Duyuru • Sep 08

Beam Therapeutics Presents Updated Clinical Data from the Phase 1/2 Trial of Beam-302 in Alpha-1 Antitrypsin Deficiency

Beam Therapeutics announced updated clinical data from the Phase 1/2 trial evaluating BEAM-302 for alpha-1 antitrypsin deficiency (AATD). BEAM-302 is Beam’s lead liver-targeted genetic disease program and a potentially best- and first-in-class therapy designed to directly correct the root cause of AATD and address both liver and lung manifestations of disease. 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 is designed to evaluate patients with AATD-associated lung disease, and Part B evaluates patients with mild to severe liver disease, with or without lung disease. As of August 17, 2026, 38 patients have been dosed with BEAM-302 across Part A and Part B. The presentation at ERS included data from 29 patients treated with a single dose of BEAM-302 in the dose-escalation portion of the study: Part A (15 mg, n=3; 30 mg, n=3; 60 mg, n=6; 75 mg, n=9) and Part B (30 mg, n=3; 60 mg, n=5). Data from patients in the multi-dose cohort (n=3) and the Part A expansion cohort (n=6) were not included. As of the June 24, 2026, data cutoff date, treatment with single-dose BEAM-302 was well tolerated with an acceptable safety profile in both Part A and Part B cohorts. Mild-to-moderate infusion-related reactions (IRRs) were the most common drug-related treatment-emergent adverse events (TEAEs), occurring in 41% of all patients. Transient and predominantly Grade 1 alanine transaminase (ALT) and/or aspartate aminotransferase (AST) elevations were also observed. One Part B patient with underlying AATD-related liver disease treated with 60 mg BEAM-302 experienced transient Grade 3 ALT/AST elevations that were not accompanied by bilirubin increases and did not require treatment. Treatment with BEAM-302 led to rapid and durable increases of total and functional AAT, decreases in mutant Z-AAT and toxic Z-polymers, and new production of corrected M-AAT. Key findings from 29 efficacy-evaluable patients as of the June 24, 2026, data cutoff date include: A single dose of BEAM-302 of 60 mg led to sustained increases in total AAT above the 11 µM protective threshold. After treatment with a single dose of 60 mg BEAM-302 in Part A, the steady-state circulating total AAT mean and median were 14.4 µM and 15.2 µM, respectively, compared to 5.0 µM at baseline. In the Part B 60 mg cohort, the post-treatment steady-state circulating total AAT mean and median were 13.5 µM and 13.8 µM, respectively, compared to 4.7 µM at baseline. Increased total AAT in circulation was functional. Functional AAT levels increased in a dose-dependent manner up to 60 mg and were durable through follow-up. Human neutrophil elastase (HNE) activity levels, another measure of AAT function, decreased in a dose proportionate manner with BEAM-302 treatment, with >80% of patients in the Part A 60 mg cohort having at least one HNE activity measurement below the lower limit of quantification. Mutant Z-AAT was significantly and durably reduced after treatment with BEAM-302, with an 84% reduction in circulating Z-AAT in both Part A and Part B 60 mg cohorts. BEAM-302 also led to a significant decrease in circulating Z-AAT protein aggregates, referred to as Z-polymers, from baseline in the 60 mg cohort. Circulating Z-polymers have been shown to correlate with liver disease severity and to amplify lung inflammation by inducing neutrophil recruitment. Evidence of AAT protein production under normal physiologic control was observed during an upper respiratory tract infection in a patient in the Part A 60 mg cohort. Following treatment with BEAM-302, newly produced corrected M-AAT comprised the majority of AAT in circulation. The proportion of M-AAT at steady state was 93% following treatment with 60 mg in both Part A and Part B, exceeding the approximately 80% M-AAT proportion associated with the MZ genotype. 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 license 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. In July, Beam dosed the first patient in this global pivotal cohort. BEAM-302 is a liver-targeting lipid-nanoparticle (LNP) formulation of base editing reagents designed to correct the PiZ mutation. Patients homozygous for this mutation (PiZZ) represent the majority of patients living with severe AATD disease. A one-time A-to-G correction of the PiZ mutation with Beam’s adenine base editor has the potential to simultaneously reduce the aggregation of mutant, misfolded AAT protein that causes toxicity to the liver (Z-AAT), generate therapeutic levels of corrected protein (M-AAT), and increase total and functional AAT in circulation, thereby addressing the underlying pathophysiology of both the liver and lung disease. In addition, the reduction in circulating PiZ has the potential to further minimize lung inflammation and dysfunction. Importantly, because BEAM-302 corrects the native AAT gene in its normal genetic location, AAT levels have been observed to increase physiologically in response to infection and inflammation in treated patients. This is a critical aspect of AAT’s normal function to regulate the body’s inflammatory response, which does not occur with currently approved protein replacement therapies. Correction of the PiZ mutation has been durable to date in patients treated in Beam's clinical trial.

Duyuru • 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.

Toplam Hissedar Getirisi	-0.6%
Gelecekteki Temettü Verimi	0%
Temettü Büyümesi	n/a
Bir sonraki temettü ödeme tarihi	n/a
Eski temettü tarihi	n/a
Hisse başına temettü	n/a
Ödeme oranı	n/a

Segment	Temettü Verimi
Şirket (BEAM)	n/a
Pazarın Alt %25'i (US)	1.4%
Pazarın En İyi %25'i (US)	4.1%
Sektör Ortalaması (Biotechs)	2.2%
Analist tahmini (BEAM) (3 yıla kadar)	0%

Veri	Son Güncelleme (UTC saati)
Şirket Analizi	2026/09/11 07:06
Gün Sonu Hisse Fiyatı	2026/09/11 00:00
Kazançlar	2026/06/30
Yıllık Kazançlar	2025/12/31

Paket	Veri	Zaman Çerçevesi	Örnek ABD Kaynağı ^*
Şirket Finansalları	10 yıl	Gelir tablosu Nakit akış tablosu Bilanço	SEC Form 10-K SEC Form 10-Q
Analist Konsensüs Tahminleri	+3 yıl	Finansal tahminler Analist fiyat hedefleri	Analist Araştırma Raporları Blue Matrix
Piyasa Fiyatları	30 yıl	Hisse senedi fiyatları Temettüler, Bölünmeler ve Eylemler	ICE Piyasa Verileri SEC Form S-1
Sahiplik	10 yıl	En büyük hissedarlar İçeriden öğrenenlerin ticareti	SEC Form 4 SEC Form 13D
Yönetim	10 yıl	Liderlik ekibi Yönetim Kurulu	SEC Form 10-K SEC Form DEF 14A
Önemli Gelişmeler	10 yıl	Şirket duyuruları	SEC Form 8-K

Analist	Kurum
William Pickering	Bernstein
Alec Stranahan	BofA Global Research
Whitney Ijem	Canaccord Genuity

Beam Therapeutics Temettüler ve Geri Alımlar

Temettü kriter kontrolleri 0/6

Anahtar bilgiler

Son temettü ve geri alım güncellemeleri

Recent updates

Beam Therapeutics: Market Selling Phase 2 AATD Data Is Understandable, In My View

Beam Therapeutics Advances BEAM-302 Base-Editing Therapy Into Global Pivotal Trial for AATD

Beam Therapeutics Presents Updated Clinical Data from the Phase 1/2 Trial of Beam-302 in Alpha-1 Antitrypsin Deficiency

Beam Therapeutics Inc. Appoints Eric Foster as Chief Commercial Officer

Consensus revenue estimates fall by 38%

Analyst Forecasts Just Became More Bearish On Beam Therapeutics Inc. (NASDAQ:BEAM)

Analysts Have Lowered Expectations For Beam Therapeutics Inc. (NASDAQ:BEAM) After Its Latest Results

Beam Therapeutics Achieves Major Clinical Milestones With Key FDA Clearance and Trial Progress

Second quarter 2026 earnings: EPS and revenues miss analyst expectations

Chief Medical Officer notifies of intention to sell stock

CEO & Director notifies of intention to sell stock

BEAM: Strong Gene Editing Pipeline Will Likely Fail To Justify Current Hype

Beam Therapeutics Granted FDA Clearance to Advance BEAM-304 Gene Editing Trial for PKU

Beam Therapeutics Receives FDA Clearance for Investigational New Drug Application for BEAM-304 for the Treatment of Phenylketonuria

Beam Therapeutics Stock Advances BEAM-302 With Positive Phase 1/2 Data and Regulatory Progress

BEAM: Positive Gene Editing Data And Regulatory Tailwinds Will Drive Future Repricing

Beam Therapeutics Advances as BEAM-302 Data Supports Accelerated FDA Pathway and Market Optimism

BEAM: Lung And Liver Gene Editing Progress Will Drive Future Repricing

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

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: 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.