Imara disappoints in sickle cell disease

Imara was always going to have a tough time living up to expectations set in sickle cell disease by the likes of Crispr Therapeutics. But the group’s lead project, IMR-687, did not even come close, with weak phase IIa data sending the company’s stock down 37% this morning. The trial evaluated both IMR-687 alone and in combination with hydroxyurea, but neither arm impressed. Focusing on the monotherapy cohort, Imara had hoped that tweaks to its dosing regimen would spur an improvement over the 1.7% mean increase in foetal haemoglobin percentage previously seen. The aim had been to get this closer to 3%, the threshold set by the FDA. However, the latest monotherapy data showed a 1% decline in foetal haemoglobin percentage versus baseline. On the bright side, there was a 25% reduction in vaso-occlusive crises with IMR-687 monotherapy versus placebo; however, there was no impact on VOCs with IMR-687 plus hydroxyurea versus hydroxyurea alone. Imara will soon have data from its SCD open-label extension trial, while the second half of the year will see results from the higher-dose Ardent and Forte studies in SCD and beta-thalassaemia respectively. The company will need to do better if it is not to become an also-ran.