Exa-Cel Sustains Promising Efficacy as SCD Treatment in Phase III Trial

Phase III data from the CLIMB SCD-121 trial verified the efficacy of exagamglogene autotemcel (exa-cel) in eliminating vaso-occlusive crises in patients with sickle cell disease (SCD), according to an article in the New England Journal of Medicine.

In the study, “treatment with exa-cel eliminated vaso-occlusive crises in 97% of patients with SCD for a period of 12 months or more,” wrote Haydar Frangoul, MD, of the Sarah Cannon Research Institute, the study’s lead author.

CLIMB SCD-121 included patients aged 12 to 35 years with SCD and at least two severe vaso-occlusive crises in each of the two years prior to screening. Patients underwent myeloablative conditioning with busulfan prior to infusion of CD34-positive hematopoietic stem and progenitor cells.

Can Sickle Cell Disease be Cured?

The primary endpoint was freedom from vaso-occlusive crises for at least 12 consecutive months, and the key secondary endpoint was freedom from inpatient hospitalization for severe vaso-occlusive crises for at least 12 consecutive months.

A total of 44 patients received exa-cel with a median follow-up of 19.3 months (range, 0.8-48.1 months). Of the 30 patients eligible for analysis, 29 (97%; 95% CI, 83-100; P<.001) achieved the primary endpoint, and all 30 (95% CI, 88-100; P<.001) achieved the secondary endpoint.

Dr. Frangoul and colleagues noted the safety profile of exa-cel was “generally consistent with that of myeloablative busulfan conditioning and autologous [hematopoietic stem and progenitor cell] transplantation.”

Related: Haydar Frangoul, MD, Details CLIMB-121 Data on Exa-Cel in Sickle Cell Disease

Reference

Frangoul H, Locatelli F, Sharma A, et al. Exagamglogene autotemcel for severe sickle cell disease. N Engl J Med. April 24, 2024. doi:10.1056/NEJMoa2309676