Beam Therapeutics Inc. (NASDAQ:BEAM) saw its shares rise 1.7% in premarket trading Monday after releasing updated results from its BEACON Phase 1/2 study evaluating ristoglogene autogetemcel (risto-cel) for sickle cell disease (SCD).
The latest data, presented at the 67th American Society of Hematology Annual Meeting, showed mean fetal hemoglobin (HbF) induction above 60% along with substantial reductions in hemoglobin S—leading to durable anemia resolution for up to 20 months in a cohort of 31 adolescents and adults with SCD. Most participants required only a single cell-collection cycle and demonstrated rapid neutrophil and platelet recovery.
Beam noted that no patients experienced investigator-reported severe vaso-occlusive crises following engraftment. Meanwhile, mean peripheral blood editing levels reached 67.4% at Month 6 and climbed to 72.8% by Month 12.
Commenting on the progress, CEO John Evans said, “Risto-cel continues to demonstrate how base editing can fulfill its promise as a next-generation precision genetic medicine that could significantly improve the treatment process and outcomes for patients with SCD.”
Safety findings remained consistent with expectations for busulfan conditioning, autologous stem cell transplantation, and the underlying disease. The most common treatment-emergent adverse events included stomatitis, febrile neutropenia, and decreased appetite.
Beam also shared new mobilization data showing that a fixed, tiered-dose plerixafor regimen produced higher CD34+ yields, faster collection times, and fewer collection cycles compared with standard weight-based dosing.
The company said it remains on track to dose the remaining participants in the study and move toward a future regulatory submission.