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Vertex and CRISPR Therapeutics Present New Data on More Patients With Longer Follow-Up Treated With exagamglogene autotemcel (exa-cel) at the 2022 European Hematology Association (EHA) Congress
– Data from 75 patients with transfusion-dependent beta thalassemia or severe sickle cell disease with follow-up of up to 37.2 months continue to demonstrate
About this update from Crispr Therapeutics Ag
[{"type":"text","content":"\n– Data from 75 patients with transfusion-dependent beta thalassemia or severe sickle cell disease with follow-up of up to 37.2 months continue to demonstrate that exa-cel has the potential to be a one-time functional cure –\n\n– Safety profile generally consistent with myeloablative conditioning and autologous stem cell transplant –\n\n BOSTON & ZUG, Switzerland & CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nVertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) announce presentation of new data on exa-cel, formerly known as CTX001™, from CLIMB-111, CLIMB-121 and CLIMB-131 highlighting the potentially transformative profile of this investigational therapy for people with transfusion-dependent beta thalassemia (TDT) or severe sickle cell disease (SCD) and provided additional program updates.\n\nNew Data for exa-cel from CLIMB Clinical Studies\nThe data presented at the European Hematology Association (EHA) Congress are from 75 patients (44 with TDT and 31 with SCD) with follow-up ranging from 1.2 to 37.2 months after exa-cel dosing.\n\nOf the 44 patients with TDT, 26 had beta-zero/beta-zero or other beta-zero-like severe genotypes. Forty-two of 44 patients with TDT were transfusion-free with follow-up ranging from 1.2 to 37.2 months after exa-cel infusion. Two patients who were not yet transfusion-free had 75% and 89% reductions in transfusion volume. TDT patients had substantial mean increases in fetal hemoglobin (HbF) and corresponding increases in mean total hemoglobin (Hb) with mean total Hb levels increasing to >11 g/dL by Month 3 and maintained thereafter.\n\nAll 31 patients with severe SCD characterized by recurrent vaso-occlusive crises (VOCs) (mean of 3.9 VOCs per year over the prior two years) were free of VOCs after exa-cel infusion through duration of follow-up, with follow-up ranging from 2.0 to 32.3 months. SCD patients had mean HbF (as a proportion of total Hb) of approximately 40% by Month 4 and maintained thereafter.\n\nThe safety was generally consistent with myeloablative conditioning with busulfan and autologous stem cell transplant. All patients engrafted neutrophils and platelets after exa-cel infusion. Among the 44 patients with TDT, two patients had serious adverse events (SAEs) considered related to exa-cel. As previously reported, one patient had three SAEs considered related...