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Beam Therapeutics Reports Promising Data from BEACON Trial of BEAM-101 for Sickle Cell Disease
SAN DIEGO, Dec. 07, 2024 — Beam Therapeutics Inc. (Nasdaq: BEAM), a pioneering biotechnology company focused on precision genetic medicines, has released encouraging findings from its BEACON Phase 1/2 clinical trial investigating BEAM-101 in patients grappling with severe sickle cell disease (SCD) complications, specifically vaso-occlusive crises (VOCs). These results were highlighted at the 66th American Society of Hematology (ASH) Annual Meeting and Exposition in San Diego and are slated for a detailed presentation during an oral session on December 8, 2024, at 10 a.m. PT.
The data, showcasing treatment outcomes for seven patients administered with BEAM-101, reveal significant and sustained increases in fetal hemoglobin (HbF) levels, alongside reductions in sickle hemoglobin (HbS). Observations indicate that patients experienced efficient neutrophil and platelet engraftment, with markers of hemolysis showing normalization or improvement post-treatment. Notably, no vaso-occlusive crises were reported following the engraftment process.
Dr. Matthew M. Heeney, associate chief of hematology at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, expressed optimism regarding these initial findings. “These results are promising and underscore the potential of BEAM-101 to offer significant clinical advantages for patients suffering from severe sickle cell disease. The therapy has demonstrated its capacity to fundamentally change the hemoglobin profile by enhancing protective fetal hemoglobin,” he stated.
In commenting on the implications of these results, John Evans, CEO of Beam, emphasized their commitment to addressing the substantial challenges faced by sickle cell patients. “Presenting these early findings at the ASH Annual Meeting serves as an opportunity to engage with the hematology community, highlighting the considerable burden that sickle cell disease imposes on patients and families. We are encouraged by the demonstrated increase in HbF levels exceeding 60% and a decrease in HbS to below 40%, as well as the complete resolution of anemia in all participants,” Evans remarked.
As part of the ongoing BEACON trial, more than 35 patients have successfully cleared screening protocols, with 11 individuals having received BEAM-101 treatment as of October 28, 2024. Among these, seven patients were included in the detailed efficacy and safety analysis, with follow-up durations ranging from one to 11 months.
Key Findings from the BEACON Trial
Key highlights from the initial findings include:
Rapid and Sustained Increases in Protective Fetal Hemoglobin (HbF): All participants surpassed 60% in endogenous HbF levels, with a consistent reduction in HbS below 40%.
Robust and Sustained Total Hemoglobin (Hb) Levels: Participants showed rapid rises in total hemoglobin, leading to the resolution of anemia after eliminating transfused blood.
Efficient Cell Collection and Rapid Engraftment: All patients met the target cell dose requirement through one or two mobilization cycles. The mean time for neutrophil engraftment was recorded at 17.1 days, with a mean neutropenic duration of only 6.3 days.
Normalization of Hemolysis Markers: Key indicators of hemolysis improved or normalized in all treated patients following BEAM-101 administration.
Consistent Safety Profile: The safety observations for BEAM-101 mirrored those typically seen with busulfan conditioning and autologous hematopoietic stem cell transplantation (HSCT). Although some treatment-emergent adverse events were noted—like febrile neutropenia and stomatitis—no vaso-occlusive crises were observed after engraftment, underscoring the treatment’s tolerability.
Upcoming Investor Event
Investor Event Information
Beam will conduct a live and webcast investor event on December 8, 2024, at 8:00 p.m. PT, to discuss key presentations from the ASH annual meeting. This event can be accessed under the Events & Presentations section of the company’s website at www.beamtx.com.
About BEAM-101
BEAM-101 is an investigational genetically modified cell therapy tailored to treat severe sickle cell disease. This innovative therapy employs autologous CD34+ hematopoietic stem and progenitor cells that have been base-edited to enhance fetal hemoglobin production, consequently aiming to mitigate the symptoms of SCD. The ongoing BEACON Phase 1/2 study is designed to evaluate the safety and efficacy of this promising treatment in adult patients experiencing severe vaso-occlusive crises.
About Beam Therapeutics
Beam Therapeutics operates at the forefront of biotechnology, with a firm commitment to developing advanced precision genetic medicines. Anchored by its proprietary base editing technology, the company is well-equipped to perform targeted genomic modifications without inducing double-stranded breaks in DNA, thus presenting a versatile platform for therapeutic development.
The commitment of Beam Therapeutics to its scientific endeavors and objectives underscores its dedication to providing enduring and effective treatments for serious medical conditions.
Cautionary Note Regarding Forward-Looking Statements
This release includes forward-looking statements per the Private Securities Litigation Reform Act of 1995. These statements involve uncertainties and risks that may materially affect actual outcomes. Investors should be cautious about relying on these insights, which may encompass various complexities related to regulatory approval, trial outcomes, and market conditions.
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