mechanism of action of IMR-687, with activity seen across both red and white blood cell biomarkers. IMR-687 is an investigational, orally administered, selective phosphodiesterase 9 (PDE9) inhibitor. Dr Andemariam explains: "In the laboratory we saw that IMR-687 inhibition of PDE9 increases intracellular cGMP levels, increases foetal haemoglobin expression, reduces sickling and haemolysis of red blood cells, and does not induce neutropenia. The interim phase 2a data reflect trends that could be indicative of meaningful clinical translation of these important measures in SCD." The phase 2a clinical trial is designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of escal...


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