Core Viewpoint - Beam Therapeutics has received orphan drug designation from the FDA for BEAM-302, a treatment aimed at correcting the genetic mutation responsible for alpha-1 antitrypsin deficiency (AATD), highlighting the urgency and potential of this therapy [1][2][3] Company Overview - Beam Therapeutics is a biotechnology company focused on precision genetic medicines, utilizing base editing technology to develop therapies [8] - The company aims to provide life-long cures for serious diseases through its integrated platform for gene editing, delivery, and manufacturing [8] Product Details - BEAM-302 is a liver-targeting lipid-nanoparticle formulation designed to correct the PiZ mutation associated with severe AATD [4] - The treatment aims to reduce the aggregation of misfolded AAT protein, generate therapeutic levels of corrected protein, and increase total AAT in circulation, addressing both liver and lung disease [4] - Initial Phase 1/2 trial results indicate BEAM-302 is well tolerated and shows durable, dose-dependent correction of the disease-causing mutation [3] Clinical Development - The FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to BEAM-302, indicating its potential as a transformative therapy for AATD [2][3] - The ongoing clinical trial has shown promising initial safety and efficacy data, with plans to report updated results at a medical conference in the second half of 2025 [3] Disease Context - AATD is an inherited genetic disorder that can lead to early onset emphysema and liver disease, with a significant unmet need for effective treatments [5][7] - Approximately 100,000 individuals in the U.S. are estimated to have the PiZZ genotype, but only about 10% are diagnosed [7]

Summary of Beam Therapeutics Conference Call Company Overview - **Company**: Beam Therapeutics (Ticker: BEAM) - **Event**: 2025 Global Health Care Conference - **Date**: May 20, 2025 - **Key Speaker**: John Evans, CEO Industry and Core Technology - **Industry**: Biotechnology, specifically focusing on gene editing and therapies - **Core Technology**: Base editing, a next-generation CRISPR technology that allows precise single base changes in genes without double-stranded breaks [3][4] Key Developments and Progress - **Clinical Progress**: - Multiple franchises in the base editing portfolio are clinically derisked with clear paths toward registration [2] - Successful data for sickle cell disease (B101) showing a differentiated profile compared to competitors [3] - First in vivo data for alpha-one antitrypsin deficiency (BEAM-302) demonstrating therapeutic thresholds [4] - First patient dosed for glycogen storage disease 1A (BEAM-301) [4] - **Financial Position**: Well-financed with successful capital financing, providing cash into 2028 to support ongoing projects [5] Regulatory Environment - **FDA Dynamics**: - New leadership at the FDA may impose a higher bar for approvals, but there is optimism about working with the FDA to bring effective therapies to market [7][10] - The company feels aligned with FDA's goals of addressing root causes of diseases [10][11] - Received RMAT designation, allowing for early engagement with the FDA regarding accelerated approval pathways [41][42] Safety and Efficacy - **Safety Profile**: - The company emphasizes a strong safety profile for its lipid nanoparticles (LNPs), differentiating from competitors [31][32] - Minimal liver toxicity observed in lung patients, allowing for future trials involving liver-involved patients [61][63] - **Efficacy Measures**: - Focus on total alpha-one antitrypsin levels and functional measures as endpoints for approval [47][48] - Plans to conduct biopsies and bronchoscopies to measure disease progression and treatment effects [49][50] Competitive Landscape - **Market Position**: - Monitoring competitors like Vertex and CRISPR, but confident in the differentiation of their data and therapies [66][68] - Plans to invest in next-generation therapies while maintaining the current pipeline [68] Future Outlook - **Next Steps**: - Engaging with the FDA over the next 6-12 months to refine the path to market and explore accelerated approval options [55][56] - Anticipating increased patient enrollment in trials to gather more data and demonstrate demand [57] Conclusion - Beam Therapeutics is positioned strongly within the biotechnology sector, leveraging its innovative base editing technology to address significant medical needs. The company is navigating a complex regulatory environment while maintaining a focus on safety and efficacy, with a clear strategy for future growth and market expansion.

Core Insights - Beam Therapeutics is presenting updated safety and efficacy data from the BEACON Phase 1/2 clinical trial of BEAM-101 for sickle cell disease at the EHA2025 Congress [1][2] - BEAM-101 is a genetically modified ex vivo cell therapy aimed at treating severe sickle cell disease, specifically targeting patients with severe vaso-occlusive crises [1][5] Company Overview - Beam Therapeutics is focused on developing precision genetic medicines through base editing technology, which allows for precise modifications in the genome without causing double-stranded breaks [7] - The company aims to provide lifelong cures for serious diseases, leveraging its integrated platform for gene editing, delivery, and manufacturing [7] Clinical Trial Details - The BEACON trial is an open-label, single-arm, multicenter study evaluating the safety and efficacy of BEAM-101 in adult patients with sickle cell disease [5] - The updated dataset includes results from 17 patients, highlighting the potential of BEAM-101 as a transformative one-time treatment for sickle cell disease [2][5] Presentation Information - The presentations at EHA2025 will cover various aspects of BEAM-101, including its safety, efficacy, and the health of red blood cells post-treatment [3][4] - The conference call and webcast to review key presentations will take place on June 13, 2025, at 4:00 p.m. ET [4]

Company Overview - Beam Therapeutics Inc. is a biotechnology company focused on developing precision genetic medicines through base editing [3] - The company aims to establish a leading, fully integrated platform for precision genetic medicines, incorporating gene editing, delivery, and internal manufacturing capabilities [3] - Beam's proprietary technology, base editing, allows for precise and efficient single base changes in targeted genomic sequences without causing double-stranded breaks in DNA [3] Upcoming Events - Management will participate in a fireside chat at the 2025 RBC Capital Markets Global Healthcare Conference on May 20, 2025, at 11:00 a.m. ET in New York [1] - The live webcast of the event will be available on the company's investor section of the website and archived for 60 days post-presentation [2] Commitment and Vision - Beam Therapeutics is a values-driven organization dedicated to its people and cutting-edge science [3] - The company envisions providing life-long cures to patients suffering from serious diseases through its diversified portfolio of base editing programs [3]

Core Insights - Beam Therapeutics has received FDA's Regenerative Medicine Advanced Therapy (RMAT) designation for BEAM-302, a treatment targeting alpha-1 antitrypsin deficiency (AATD) [1][2] - The RMAT designation highlights the potential of BEAM-302 as a transformative one-time treatment for AATD, which currently lacks effective therapies [2][3] - Initial Phase 1/2 trial results indicate BEAM-302 is well tolerated and shows durable, dose-dependent correction of the disease-causing mutation [3] Company Overview - Beam Therapeutics is focused on precision genetic medicines, utilizing base editing technology to develop therapies [8] - The company aims to provide life-long cures for serious diseases through its integrated gene editing and delivery platform [8] Product Details - BEAM-302 is a liver-targeting lipid-nanoparticle formulation designed to correct the PiZ mutation associated with severe AATD [4] - The treatment aims to reduce toxic protein aggregation in the liver and increase therapeutic levels of corrected protein in circulation [4] Disease Context - AATD is an inherited disorder leading to early onset emphysema and liver disease, primarily caused by the PiZ mutation in the SERPINA1 gene [5][6] - Approximately 100,000 individuals in the U.S. are estimated to have the PiZZ genotype, with only about 10% diagnosed [7] - Current treatments, such as intravenous AAT protein replacement, do not prevent lung function decline [7]

Core Insights - Beam Therapeutics reported a wider loss of $1.24 per share for Q1 2025, compared to the Zacks Consensus Estimate of a loss of $1.11 and a loss of $1.21 per share in the same quarter last year [1] - Total revenues for the first quarter were $7.5 million, slightly up from $7.4 million year-over-year, but below the Zacks Consensus Estimate of $15 million [2] - The company's stock fell 19.3% on May 6 due to disappointing Q1 results, and it has declined 35.6% year-to-date, while the industry has only declined by 2.8% [2][3] Financial Performance - Research and development expenses increased to $98.8 million in Q1, up nearly 16.5% from the previous year [4] - General and administrative expenses rose to $27.9 million, an increase of around 4.5% year-over-year [4] - As of March 31, 2025, Beam Therapeutics had cash and marketable securities totaling $1.20 billion, up from $850.7 million as of December 31, 2024, and expects this to fund operations into 2028 [5] Pipeline Developments - The company is advancing its ex-vivo genome-editing candidate, BEAM-101, in a phase I/II BEACON study for treating adult patients with sickle cell disease, aiming to dose 30 patients by mid-2025 [6] - Beam is also expanding its pipeline with BEAM-301 and BEAM-302 for glycogen storage disease type 1a and alpha-1 antitrypsin deficiency, respectively [8] - Initial safety and efficacy data for BEAM-302 showed promising results, with treatment leading to significant increases in total and functional alpha-1 antitrypsin levels and reductions in toxic mutant Z-AAT [10][11] - The FDA has cleared the IND application for BEAM-302, and the company is activating study sites in the U.S. [13]

Core Viewpoint - Beam Therapeutics experienced a significant decline in stock price, dropping over 19% following the release of its quarterly results, contrasting with a minor dip in the S&P 500 [1] Financial Performance - Beam reported nearly $7.5 million in revenue from licenses and collaborations for the first quarter, remaining flat year-over-year [2] - The company's net loss increased to over $109 million ($1.24 per share), compared to a loss of less than $99 million in the same quarter of the previous year [2] - Analysts had anticipated higher revenue, with an average expectation of slightly over $13.9 million, and the company also missed earnings estimates, which were projected at a loss of $1.21 per share [4] Technology and Pipeline - Beam specializes in base editing, a gene editing technique that effectively erases mutations rather than slicing them out, which is central to its pipeline programs [4] - The most advanced program in Beam's pipeline is the BEAM-101 treatment for sickle cell disease [4] Cash Position and Future Outlook - As of the end of March, Beam had $1.2 billion in cash, equivalents, and marketable securities, an increase from less than $851 million at the end of 2024 [5] - Management indicated that this cash position is sufficient to fund operations into 2028, allowing time for the development of current programs [5]

Beam Therapeutics Inc. (BEAM) came out with a quarterly loss of $1.24 per share versus the Zacks Consensus Estimate of a loss of $1.11. This compares to loss of $1.21 per share a year ago. These figures are adjusted for non-recurring items.This quarterly report represents an earnings surprise of -11.71%. A quarter ago, it was expected that this company would post a loss of $1.10 per share when it actually produced a loss of $1.09, delivering a surprise of 0.91%.Over the last four quarters, the company has s ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2025 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE TRANSITION PERIOD FROM TO Commission File Number 001-39208 Beam Therapeutics Inc. (Exact name of Registrant as specified in its Charter) Delaware 81-5238376 (State or other jurisdic ...

Exhibit 99.1 Beam Therapeutics Reports First Quarter 2025 Financial Results and Recent Business Highlights First Patient Dosed in the Phase 1/2 Study of BEAM-301 in Glycogen Storage Disease Type Ia, Beam's Second Clinical Stage In Vivo Editing Program Updated Data from BEACON Phase 1/2 Clinical Trial of BEAM-101 Accepted for Presentation at the European Hematology Association 2025 Congress in June Following Positive Initial Data for BEAM-302 in Alpha-1 Antitrypsin Deficiency, Company Initiated Dosing of Fou ...