EHA24: Vertex highlights longer-term benefits of Casgevy in blood disorders

16 Jun 2024
firstwordpharma.com
Drug ApprovalGene TherapyClinical ResultClinical Study
Vertex Pharmaceuticals has unveiled longer-term data from clinical trials of its CRISPR-based gene-editing therapy Casgevy (exagamglogene autotemcel), confirming "consistent and durable" benefits of the treatment in patients with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT).
The findings, presented at the European Hematology Association (EHA) annual meeting on Friday, are likely to help Vertex and partner CRISPR Therapeutics keep up the launch momentum for Casgevy, which was approved by the FDA in December for SCD – along with bluebird bio's rival gene therapy Lyfgenia (lovotibeglogene autotemcel) – and later also had TDT added to its label.
The readouts were centred on the CLIMB-111 and -121 trials, as well as the CLIMB-131 long-term follow-up study. Overall, Vertex said the data were drawn from over 100 patients treated with Casgevy, with the longest follow-up now extending beyond five years.
According to the company, efficacy data remain "consistent" with the previously reported primary and key secondary endpoints, and continue to show sustained and "stable levels" of foetal haemoglobin (HbF) and allelic editing over time.
Vertex said that 36 of 39 SCD evaluable patients (92.3%) in CLIMB-121 study were free from vaso-occlusive crises (VOCs) for a minimum of 12 consecutive months. On average, patients remained VOC-free for 27.9 months, with the longest duration being 54.8 months. Further, 38 patients (97.4%) had no hospitalisations related to VOCs for at least 12 consecutive months.
In the CLIMB-111 study, 49 out of 52 evaluable TDT patients (94.2%) achieved transfusion independence for at least 12 consecutive months, with a mean weighted haemoglobin of at least 9 g/dL. The average duration of transfusion independence was 31 months, with a maximum of 59.4 months. Moreover, all TDT patients with a minimum of 16 months of follow-up are now transfusion-free. Two of three patients who initially did not achieve transfusion independence later achieved this milestone in the long-term follow-up study, while the third has been transfusion free for 3.4 months.
For both SCD and TDT cohorts, Vertex said that edited BCL11A alleles persisted over time in bone marrow and peripheral blood, "indicating successful editing in the long-term haematopoietic stem cells." All patients also achieved successful engraftment of neutrophils and platelets after their Casgevy infusion.
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