Full Press Release Details
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
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
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
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.
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
technology to provide a differentiated option for sickle cell patients, having demonstrated a robust increase in fetal hemoglobin of >60%, a decrease in hemoglobin S to <40% and resolution
of anemia in all patients. Additionally, the data from our ESCAPE nongenotoxic conditioning program to be presented on Sunday highlight our commitment to expanding access to treatment by decreasing the burden and complications patients
potentially face when undergoing transplantation. We look forward to continuing to rapidly advance both programs for patients with sickle cell disease.
To date, more than 35 patients have cleared screening and enrolled in the BEACON Phase 1/2 clinical trial, and of these, 11 patients have been dosed with BEAM-101. As of an Oct. 28, 2024, data cut-off, a total of seven patients with severe SCD were treated with BEAM-101 and included in
the safety and efficacy analysis with follow up ranging from 1 to 11 months.
Key highlights include the following:
Beam will host a live and webcast investor event on Dec. 8, 2024, at 8:00 p.m. PT in San Diego to review the key presentations from this
year s ASH meeting. The event will be webcast live and can be accessed under Events & Presentations in the Investors section of the company s website at www.beamtx.com. The archived webcast will be available on the
company s website beginning approximately two hours after the event.
BEAM-101 is an investigational genetically modified cell therapy for the treatment of severe sickle cell disease (SCD).
The one-time therapy consists of autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) that have been base-edited in the promotor regions of the HBG1/2 genes and are administered via a
hematopoietic stem cell transplant procedure. The BEAM-101 edit is designed to inhibit the transcriptional repressor BCL11A from binding to the promoter without disrupting BCL11A expression, leading to
increased production of non-sickling and anti-sickling fetal hemoglobin (HbF) and thus mimicking the effects of naturally occurring variants seen in hereditary persistence of fetal hemoglobin. HbF is the
predominant hemoglobin variant during development and early life. The safety and efficacy of BEAM-101 is being evaluated in the ongoing BEACON Phase 1/2 study, an open-label,
single-arm, multicenter trial in adult patients with SCD with severe vaso-occlusive crises (VOCs).
Beam Therapeutics (Nasdaq: BEAM) is a biotechnology company committed to establishing the leading, fully integrated platform for
precision genetic medicines. To achieve this vision, Beam has assembled a platform with integrated gene editing, delivery and internal manufacturing capabilities. Beam s suite of gene editing technologies is anchored by base editing, a
proprietary technology that is designed to enable precise, predictable and efficient single base changes, at targeted genomic sequences, without making double-stranded breaks in the DNA. This has the potential to enable a wide range of potential
therapeutic editing strategies that Beam is using to advance a diversified portfolio of base editing programs. Beam is a values-driven organization committed to its people, cutting-edge science, and a vision of providing life-long cures to patients
suffering from serious diseases.
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Investors are cautioned not
to place undue reliance on these forward-looking statements, including, but not limited to, statements related to: the therapeutic applications and potential of our technology, including with respect to SCD; our plans, and anticipated timing, to
advance our programs; the clinical trial designs and expectations for BEAM-101 and ESCAPE; our presentations at the ASH annual meeting; and our ability to develop life-long, curative, precision genetic
medicines for patients through base editing. Each forward-looking statement is subject to important risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement, including, without
limitation, risks and uncertainties related to: our ability to develop, obtain regulatory approval for, and commercialize our product candidates, which may take longer or cost more than planned; our ability to raise additional funding, which may not
be available; our ability to obtain, maintain and enforce patent and other intellectual property protection for our product candidates; the uncertainty that our product candidates will receive regulatory approval necessary to initiate human clinical
trials; that preclinical testing of our product candidates and preliminary or interim data from preclinical studies and clinical trials may not be predictive of the results or success of ongoing or later clinical trials; that initiation and
enrollment of, and anticipated timing to advance, our clinical trials may take longer than expected; that our product candidates or the
delivery modalities we rely on to administer them may cause serious adverse events; that our product candidates may experience manufacturing or supply interruptions or failures; risks related to
competitive products; and the other risks and uncertainties identified under the headings Risk Factors Summary and Risk Factors in our Annual Report on Form 10-K for the year ended
December 31, 2023, our Quarterly Reports on Form 10-Q for the quarterly period ended September 30, 2024 and in any subsequent filings with the Securities and Exchange Commission. These
forward-looking statements speak only as of the date of this press release. Factors or events that could cause our actual results to differ may emerge from time to time, and it is not possible for us to predict all of them. We undertake no
obligation to update any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by applicable law.