CLIMB SCD-121 Study Shows Exagamglogene Autotemcel Reduces VOCs in Sickle Cell Disease

Nearly all patients with severe sickle cell disease (SCD) who were treated with exagamglogene autotemcel (exa-cel) had no vaso-occlusive crisis (VOC) episodes and eliminated inpatient hospital visits, according to study findings presented at the 65th American Society of Hematology (ASH) Annual Meeting & Exposition. Thus, exa-cel improved quality of life.

The ongoing, phase III CLIMB SCD-121 study evaluated the proportion of patients free of severe VOCs for at least 12 months. Key secondary endpoints included the proportion of patients free from inpatient hospitalization for severe VOCs for 12 or more consecutive months and the proportion of patients free from severe VOCs for nine or more consecutive months.

Patients included in the study were aged 12 to 35 years and all had SCD and a history of two or more VOCs a year in the two years prior to screening. Exa-cel was administered to 42 patients and, following infusion, all patients achieved engrafted neutrophils and platelets.

The study showed that 95% of patients had no VOCs for 12 months or longer and were free from hospitalizations during that same time. Furthermore, 96.7% of patients were free of VOCs for at least nine months.

In addition, patients who received exa-cel had early and sustained increases in total hemoglobin (Hb) and fetal hemoglobin (HbF). At three months, total Hb was 12.1 g/dL and was maintained at ≥11.0 g/dL from six months onward. At three months, HbF was 36% and was maintained at ≥40% from six months onward with pancellular distribution.

“These results show exa-cel has the potential to deliver a one-time functional cure to patients with severe SCD,” the investigators said.

Adverse events (AEs) were experienced by all patients and most occurred within the first six months after exa-cel infusion. The most common AEs were nausea (66.7%), stomatitis (61.9%), febrile neutropenia (52.4%), headache (52.4%), and vomiting (52.4%). Grade 3 or 4 AEs were seen in 40 patients.

Reference

Frangoul H, Locatelli F, Sharma A, et al. Exagamglogene autotemcel for severe sickle cell disease. Abstract #1052. Presented at the 65th American Society of Hematology Annual Meeting & Exposition; December 9-12; San Diego, California.