Imara (IMRA -13.6%) presented interim results from its ongoing Phase 2a clinical trial of IMR-687 in adult patients with sickle cell disease (SCD) at the 25th European Hematology Association Annual Congress.
The data demonstrated that IMR-687, a highly selective and potent small molecule inhibitor of an enzyme called PDE9, was safe and well tolerated as a monotherapy and in combination with hydroxyurea (HU).
In the higher dose cohort, IMR-687 monotherapy showed a statistically significant (p=0.022) increase in the number of F-cells, which are red blood cells containing fetal hemoglobin (HbF) compared to placebo after 24 weeks of dosing.
A dose-dependent increase in HbF levels in adult patients with SCD in the monotherapy arm was also observed.
IMR-687 was well tolerated. There was no hypotension or neutropenia observed in either the monotherapy or combination arms.