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Beam Therapeutics to Present Data Across Hematology Franchise, Including First Clinical Data for BEAM-101 in Sickle Cell Disease and ESCAPE Non-human Primate Data, at American Society of Hematology (ASH) Annual Meeting

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Beam Therapeutics announced multiple presentations at the ASH Annual Meeting, including first clinical data from its BEAM-101 program for sickle cell disease. Initial results from the BEACON Phase 1/2 trial showed promising safety profile and efficacy in treated patients, with rapid fetal hemoglobin induction and sickle hemoglobin reduction. The company also presented positive preclinical data for its ESCAPE program, demonstrating successful non-genotoxic antibody-based conditioning in non-human primates. Additionally, preliminary data from the BEAM-201 trial showed complete response in 2 of 3 patients treated for T-cell lymphoblastic conditions.

Beam Therapeutics ha annunciato diverse presentazioni durante l'Assemblea Annuale ASH, inclusi i primi dati clinici del suo programma BEAM-101 per la malattia falciforme. I primi risultati del trial BEACON di Fase 1/2 hanno mostrato un profilo di sicurezza promettente e un'efficacia nei pazienti trattati, con una rapida induzione di emoglobina fetale e riduzione di emoglobina falciforme. L'azienda ha anche presentato dati preclinici positivi per il suo programma ESCAPE, dimostrando un efficace condizionamento basato su anticorpi non genotossici nei primati non umani. Inoltre, i dati preliminari del trial BEAM-201 hanno mostrato una risposta completa in 2 dei 3 pazienti trattati per condizioni di linfoblastosi T.

Beam Therapeutics anunció múltiples presentaciones en la Reunión Anual ASH, incluidas las primeras datos clínicos de su programa BEAM-101 para la enfermedad de células falciformes. Los resultados iniciales del ensayo BEACON de Fase 1/2 mostraron un perfil de seguridad prometedor y eficacia en los pacientes tratados, con rápida inducción de hemoglobina fetal y reducción de hemoglobina falciforme. La compañía también presentó datos preclínicos positivos para su programa ESCAPE, demostrando un exitoso acondicionamiento basado en anticuerpos no genotóxicos en primates no humanos. Además, los datos preliminares del ensayo BEAM-201 mostraron una respuesta completa en 2 de 3 pacientes tratados por condiciones de linfoblastosis T.

Beam Therapeutics는 ASH 연례 회의에서 여러 발표를 하였으며, 그 중에는 겸형 적혈구병에 대한 BEAM-101 프로그램의 첫 번째 임상 데이터도 포함되어 있습니다. BEACON 1/2상 시험의 초기 결과는 치료받은 환자에서 유망한 안전성 프로필과 효능을 보여주었으며, 빠른 태아 헤모글로빈 유도 및 겸형 헤모글로빈 감소가 관찰되었습니다. 회사는 또한 비유전독성 항체 기반의 비임상 조건을 성공적으로 적용한 ESCAPE 프로그램에 대한 긍정적인 전임상 데이터를 발표했습니다. 추가로, BEAM-201 시험의 초기 데이터는 T세포 림프oblastosis 상태에서 치료받은 3명 중 2명에서 완전 응답을 보였습니다.

Beam Therapeutics a annoncé plusieurs présentations lors de la Réunion Annuelle de l'ASH, y compris les premières données cliniques de son programme BEAM-101 pour la drépanocytose. Les résultats initiaux de l'essai BEACON de Phase 1/2 ont montré un profil de sécurité prometteur et une efficacité chez les patients traités, avec une induction rapide de l'hémoglobine fœtale et une réduction de l'hémoglobine S. L'entreprise a également présenté des données précliniques positives pour son programme ESCAPE, démontrant un conditionnement basé sur des anticorps non génotoxiques chez des primates non humains. De plus, des données préliminaires de l'essai BEAM-201 ont montré une réponse complète chez 2 des 3 patients traités pour des conditions de lymphoblastose T.

Beam Therapeutics gab mehrere Präsentationen auf dem ASH-Jahrestreffen bekannt, darunter die ersten klinischen Daten aus seinem BEAM-101-Programm für Sichelzellenanämie. Die ersten Ergebnisse der BEACON-Phase-1/2-Studie zeigten ein vielversprechendes Sicherheitsprofil und eine Wirksamkeit bei den behandelten Patienten, mit schneller Induktion von fethemoglobin und Reduzierung von sichelhemoglobin. Das Unternehmen präsentierte auch positive präklinische Daten für sein ESCAPE-Programm, die eine erfolgreiche nicht-genotoxische Antikörper-basierte Konditionierung bei nichtmenschlichen Primaten zeigten. Darüber hinaus zeigten vorläufige Daten der BEAM-201-Studie eine vollständige Reaktion bei 2 von 3 Patienten, die wegen T-Zell-Lymphoblastosen behandelt wurden.

Positive
  • Initial BEAM-101 trial showed rapid fetal hemoglobin induction (>60%) and sickle hemoglobin reduction (≤36%) in all 4 patients with ≥1 month follow-up
  • No vaso-occlusive crises reported post-treatment in BEAM-101 patients
  • BEAM-201 achieved complete response in 2 of 3 patients at CAR-T cell doses <200 million
  • ESCAPE program demonstrated 85% F-cells and ~55% HbF levels at 35 weeks post-transplant in non-human primates
Negative
  • One patient death reported four months after BEAM-101 infusion due to respiratory failure (related to busulfan conditioning)
  • Multiple mobilization cycles required for some patients to achieve target cell dose (mean: 1.5 cycles)

Insights

Initial clinical data for BEAM-101 in sickle cell disease shows promising results. Key findings include: rapid HbF induction >60% and sickle hemoglobin reduction to ≤36% by Month 1 in treated patients. The safety profile aligns with standard conditioning protocols, though one patient death occurred due to conditioning-related complications.

The ESCAPE program demonstrated significant progress in non-human primates, achieving ~85% F-cells and ~55% HbF levels at 35 weeks post-transplant without chemotherapy. This represents a potential breakthrough in eliminating genotoxic conditioning.

BEAM-201's early data in T-cell malignancies shows complete responses in 2 of 3 patients, suggesting efficacy at relatively low doses. The multiplex base editing approach appears viable for developing off-the-shelf CAR-T therapies.

The data validates Beam's base editing platform across multiple applications. In sickle cell disease, the editing efficiency and uniform HbF induction suggest potential advantages over existing gene therapy approaches. The ESCAPE platform's success with antibody-based conditioning could revolutionize stem cell transplantation by eliminating toxic chemotherapy.

The successful demonstration of quadruplex editing in BEAM-201 is technically significant, showing the platform can handle complex multiplex editing for cell therapy applications. This capability could enable more sophisticated genetic modifications for future therapeutic development.

Initial Results from BEACON Phase 1/2 Clinical Trial Demonstrate Potential for Differentiation of Base Editing and BEAM-101

Preclinical ESCAPE Data Establish Proof-of-concept for Non-genotoxic, Antibody-based Conditioning and Engraftment in Non-human Primates

Clinical Data from Phase 1/2 BEAM-201 Trial Demonstrate Therapeutic Potential of First Quadruplex-edited Allogeneic CAR-T Cell Therapy

Beam to Host Investor Event on Dec. 8, 2024, at 8 p.m. PT

CAMBRIDGE, Mass., Nov. 05, 2024 (GLOBE NEWSWIRE) --  Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced the acceptance of multiple oral and poster presentations, including the first clinical data from a Beam program, at the 66th American Society of Hematology (ASH) Annual Meeting and Exposition, taking place December 7-10, 2024, in San Diego.

“For people living with severe sickle cell disease, a serious medical condition with reduced life expectancy, stem cell transplant with genotoxic chemotherapy is the only currently available curative option. At Beam, we have a long-term commitment to delivering better treatments for these patients, first with BEAM-101 as a potentially superior autologous cell product, followed by ESCAPE, which seeks to eliminate chemotherapy from the transplant process altogether,” said John Evans, chief executive officer of Beam. “Today represents an important milestone toward this vision as we unveil data with our base editing technology across both approaches to treating sickle cell disease. The initial results for BEAM-101 provide emerging clinical validation of base editing and of our preclinical hypothesis that more precise and efficient editing, without double-stranded DNA breaks, can lead to a differentiated product profile, with greater and more uniform induction of fetal hemoglobin, deeper reduction of sickle hemoglobin, and potentially faster engraftment.”

“Our proof-of-concept data for ESCAPE in non-human primates demonstrate that base editing could enable antibody conditioning and engraftment for stem cell transplant without chemotherapy, a potential breakthrough in the field of hematology and for patients,” said Giuseppe Ciaramella, Ph.D., president of Beam. “Along with the strong translation from preclinical to clinical of our BEAM-101 program, these data reflect the potential of base editing to enable new therapeutic possibilities for people suffering from serious diseases.”

BEAM-101 Oral and Poster Presentations

Title: Initial Results from the BEACON Clinical Study: A Phase 1/2 Study Evaluating the Safety and Efficacy of a Single Dose of Autologous CD34+ Base Edited Hematopoietic Stem Cells (BEAM-101) in Patients with Sickle Cell Disease with Severe Vaso-Occlusive Crises
Abstract: 513
Oral Session: 801. Gene Therapies: Gene Editing and Replacement Therapies for Hemoglobinopathies: From Bench to Bedside
Presentation Time: Sunday, Dec. 8, 2024, at 10 a.m. PT
Location: San Diego Convention Center, Room 30
Presenter: Matthew M. Heeney, M.D., Dana-Farber/Boston Children’s Cancer and Blood Disorders Center
Key Highlights:

  • Preliminary data as of a July 2, 2024, data cut from BEACON, a Phase 1/2 single-arm, open-label clinical trial evaluating the safety and efficacy of a single dose of BEAM-101 in patients with sickle cell disease (SCD) with severe vaso-occlusive crises (VOCs).
  • Initial safety profile was consistent with busulfan conditioning and autologous hematopoietic stem cell transplantation (HSCT).
    • In all patients dosed (n=6), there were no ≥Grade 3 adverse events (AEs) or serious AEs related to treatment with BEAM-101.
    • One patient died four months after BEAM-101 infusion due to respiratory failure that was determined by the investigator to be likely related to busulfan conditioning and deemed unrelated to BEAM-101. The case was reviewed by the Data Safety Monitoring Committee and the U.S. Food and Drug Administration. Pulmonary complications are a known cause of morbidity and, in rare cases, mortality in patients undergoing myeloablation with chemotherapy, such as busulfan, and stem cell transplantation.
  • All patients dosed achieved their target cell dose with either 1 or 2 mobilization cycles (mean: 1.5).
  • All 4 patients with ≥1 month of follow-up achieved neutrophil and platelet engraftment at a median of 17 (15–19) and 20 (11–34) days, respectively.
  • All 4 patients experienced rapid and robust fetal hemoglobin (HbF) induction by Month 1 (>60%) and corresponding sickle hemoglobin (HbS) reduction (≤36%) in non-transfused blood, which was sustained over time.
  • Markers of hemolysis normalized or improved for all 4 patients.
  • No VOCs were reported by investigators post-treatment.
  • These data support base editing of the HBG1/2 promoters as a therapeutic modality for the treatment of SCD and the ongoing development of BEAM-101.
  • The presentation at ASH will include additional data with more patients and longer follow-up.

Title: Impact of BEAM-101 Treatment on Red Blood Cell Hemoglobin Expression, Rheology and Sickling Properties: Initial data from the BEACON Phase 1/2 study of Autologous CD34+ Base Edited Hematopoietic Stem Cells in Sickle Cell Disease
Abstract: 4957
Poster Session: 801. Gene Therapies: Poster III
Session Time: Monday, Dec. 9, 2024, from 6-8 p.m. PT
Location: San Diego Convention Center, Halls G-H
Presenter: Priya S. Chockalingam, Ph.D., Beam Therapeutics
Key Highlights:

  • Preliminary data as of a July 2, 2024, data cut includes exploratory biomarker assessments of red blood cell (RBC) hemoglobin expression, health and function in 3 patients for two or more of the following visits: screening, Month 1, 2, 3 and 6.
  • Initial results demonstrated 98-99% of RBCs expressing HbF as early as Month 1, with near complete elimination of RBCs expressing solely HbS post-treatment with BEAM-101.
  • The data showed reduction in RBC sickling, comparable to sickle cell trait, reduced cell adhesion and improved hemorheological properties of blood post-treatment with BEAM-101.
  • The presentation at ASH will include additional data with more patients and longer follow-up.

ESCAPE Oral Presentation:

Title: CD117 Antibody Conditioning and Multiplex Base Editing Enable Rapid and Robust Fetal Hemoglobin Reactivation in a Rhesus Autologous Transplantation Model
Abstract: 516
Oral Session: 801. Gene Therapies: Gene Editing and Replacement Therapies for Hemoglobinopathies: From Bench to Bedside
Presentation Time: Sunday, Dec. 8, 2024, at 10:45 a.m. PT
Location: San Diego Convention Center, Room 30
Presenter: Selami Demirci, Ph.D., National Institutes of Health
Key Highlights:

  • Study investigated whether Beam’s Engineered Stem Cell Antibody Evasion (ESCAPE) approach, using CD117 monoclonal antibody (mAb) conditioning, could successfully achieve long-term engraftment and induce robust levels of HbF in an immunocompetent host. Researchers used a non-human primate (NHP) model where autologous CD34+ hematopoietic stem and progenitor cells were multiplex edited to target a single epitope on CD117 and the promoter regions of HBG1/2, allowing cells to avoid detection by the conditioning mAb while upregulating HbF.
  • Two NHPs were conditioned with different doses (10 mg/kg and 25 mg/kg) of CD117 mAb, instead of busulfan, 7 days prior to transplantation. Post-transplant, further mAb treatments were administered to maintain competitive advantage for the edited cells.
  • mAb-based conditioning was well tolerated, with no need for NHP supportive care.
  • Both NHPs showed rapid and significant induction of HbF. The percentage of red blood cells expressing HbF (F-cells) reached 61% as early as 8 weeks post-transplant in one NHP and stabilized at ~85% by 35 weeks in both animals. Concurrently, levels of HbF increased rapidly, reaching ~55% at 35 weeks post-transplant in both animals.
  • Data suggest that ESCAPE, combined with CD117 mAb conditioning and selection, can achieve long-term engraftment and induce high levels of HbF, offering a promising alternative to traditional genotoxic conditioning in autologous HSCT.
  • The presentation at ASH will include additional data.

BEAM-201 Poster Presentation:

Title: BEAM-201 for the Treatment of Relapsed and/or Refractory (R/R) T-Cell Acute Lymphoblastic Leukemia (T-ALL) or T-Cell Lymphoblastic Lymphoma (T-LL): Initial Data from the Phase (Ph) 1/2 Dose-Exploration, Dose-Expansion, Safety, and Efficacy Study of Multiplex Base-Edited Allogeneic Anti CD7 CAR-T-Cells
Abstract: 4838
Poster Session: 704. Cellular Immunotherapies: Early Phase Clinical Trials and Toxicities: Poster III
Session Time: Monday, Dec. 9, 2024, from 6-8 p.m. PT
Location: San Diego Convention Center, Halls G-H
Presenter: Caroline Diorio, M.D., FRCPC, FAAP, Children’s Hospital of Philadelphia
Key Highlights:

  • Initial data as of a June 11, 2024, data cut in 3 patients treated with BEAM-201 show a safety profile consistent with underlying disease, lymphodepletion and AEs associated with CAR-T therapy.
  • A complete response (CRi/CR) was demonstrated in 2 of 3 patients at CAR-T cell doses <200 million. Both patients achieving a CRi/CR were deemed suitable for stem cell transplant following therapy.
  • The presentation at ASH will include additional data with more patients and longer follow-up.

ASH Investor Event Information
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.

About Beam Therapeutics
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, T-ALL/T-LL, and ESCAPE; our plans, and anticipated timing, to advance our programs; the clinical trial designs and expectations for BEAM-101, BEAM-201, and ESCAPE; our potential 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 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.

Contacts:

Investors:
Holly Manning
Beam Therapeutics
hmanning@beamtx.com

Media:
Dan Budwick
1AB
dan@1abmedia.com


FAQ

What were the initial results of BEAM-101 in the BEACON Phase 1/2 trial for sickle cell disease?

Initial results showed rapid fetal hemoglobin induction (>60%) and sickle hemoglobin reduction (≤36%) in all 4 patients with ≥1 month follow-up, with no vaso-occlusive crises reported post-treatment.

How effective was BEAM-201 in treating T-cell lymphoblastic conditions?

BEAM-201 demonstrated complete response (CRi/CR) in 2 out of 3 treated patients at CAR-T cell doses below 200 million, with both responding patients becoming eligible for stem cell transplant.

What were the key findings from Beam Therapeutics' ESCAPE program in non-human primates?

The ESCAPE program showed successful non-genotoxic antibody-based conditioning, achieving 85% F-cells and approximately 55% HbF levels at 35 weeks post-transplant in non-human primates.

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