Editas Medicine reports updated clinical data from RUBY trial of Reni-cel

Editas Medicine (EDIT) will present updated safety and efficacy data in 28 patients living with severe sickle cell disease treated with renizgamglogene autogedtemcel in the Phase 1/2/3 RUBY clinical trial. The data will be presented by Dr. Rabi Hanna, M.D., Department of Pediatric Hematology Oncology and Blood and Marrow Transplantation, Cleveland Clinic Children’s, during a poster presentation at the American Society of Hematology Annual Meeting in San Diego, CA, at 6:00 p.m. PT. In the RUBY trial as of the data cutoff date, reni-cel was well-tolerated and continued to demonstrate a safety profile consistent with myeloablative busulfan conditioning and autologous hematopoietic stem cell transplant by all patients. Patients were at a median of 9.5 months post-reni-cel infusion, with 11 patients having greater than1 year follow-up. Since reni-cel treatment, 27 of the 28 patients were free of vaso-occlusive events. Patients were observed to have early normalization of total hemoglobin, with a mean total hemoglobin increasing from 9.8 g/dL at baseline to 13.8 g/dL at Month 6. Patients were also observed to have rapid and sustained improvements in fetal hemoglobin greater than or equal to40% and mean corpuscular concentration of HbF per F-cells, well above the anti-sickling threshold. In addition, sustained clinically meaningful improvements were observed in patient-reported outcome domains for pain, physical function, and social roles and activities. Patients were a median of 9.5 months post-reni-cel infusion, with 11 patients having greater than1 year follow-up. Of 28 patients, 27 were VOE-free post-reni-cel infusion. Reni-cel administration led to early, robust increases and sustained levels of total Hb and HbF. At month 6, the mean total Hb was 13.8 g/dL with a mean HbF percentage of 48.1%. The mean percentage of F-cells increased early and was sustained at greater than90% from month 4 through last follow-up. MCH-F of HbF-containing red cells increased early, with mean value of 16.3 pg/F-cells at month 4 visit and sustained above the anti-sickling threshold of 10 pg/F-cell through last follow-up. Markers of hemolysis, including absolute reticulocyte count, indirect bilirubin, lactate dehydrogenase, and haptoglobin, improved or normalized by Month 6 and were generally maintained or improved as of last follow-up. Sustained clinically meaningful improvements were observed in pain, physical, and social patient-reported outcome domains following treatment with reni-cel. 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. After reni-cel infusion, all evaluable patients achieved successful engraftment; with median time to neutrophil engraftment of 23 days and median time to platelet engraftment of 25 days, which is important for limiting infection and bleeding risk. Two serious adverse events assessed by the investigators as possibly related to reni-cel treatment have been reported in the RUBY trial.