
Two drug makers — Forma Therapeutics and Agios Pharmaceuticals — are each developing their own oral medicines that aim to benefit patients with sickle cell disease by boosting the energy levels and health of red blood cells. But the competing clinical trials reported Monday have produced divergent results, with Forma’s drug performing better than expected and Agios’ drug falling short.
In the Forma study, six of seven patients (86%) treated with its FT-4202 drug saw an increase in oxygen-carrying hemoglobin of at least 1 gram per deciliter — the predetermined threshold for a response.
In the Agios study, six of 11 patients (55%) treated with its drug, mitapivat, achieved the same hemoglobin response.