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 ...

Core Insights - Beam Therapeutics has made significant progress in its clinical trials, including the first patient dosed in the Phase 1/2 study of BEAM-301 for Glycogen Storage Disease Type Ia and the initiation of the fourth cohort in the BEAM-302 trial for Alpha-1 Antitrypsin Deficiency [1][4][3] - The company reported a strong financial position with $1.2 billion in cash and equivalents, which is expected to support operations into 2028 following a $500 million financing [2][8][11] - Positive initial data from the BEAM-302 trial has been presented, demonstrating safety and efficacy, and further data is expected to be shared at upcoming medical conferences [3][4][11] Clinical Development Progress - The first patient was dosed in the BEAM-301 trial, targeting the R83C mutation in Glycogen Storage Disease Type Ia [4] - The BEACON Phase 1/2 trial of BEAM-101 for sickle cell disease is on track to complete dosing for 30 patients by mid-2025, with updated data presentation scheduled for the EHA 2025 Congress [4][11] - The BEAM-302 trial has shown promising results, with initial data indicating that the treatment is well tolerated and leads to durable, dose-dependent correction of the disease-causing mutation [4][3] Financial Overview - As of March 31, 2025, Beam Therapeutics reported cash, cash equivalents, and marketable securities totaling $1.2 billion, an increase from $850.7 million at the end of 2024 [11][14] - Research and Development expenses for Q1 2025 were $98.8 million, up from $84.8 million in Q1 2024, while General and Administrative expenses increased to $27.9 million from $26.7 million [11][16] - The net loss for Q1 2025 was $109.3 million, or $1.24 per share, compared to a net loss of $98.7 million, or $1.21 per share, in the same period of 2024 [11][16]

Core Insights - Beam Therapeutics presented new data from the Phase 1/2 clinical trial of BEAM-302, showing a mean of 91% corrected M-AAT in circulation at Day 28 in the 60 mg cohort, alongside a 79% decrease in mutant Z-AAT levels [1][3][4] Group 1: Clinical Trial Data - The Phase 1/2 trial of BEAM-302 has demonstrated positive initial safety and efficacy, establishing clinical proof of concept for treating alpha-1 antitrypsin deficiency (AATD) [2] - Updated biomarker data from the 60 mg cohort indicated that the proportion of corrected M-AAT reached a mean of 91% of total AAT in circulation, surpassing levels typically seen in patients with the MZ genotype [3] - The treatment led to a mean decrease of 79% in circulating mutant Z-AAT from baseline as of Day 28 [3] Group 2: Future Plans and Developments - Beam Therapeutics plans to continue the dose-escalation portion of the trial, initiating a fourth cohort at 75 mg and expects to present further data at a medical conference in the second half of 2025 [4] - The company aims to dose the first patient in Part B of the trial, which will include AATD patients with mild to moderate liver disease, in the second half of 2025 [4] - The investigational drug application (IND) for BEAM-302 has been cleared by the U.S. FDA, allowing the activation of trial sites in the U.S. [4] Group 3: About BEAM-302 and AATD - BEAM-302 is a liver-targeting lipid-nanoparticle formulation designed to correct the PiZ mutation, which is prevalent among patients with severe AATD [5] - The treatment aims to reduce the aggregation of mutant AAT protein, generate therapeutic levels of corrected protein, and increase total AAT in circulation, addressing the underlying pathophysiology of liver and lung disease [5] - AATD is an inherited genetic disorder that can lead to early onset emphysema and liver disease, with an estimated 100,000 individuals in the U.S. affected by the severe form of the disease [6][8]

Core Insights - Beam Therapeutics Inc. has received FDA clearance for its investigational new drug application for BEAM-302, aimed at treating alpha-1 antitrypsin deficiency (AATD) [1] - AATD is a genetic disorder leading to early emphysema and liver disease, with no approved curative treatments currently available [2] - The company has reported positive initial safety and efficacy data from the phase I/II study of BEAM-302, indicating it was well tolerated and led to dose-dependent correction of the disease-causing mutation [4][5] Company Developments - The phase I/II study of BEAM-302 consists of two parts: Part A focuses on AATD patients with lung disease, while Part B will target patients with mild-to-moderate liver disease [6] - The first patient in Part B is expected to be dosed in the second half of 2025, with additional patients to be enrolled in a fourth-dose cohort [8] - Year-to-date, shares of Beam Therapeutics have declined by 10.6%, contrasting with a 3.3% increase in the industry [3] Market Context - Beam Therapeutics currently holds a Zacks Rank of 3 (Hold), while other biotech stocks like Jazz Pharmaceuticals, Dynavax Technologies, and Krystal Biotech have better rankings [9]

Core Viewpoint - The FDA has cleared the investigational new drug (IND) application for BEAM-302, a potential treatment for alpha-1 antitrypsin deficiency (AATD), marking a significant advancement in the development of genetic medicines for this condition [2][3]. Company Overview - Beam Therapeutics is a biotechnology company focused on precision genetic medicines through base editing, with a commitment to developing a fully 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 in patients with AATD, which is expected to provide a one-time treatment option [3][5]. - The ongoing Phase 1/2 clinical trial of BEAM-302 aims to evaluate its safety, tolerability, pharmacodynamics, pharmacokinetics, and efficacy, with initial results indicating proof of concept for the treatment [3][4]. Clinical Trial Progress - The company plans to continue the dose-escalation portion of the trial and expects to report further data at a medical conference in the second half of 2025, including the initiation of dosing for patients with mild to moderate liver disease [4][3]. Disease Background - AATD is an inherited genetic disorder that can lead to early onset emphysema and liver disease, primarily affecting individuals with the PiZZ genotype, which is estimated to be present in about 100,000 individuals in the U.S. [6][7]. - Current treatments for AATD are limited, with no curative options available, highlighting the potential impact of BEAM-302 [7].