Exhibit 99.1
Beam Therapeutics Announces New Data from BEACON Phase 1/2 Clinical Trial of BEAM-101 in Sickle Cell Disease at American Society of Hematology (ASH) Annual Meeting
All Seven Patients Treated with BEAM-101 Achieved Hemoglobin F (HbF) Induction of >60%, Hemoglobin S (HbS) Reduction to <40%, and Resolution of Anemia Post-BEAM-101 Treatment
Initial Safety Profile Consistent with Busulfan Conditioning and Autologous Hematopoietic Stem Cell Transplantation
All Seven Patients Dosed Achieved Target Cell Dose with One or Two Mobilization Cycles and Experienced Rapid Neutrophil and Platelet Engraftment
Markers of Hemolysis Normalized or Improved in All Patients
Beam to Host Investor Event on Dec. 8, 2024, at 8 p.m. PT
San Diego, December 7, 2024 – Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced new safety and efficacy data from its BEACON Phase 1/2 clinical trial of BEAM-101 in patients with sickle cell disease (SCD) with severe vaso-occlusive crises (VOCs). The data were featured today in the press program for the 66th American Society of Hematology (ASH) Annual Meeting and Exposition in San Diego and will be presented in an oral session on Sunday, Dec. 8, 2024, at 10 a.m. PT.
Consistent with Beam’s previously announced data, updated data from seven patients treated with investigational base-editing therapy BEAM-101 demonstrated robust and durable increases in fetal hemoglobin (HbF) and reductions in sickle hemoglobin (HbS), rapid neutrophil and platelet engraftment, and normalized or improved markers of hemolysis. No VOCs were reported post-engraftment. A summary of the results from the ongoing clinical study is provided below.
“These initial data from the BEACON trial are very encouraging and highlight the potential of BEAM-101 to deliver meaningful clinical benefits to patients with severe sickle cell disease,” said Matthew M. Heeney, M.D., associate chief of hematology at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center. “The data from the first seven patients demonstrate the ability for BEAM-101 to dramatically modify the hemoglobin profile to express a majority of protective fetal hemoglobin. All patients mobilized efficiently and had rapid engraftment with a low number of neutropenic days. I look forward to the continued maturation of the data to provide further insights into the long-term benefits of BEAM-101 for people living with sickle cell disease.”
“It’s an honor to share the initial results from BEACON with the hematology community at the ASH Annual Meeting, where there is broad recognition of the significant burden that sickle cell disease places on patients and their families,” said John Evans, chief executive officer of Beam. “We believe these early data for BEAM-101 are a testament to the potential of our base-editing