Editas Medicine, Inc. has recently announced new safety and efficacy data in 18 patients with sickle cell disease (SCD) who were treated with renizgamglogene autogedtemcel (reni-cel) in the phase 1/2/3 ruby clinical trial. The data was presented at the European Hematology Association (EHA) annual congress.
Since treatment with reni-cel, patients have been free of vaso-occlusive events (VOEs) for up to 22.8 months of follow-up. The patients also experienced early normalization of total hemoglobin (HB) with a mean within the normal range at >14 g/dl and rapid and sustained improvements in fetal hemoglobin (HBF) well above levels of >40%.
The data also revealed that all patients in the ruby trial showed sustained high levels of editing in the hbg1 and hbg2 promoter regions. Additionally, markers of hemolysis have been normalized or improved in patients treated with reni-cel.
Furthermore, reni-cel was well-tolerated and demonstrated a safety profile consistent with myeloablative conditioning with busulfan and autologous hematopoietic stem cell transplant by all evaluated ruby trial patients (n=18). After reni-cel infusion, all patients demonstrated successful neutrophil and platelet engraftment.
In terms of the efficacy of reni-cel, all patients are free of VOEs since reni-cel infusion with follow-up ranging from 2.4 to 22.8 months. Reni-cel treatment led to early, robust increases and sustained levels of total HB and HBF. Across patients with ≥6 months follow-up, at month 6, the mean total HB was 14.3 g/dl with a mean HBF of 48.5%. The mean percentage of f-cells increased early and were sustained at >90% from month 4 through subsequent follow-ups for all patients with ≥4 months follow-up.
These results from the ruby trial are encouraging and demonstrate the investigational gene editing medicine's favorable safety profile and promising preliminary efficacy for people living with sickle cell disease. This supports further investigation as a differentiated gene-edited medicine for patients with SCD.
Reni-cel is currently being investigated in a clinical study in patients with severe sickle cell disease (ruby trial, nct04853576) and transfusion-dependent beta thalassemia (edithal trial, nct05444894). The company will present a further clinical update from both trials by year-end 2024. The market has reacted to these announcements by moving the company's shares -8.1% to a price of $5.04. For the full picture, make sure to review Editas Medicine's 8-K report.
