Haydar Frangoul, MD, on Clinically Meaningful and QoL Improvements in Sickle Cell With Exa-Cel

“Across the board, all the quality of life measures we measured showed significant improvement over time. And that's a lot to be said for a product that is using your own cells, not somebody else's cells, and hopefully would be available for every patients with sickle cell disease.”

The CLIMB SCD-121 trial met primary and key secondary endpoints, with exa-cel treatment resulting in early and sustained increases in Hb and HbF leading to elimination of VOCs in 95% of pts, elimination of inpatient hospitalization for VOCs in 100% of pts and improved QOL. Safety profile of exa‑cel was generally consistent with myeloablative busulfan conditioning and autologous transplantation. These results show exa-cel has the potential to deliver a one-time functional cure to pts with severe SCD.

Exagamglogene autotemcel (exa-cel; Vertex Pharma, CRISPR Therapeutics), recently approved as Casgevy for treating sickle cell disease (SCD), yielded early and sustained increases in Hb and HbF leading to elimination of vaso-occlusive crises in 95% of patients treated in the phase 3 CLIMB SCD121 trial (NCT03745287), which has met its primary and key secondary endpoints.

Updated data from the trial were presented at the 2023 American Society of Hematology (ASH) Annual Meeting & Exposition, held December 9-12, in San Diego, California, by Haydar Frangoul, MD, Medical Director of Pediatric Hematology/Oncology at Sarah Cannon Research Institute and Pediatric Transplant and Cellular Therapy Program at TriStar Centennial.

CGTLive spoke with Frangoul to learn more about the new data being presented. He emphasized how the improvements seen in treated patients are clinically meaningful and also touched on quality-of-life improvements presented in an additional poster.

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REFERENCE
Frangoul H, Locatelli F, Sharma A, et al. Exagamglogene autotemcel for severe sickle cell disease. Presented at: ASH 2023 Annual Meeting & Exposition. December 9-12; San Diego, CA. Abstract #1052