EHA23: Vertex, CRISPR sickle cell data continues to impress, as Editas provides early glimpse
09 Jun 2023
Clinical ResultPhase 3Priority Review
Vertex Pharmaceuticals and CRISPR Therapeutics on Friday presented the latest update from key studies of exagamglogene autotemcel (exa-cel) for transfusion-dependent beta thalassemia (TDT) and severe sickle-cell disease, while Editas Medicine released results from the first patients treated in pivotal trials of EDIT-301. The data were disclosed at the European Hematology Association (EHA) annual congress.
At the same conference last year, Vertex and CRISPR provided an update on 75 patients treated with exa-cel, an autologous, ex vivo CRISPR/Cas9 gene-edited therapy being, in the Phase III CLIMB-111 and CLIMB-121 studies. Those results showed that in the first trial, 42 of 44 TDT patients were transfusion-free, while in the second study, all 31 severe SCD subjects were free of vaso-occlusive crises (VOCs).
The most recent update includes 83 patients across the two trials: 48 in CLIMB-11 and 35 in CLIMB-121. Results showed that 88.9% of the 27 subjects with TDT who were evaluable at the time of the data cut achieved the primary endpoint of transfusion-independence for at least 12 consecutive months and the secondary goal of transfusion-independence for at least 6 consecutive months.
Meanwhile, 17 patients were evaluable in CLIMB-121, with all but one, or 94.1%, achieving the primary endpoint of freedom from VOCs for at least 12 consecutive months. Further, all patients achieved the key secondary goal of being free from hospitalisations related to VOCs for at least 12 consecutive months.
Results from the studies have been used to support marketing applications in Europe and the US, with the companies disclosing Friday that the FDA accepted the filings. Vertex and CRISPR noted that the agency granted priority review in SCD, setting a target data of December 8, while the submission in TDT will be assessed on a standard timeline, with a decision due by March 30 next year.
Playing catchup
Separately on Friday, Editas presented initial safety and efficacy data from the first four SCD patients treated with EDIT-301 in the Phase III RUBY trial and from the first TDT subject in the late-stage EdiTHAL study. The cell therapy consists of patient-derived CD34+ haematopoietic stem and progenitor cells edited at the gamma globin gene promoters.
Results to be presented at EHA showed that the first two SCD patients given EDIT-301 achieved normal levels of total haemoglobin and foetal haemoglobin of >40% at 5 months and maintained these levels after between 6 and 10 months of follow-up. Editas noted that the two other participants saw increases at 2 and 3 months of follow-up that "follow similar trajectories" to the first two patients. All four subjects are also free of vaso-occlusive events.
Meanwhile, the TDT patient given EDIT-301 demonstrated successful neutrophil and platelet engraftment, and, at 1.5 months post-infusion, their response resembles that of those in RUBY. "These promising data support our belief that EDIT-301 can be a clinically differentiated, one-time, durable medicine" remarked Baisong Mei, chief medical officer at Editas, adding "we remain on-track to dose 20 RUBY patients by year-end."
More to follow.
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