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.

The data, while encouraging, are not as profound as those presented by bluebird bio and CRISPR Therapeutics on their respective gene therapy candidates.



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