Beam Therapeutics Inc. · NASDAQ:BEAM
Beam Therapeutics — 67th ASH Annual Meeting 2025
15 slides from Beam Therapeutics's conference presentation, December 2025
Slide 1: Enhanced CD34+ cell mobilizations with fixed-dose versus weight-based plerixafor in BEACON study
Enhanced CD34+ cell mobilizations, collections, and comparable safety profile with fixed-dose versus weight-based plerixafor dosing in patients with sickle cell disease receiving autologous CD34+ base-edited hematopoietic stem cells (ristoglogene autogetemcel; BEAM-101) in the ongoing BEACON study H
Slide 2: Disclosures
Disclosures
Slide 3: BEACON is a Phase 1/2 clinical study evaluating risto-cel (BEAM-101)
BEACON is a Phase 1/2 clinical study evaluating the safety and efficacy of risto-cel (BEAM-101) in patients with SCD and sVOCs Base editing with risto-cel1,2 BEACON treatment process1 ► Age from ≥12 years to ≤35 years 1 Screening and eligibility ► SCD with βS/βS, βS/β0, or βS/β+ genotypes ► ≥4 sVOCs
Slide 4: Fixed-dose plerixafor regimen has been previously shown to enhance HSPC mobilization
Fixed-dose plerixafor regimen has been previously shown to enhance HSPC mobilization into peripheral blood ► Dose optimization can improve mobilization and lower the number of collection cycles, which can decrease patient burden and hospital resource utilization1,2 ► Detailed PKPD modeling (weight-b
Slide 5: Baseline demographics and characteristics of patients enrolled in the BEACON clinical study
Baseline demographics and characteristics of patients enrolled in the BEACON clinical study, by plerixafor dosing group All treated Fixed-dose Weight-based Weight-based Baseline characteristics population plerixafor (<83 kg) plerixafor (<83 kg) plerixafor (≥83 kg) Enrolled N=31 N=27 N=7 N=8 N=54 Mea
Slide 6: More CD34+ cells were mobilized with fixed-dose plerixafor than weight-based dosing
More CD34+ cells were mobilized with fixed-dose plerixafor than weight-based dosing CD34+ plasma profile in patients with bodyweight ≤83 kg Day 1 Day 2 Day 3 Day 4 200 N= 37 13 37 13 35 12 35 12 32 12 32 12 20 5 20 5 Plasma CD34+ cells (106/L) Regimen Fixed Weight-based 150 100 50 0 Prior to Prior t
Slide 7: More CD34+ cells were collected with fixed-dose plerixafor than weight-based dosing
More CD34+ cells were collected with fixed-dose plerixafor than weight-based dosing CD34+ cells collected by apheresis in patients with bodyweight ≤83 kg Day 1 Day 2 Day 3 Day 4 20 N= 37 13 35 12 32 12 20 5 Collected CD34+ cells (106/kg) 15 10 5 0 Fixed Weight-based Fixed Weight-based Fixed Weight-b
Slide 8: Patients receiving a fixed-dose regimen required fewer mobilization days and collection cycles
Patients receiving a fixed-dose regimen required fewer mobilization days and collection cycles to achieve dose Fixed-dose Weight-based Weight-based plerixafor (<83 kg) plerixafor (<83 kg) plerixafor (≥83 kg) N=27 N=7 N=8 ► 70% of patients with bodyweight <83 kg who received fixed-dose Mobilization/a
Slide 9: The safety profile was comparable between fixed-dose plerixafor and weight-based dosing
The safety profile was comparable between fixed-dose plerixafor and weight-based dosing Fixed-dose Weight-based Weight-based Patients with, n (%) plerixafor (<83 kg) plerixafor (<83 kg) plerixafor (≥83 kg) N=27 N=7 N=8 AEs during mobilization period AEs 7 days from the start of plerixafor 15 (55.6)
Slide 10: Patients achieved rapid neutrophil and platelet engraftment following treatment with risto-cel
Patients achieved rapid neutrophil and platelet engraftment following treatment with risto-cel (N=31) All treated population Treatment characteristics N=31* Median (range) risto-cel dose infused, ×106 viable CD34+ cells/kg 6.2 (3.2–23.4) Neutrophil engraftment N=28† Median (range) time to neutrophil
Slide 11: The safety profile of the risto-cel treatment regimen is consistent with busulfan conditioning
The safety profile of the risto-cel treatment regimen is consistent with busulfan conditioning, autologous HSCT, and underlying SCD Most common TEAEs , n (%) N=31 Stomatitis 24 (77.4) Febrile neutropenia 22 (71.0) Patients with, n (%) N=31 Decreased appetite 10 (32.3) Any TEAEs 31 (100) Hypokalemia
Slide 12: Rapid and robust HbF induction with corresponding HbS reduction following risto-cel infusion
Patients achieved rapid and robust HbF induction with corresponding HbS reduction following risto-cel infusion (N=31) Patients achieved endogenous HbF >60% and HbS <40% by 1 month after risto-cel treatment that was sustained through follow up, suggesting resolution of anemia 22 HbF HbS HbA HbA2 Hb O
Slide 13: No patients experienced any investigator-reported sVOCs after engraftment
No patients (N=31) experienced any investigator-reported sVOCs after engraftment Investigator-reported sVOCs following treatment with risto-cel P sVOC, n P1 12 P2 11 P3 9 # P4 13 P5 7 P6 8 P7 12 * P8 6 P9 4 P10 21 P11 12 P12 5 P13 14 ‡ P14 6 P15 4 P16 7 * Visit Poster 2532 for more P17 16 P18 9 BEAC
Slide 14: Conclusions
Conclusions ► Risto-cel’s automated, closed, and wholly owned manufacturing process ensured robust CD34+ cell yield and high editing efficiency with no manufacturing failures – Median time from start of first mobilization to drug product release was 2.9 (range 2.2–7.8) months ► Efficacy and safety r
Slide 15: Acknowledgments
Acknowledgments Thank you to the study participants, their families, and their caregivers for their participation, and the study investigators for their contributions Ashish O Gupta, MD, MPH Matthew M Heeney, MD University of Minnesota Boston Children’s Hospital Rabi Hanna, MD Cleveland Clinic Mary