공시 • Apr 02

Beam Therapeutics Publishes Beacon Phase 1/2 Data for Risto-Cel in Patients with Sickle Cell Disease

Beam Therapeutics Inc. announced the publication of data from the ongoing Phase 1/2 BEACON clinical trial evaluating ristoglogene autogetemcel (risto-cel, formerly known as BEAM-101) for the treatment of sickle cell disease (SCD) with severe vaso-occlusive crises (VOCs) in The New England Journal of Medicine. The data included in the NEJM publication were most recently presented at the 67th American Society of Hematology (ASH) Annual Meeting and Exposition and continue to show evidence of risto-cel’s differentiated treatment profile. As of the August 6, 2025, data cut-off, a total of 31 patients with SCD with severe VOCs were treated with risto-cel in the BEACON Phase 1/2 trial and are included in the safety and efficacy analysis. Follow-up ranged from 0.3 to 20.4 months. Risto-cel’s efficient cell collection and manufacturing processes, combined with high, predictable yields from base editing, resulted in patients requiring a median of one stem cell collection cycle to manufacture risto-cel. The median time from start of cell collection to drug product release was 2.9 months, and the median time from start of cell collection to dosing was 4.5 months. Patients achieved rapid and robust bone marrow reconstitution post-risto-cel treatment, and no patients experienced any investigator-reported severe VOCs post-engraftment. Patients achieved mean hemoglobin F (HbF) levels above 60% and a mean durable reduction in corresponding hemoglobin S (HbS) below 40%. Total Hb levels increased rapidly with all patients experiencing resolution of anemia after elimination of the transfused blood, and key markers of hemolysis normalized or improved in all patients following risto-cel treatment. Sickling parameters all decreased in the blood following risto-cel treatment to levels comparable to those seen in individuals with sickle cell trait. The safety profile of risto-cel was consistent with busulfan conditioning, autologous hematopoietic stem cell transplantation (HSCT) and underlying SCD. The adult and adolescent cohorts of the BEACON trial were fully enrolled in mid-2025 and manufacturing of all doses was completed as of December 2025. The FDA previously granted risto-cel orphan drug designation and Regenerative Medicine Advanced Therapy (RMAT) designation. Risto-cel has also been accepted into the FDA’s Chemistry, Manufacturing, and Controls Development and Readiness Pilot (CDRP) program. The full publication, “Base Editing of HBG1 and HBG2 Promoters for Sickle Cell Disease,” appears in the April 1, 2026, issue of The New England Journal of Medicine. Risto-cel is an investigational genetically modified cell therapy for the treatment of 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 promoter regions of the HBG1/2 genes and are administered via a hematopoietic stem cell transplant procedure. The risto-cel 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 risto-cel are being evaluated in the ongoing BEACON Phase 1/2 study, an open-label, single-arm, multicenter trial in patients with SCD with severe vaso-occlusive crises (VOCs). Sickle cell disease (SCD), a severe inherited blood disease, is caused by a single point mutation, E6V, in the beta globin gene. This mutation causes the mutated form of sickle hemoglobin (HbS) to aggregate into long, rigid molecules that bend red blood cells into a sickle shape under conditions of low oxygen. Sickled cells obstruct blood vessels and die prematurely, ultimately resulting in anemia, severe pain (crises), infections, stroke, organ failure and early death. SCD is the most common inherited blood disorder in the United States, affecting an estimated 100,000 individuals within the U.S. and approximately eight million people worldwide.

공시 • Mar 25

Beam Therapeutics Announces Compelling Updated Clinical Data from the Ongoing Phase 1/2 Trial of BEAM-302 in Alpha-1 Antitrypsin Deficiency

Beam Therapeutics Inc. announced updated safety and efficacy data from the ongoing Phase 1/2 trial of BEAM-302 and the selection of 60 mg as the optimal biological dose to advance into pivotal development to support potential accelerated approval. BEAM-302 is a liver-targeting lipid-nanoparticle (LNP) formulation designed to directly correct the underlying genetic mutation that causes the severe form of alpha-1 antitrypsin deficiency (AATD) through base editing. BEAM-302 is being evaluated in a Phase 1/2, open-label, dose exploration and dose expansion clinical trial to investigate its safety, tolerability, pharmacodynamics, pharmacokinetics and efficacy. Part A of the trial is designed to evaluate patients with AATD-associated lung disease, while Part B evaluates patients with mild to severe liver disease, with or without lung disease. As of the February 10, 2026 data cutoff date, 29 patients have been treated with BEAM-302 in Part A (15 mg, n=3; 30 mg, n=3; 60 mg, n=6; 75 mg, n=9; 2x 60 mg, n=3) and Part B (30 mg, n=3; 60 mg, n=2) and followed for up to 18 months. Data from 26 patients treated with single-dose BEAM-302 support a well-tolerated safety profile up to 75 mg that is consistent across Part A and Part B. Adverse events (AEs) were mild to moderate, with no serious AEs reported and no dose-limiting toxicities as of the data cutoff. Transient Grade 1 and Grade 2 infusion-related reactions (IRRs) and Grade 1 asymptomatic alanine transaminase (ALT) and aspartate aminotransferase (AST) elevations were observed. In the multi-dose cohort (n=3), following the second dose of BEAM-302, patients experienced Grade 2 IRRs, one patient had Grade 4 ALT and Grade 3 AST elevations, and one patient had a Grade 2 ALT elevation. All ALT/AST elevations were asymptomatic and did not require treatment. No bilirubin increases were observed in any patient. Treatment with BEAM-302 led to rapid and durable increases of total and functional AAT, decreases in mutant Z-AAT, and new production of corrected M-AAT. Key data from 28 efficacy evaluable patients include the following: After treatment with a single dose of BEAM-302 in Part A, the steady-state circulating total AAT mean (LC-MS) was 16.1 µM in the 60 mg cohort (follow-up ranging from 5-12 months) and 14.4 µM in the 75 mg cohort (follow-up ranging from 2-9 months). In the multi-dose cohort, patients achieved a mean of 16.5 µM total AAT at Day 84, 28 days after the second 60 mg dose. These early data suggest a single dose of 60 mg BEAM-302 has achieved near saturation editing. Across all cohorts, increased total AAT in circulation was functional as demonstrated by a neutrophil elastase inhibition assay. Mutant Z-AAT was durably and significantly reduced after treatment with BEAM-302. The steady-state mean reduction in Z-AAT was 84% in the 60 mg cohort and 79% in the 75 mg cohort. In the multi-dose cohort, the mean reduction in Z-AAT was 80% at Day 84. Evidence of dynamic induction of AAT expression was observed during a respiratory infection around Month 8 in a patient in the 60 mg Part A cohort. During the infection, total AAT levels increased from steady-state levels of 15.9 µM to 29.5 µM while maintaining consistent AAT composition of 95% M-AAT. Following treatment with BEAM-302, newly produced corrected M-AAT comprised the majority of AAT in circulation. The steady-state mean proportion of M-AAT was 94% in the 60 mg cohort and 91% in the 75 mg cohort. In the multi-dose cohort, the mean proportion of M-AAT was 93% at Day 84. In Part B patients with AATD-associated liver disease, single doses of 30 mg and 60 mg BEAM-302 demonstrated consistent efficacy comparable to results observed in Part A patients without liver disease. Based on feedback from the U.S. Food and Drug Administration (FDA), Beam intends to pursue an accelerated approval pathway for BEAM-302 based on a primary endpoint of AAT biomarkers evaluated over 12 months, with 60 mg as the selected dose. To support a future biologics licensing application (BLA) submission, the company anticipates enrolling approximately 50 additional patients with AATD-associated lung disease, with or without liver disease, in an expansion of the ongoing open-label Phase 1/2 trial. Beam expects to initiate the pivotal cohort in the second half of 2026, leveraging its existing global clinical trial network.

총 주주 수익률	-0.3%
미래 배당 수익률	0%
배당 성장률	n/a
다음 배당 지급일	n/a
배당락일	n/a
주당 배당금	n/a
배당 성향	n/a

구분	배당 수익률
회사 (BEAM)	n/a
시장 하위 25% (US)	1.4%
시장 상위 25% (US)	4.3%
업계 평균 (Biotechs)	2.4%
분석가 예측 (BEAM) (최대 3년)	0%

데이터	최종 업데이트 (UTC 시간)
기업 분석	2026/05/21 04:31
종가	2026/05/21 00:00
수익	2026/03/31
연간 수익	2025/12/31

패키지	데이터	기간	미국 소스 예시 ^*
기업 재무제표	10년	손익계산서 현금흐름표 대차대조표	SEC 양식 10-K SEC 양식 10-Q
분석가 컨센서스 추정치	+3년	재무 예측 분석가 목표주가	분석가 리서치 보고서 Blue Matrix
시장 가격	30년	주가 배당, 분할 및 기타 조치	ICE 시장 데이터 SEC 양식 S-1
지분 구조	10년	주요 주주 내부자 거래	SEC 양식 4 SEC 양식 13D
경영진	10년	리더십 팀 이사회	SEC 양식 10-K SEC 양식 DEF 14A
주요 개발	10년	회사 공시	SEC 양식 8-K

분석가	기관
William Pickering	Bernstein
Alec Stranahan	BofA Global Research
Whitney Ijem	Canaccord Genuity

Beam Therapeutics 배당 및 자사주 매입

배당 기준 점검 0/6

핵심 정보

최근 배당 및 자사주 매입 업데이트

Recent updates

Beam Therapeutics Inc. Presents Topline Clinical Data for BEAM-302 in Alpha-1 Antitrypsin Deficiency At American Thoracic Society International Conference

Beam Therapeutics Targets Accelerated FDA Path for Gene-Editing Therapy as US$40 Million Investment Arrives

Consensus revenue estimates increase by 49%, EPS downgraded

BEAM: Liver Editing Platform And Non Dilutive Funding Will Drive Future Repricing

First quarter 2026 earnings released: US$0.91 loss per share (vs US$1.24 loss in 1Q 2025)

BEAM: Liver Editing Pipeline And Regulatory Momentum Will Drive Future Repricing

Beam Therapeutics Inc., Annual General Meeting, Jun 03, 2026

BEAM: FDA Alignment And Liver Programs Will Drive Future Upside Repricing

CEO & Director notifies of intention to sell stock

Beam Therapeutics Publishes Beacon Phase 1/2 Data for Risto-Cel in Patients with Sickle Cell Disease

BEAM: FDA Alignment And Liver Programs Will Drive Future Repricing

Beam Therapeutics Announces Compelling Updated Clinical Data from the Ongoing Phase 1/2 Trial of BEAM-302 in Alpha-1 Antitrypsin Deficiency

BEAM: FDA Alignment And New Liver Programs Will Drive Future Repricing

Liver Base Editing Platform Will Transform Rare Disease Treatment Over The Long Term

Beam Therapeutics: Great Potential, But Patience Is Needed

BEAM: FDA Program Alignment On Sickle Cell Will Drive Future Repricing

Full year 2025 earnings: EPS and revenues exceed analyst expectations

New major risk - Revenue and earnings growth

President notifies of intention to sell stock

Base Editing Platform And PKU Umbrella Trials Will Struggle To Justify Long Runway

Beam Therapeutics Inc. announced that it expects to receive $500 million in funding

Price target increased by 8.4% to US$49.00

BEAM: FDA Program Alignment And Upcoming Data Readout Will Drive Repricing

Beam Therapeutics Inc. Announces Resignation of Carole Ho from the Board of Directors, Effective February 27, 2026

CEO & Director notifies of intention to sell stock

BEAM: FDA Alignment And Upcoming Pulmonary Data Will Drive Bullish Repricing

Senior VP notifies of intention to sell stock

Next Milestones For Beam in 2026.

Beam Therapeutics will revolutionize hospital throughput with breakthrough gene therapy

Beam Therapeutics Inc. Reports Updated Data from BEACON Phase 1/2 Trial of Ristoglogene Autogetemcel (risto-cel)

Beam Rewrites Gene Therapy Playbook with Base Editing Breakthrough

Incredible Buying Opportunity

New major risk - Revenue and earnings growth

Consensus revenue estimates fall by 14%

Third quarter 2025 earnings: EPS and revenues miss analyst expectations

The "Molecular Pencil": Why Beam's Technology is Built to Win

CEO & Director notifies of intention to sell stock

Advancing BEAM-101 And BEAM-302 Will Transform Treatment Of Hematology And Liver Genetic Diseases

Beam Therapeutics Inc. Announces U.S. FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to BEAM-101 for the Treatment of Sickle Cell Disease

Consensus revenue estimates decrease by 16%, EPS upgraded

New major risk - Revenue and earnings growth

Second quarter 2025 earnings: EPS exceeds analyst expectations while revenues lag behind

Is Beam Therapeutics (NASDAQ:BEAM) In A Good Position To Invest In Growth?

Beam Therapeutics Inc.(NasdaqGS:BEAM) dropped from Russell Small Cap Comp Value Index

Beam Therapeutics Inc. Announces New Data from BEACON Phase 1/2 Clinical Trial of BEAM-101 Supporting Differentiated Profile in Sickle Cell Disease (SCD) at European Hematology Association (EHA) 2025 Congress

Shareholders May Find It Hard To Justify Increasing Beam Therapeutics Inc.'s (NASDAQ:BEAM) CEO Compensation For Now

Beam Therapeutics Inc to Highlight New Data from BEAM-101 Program in Sickle Cell Disease at European Hematology Association 2025 Congress

Price target decreased by 7.2% to US$44.40

Beam Therapeutics Announces U.S. FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to BEAM-302 for the Treatment of Alpha-1 Antitrypsin Deficiency (AATD)

First quarter 2025 earnings: EPS and revenues miss analyst expectations

Beam Therapeutics Inc., Annual General Meeting, Jun 04, 2025

New minor risk - Shareholder dilution

Beam Therapeutics Inc. Presents Additional Data for Beam-302 in Alpha-1 Antitrypsin Deficiency At 2025 Alpha-1 Foundation 7Th Global Research Conference and 10Th Patient Congress

New minor risk - Share price stability

Chief Medical Officer notifies of intention to sell stock

Beam Therapeutics Inc. Announces Clearance of Investigational New Drug Application for BEAM-302 for the Treatment of Alpha-1 Antitrypsin Deficiency (AATD) by the United States (U.S.) Food and Drug Administration

Advancing BEAM-101 And BEAM-302 Will Transform Treatment Of Hematology And Liver Genetic Diseases

Upgrading Beam Therapeutics: Promising BEAM-302 Data Opens Door For Durable Gene Therapy

Beam Therapeutics Inc. has completed a Follow-on Equity Offering in the amount of $500.000026 million.

Beam Therapeutics: Single-Base Editing Seems To Work

Beam Therapeutics Inc. Announces Positive Initial Data for BEAM-302 in the Phase 1/2 Trial in Alpha-1 Antitrypsin Deficiency

Full year 2024 earnings: EPS and revenues exceed analyst expectations

Senior VP notifies of intention to sell stock

Beam Therapeutics Inc.'s (NASDAQ:BEAM) Prospects Need A Boost To Lift Shares

Beam Therapeutics Inc. to Encore Data from Beacon Phase 1/2 Clinical Trial of Beam-101 in Sickle Cell Disease At 2025

Beam Therapeutics: Too Expensive For Me, But Watch For Important Catalysts

New minor risk - Share price stability

Here's Why We're Not At All Concerned With Beam Therapeutics' (NASDAQ:BEAM) Cash Burn Situation

Beam Therapeutics Inc. 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

Beam Therapeutics Inc. Appoints Sravan K. Emany as Chief Financial Officer, Effective December 19, 2024

Beam Therapeutics Inc. Announces the Appointment of Chirfi Guindo to Its Board of Directors

Beam Therapeutics: Additional BEAM-101 And ESCAPE Tech Data At ASH 2024 Makes This A Watch

Third quarter 2024 earnings: EPS misses analyst expectations

Insufficient Growth At Beam Therapeutics Inc. (NASDAQ:BEAM) Hampers Share Price

President notifies of intention to sell stock