Imara announces oral dosing of the first β-thalassaemia patient with IMR-687

Imara's IMR-687 is an oral disease-modifying treatment for sickle cell disease (SCD) and β-thalassaemia. IMR-687 is a highly selective and potent small molecule inhibitor of PDE9. Blocking PDE9 acts to increase cyclic GMP levels, which is associated with reactivation of foetal haemoglobin, or HbF, a well-established modifier of disease severity. Having received Orphan Drug Designation by the FDA for IMR-687 for treatment of β-thalassaemia earlier in the year (June 2020 press release), Imara announces dosing of the first β-thalasaemia patient in the Forte Phase 2b clinical trial (NCT04411082). Multiple preclinical studies show that treatment with IMR-687 enhances both the maturation and production of red blood cells in β-thalassaemia. This is exciting news towards the development of β-thalassaemia therapeutics as there are currently no approved oral therapies to increase HbF in β-thalasaemia. Read more on Imara's press release.