BEAM Beam Therapeutics Inc.

69.15
-2.77  -4%
Previous Close 71.92
Open 70.63
52 Week Low 18.76
52 Week High 126.9
Market Cap $4,327,274,094
Shares 62,578,078
Float 39,548,523
Enterprise Value $4,133,729,369
Volume 642,447
Av. Daily Volume 964,791
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Latest News

  1. Data from Beam's Novel LNP-mRNA Formulation Demonstrates In Vivo Editing in Liver Cells of Non-human Primates Up to 52%

    Company On-track to Submit First IND for BEAM-101 in the Second Half of 2021

    CAMBRIDGE, Mass., May 11, 2021 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (NASDAQ:BEAM), a biotechnology company developing precision genetic medicines through base editing, today reported recent business highlights and pipeline updates, as well as first quarter 2021 financial results.

    "As Beam prepares to enter the clinic, we also continue to extend our leadership position in the field of base editing by expanding our platform and delivery capabilities," said John Evans, chief executive officer of Beam. "The data presented at ASGCT on our proprietary…

    Data from Beam's Novel LNP-mRNA Formulation Demonstrates In Vivo Editing in Liver Cells of Non-human Primates Up to 52%

    Company On-track to Submit First IND for BEAM-101 in the Second Half of 2021

    CAMBRIDGE, Mass., May 11, 2021 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (NASDAQ:BEAM), a biotechnology company developing precision genetic medicines through base editing, today reported recent business highlights and pipeline updates, as well as first quarter 2021 financial results.

    "As Beam prepares to enter the clinic, we also continue to extend our leadership position in the field of base editing by expanding our platform and delivery capabilities," said John Evans, chief executive officer of Beam. "The data presented at ASGCT on our proprietary lipid nanoparticle (LNP) formulation show efficient in vivo base editing in hepatocytes of non-human primates (NHPs), while our novel analytical assays confirm specificity and efficiency of base editing to directly correct disease-causing mutations. As we look to the rest of 2021, we are on-track with our plans to submit our first Investigational New Drug (IND) application for BEAM-101 and initiate IND-enabling studies for BEAM-102 and BEAM-201, as well as nominate our first development candidate from our liver portfolio. We are well positioned today to advance our base editing platform and pipeline and remain focused on achieving our vision of providing life-long cures for patients suffering from serious diseases."

    "We are pleased to share our development of a proprietary LNP formulation for non-viral in vivo delivery to the liver at ASGCT," said Giuseppe Ciaramella, Ph.D., president and chief scientific officer of Beam. "These data demonstrate that our LNPs are well tolerated in NHPs and can achieve levels of editing of hepatocytes that we expect would be therapeutic for many genetic diseases. Initial data also demonstrated the stability of our LNPs at -20 degrees Celsius after 8 weeks, which is important for enabling broad clinical and commercial development. We are continuing to work on optimizing our LNP formulations and are on track to nominate our first development candidate from our liver portfolio in the second half of 2021."

    Base Editing Progress

    • Data Demonstrating Optimization of LNP-mRNA Formulation for Liver Editing Presented at the 24th American Society of Gene and Cell Therapy (ASGCT) Annual Meeting: Beam's approach to developing a novel lipid nanoparticle (LNP) formulation for in vivo liver editing will be presented during a poster session titled, "Using Base Editing and LNP Delivery to Correct Disease-Causing Mutations Underlying Genetic Liver Diseases" at ASGCT. Using an mRNA-encoding adenine base editor (ABE) and guide RNA to target the ALAS1 gene, a surrogate payload for genetic liver diseases, Beam evaluated various LNP formulations and mRNA production processes to improve in vivo editing in the livers of NHPs from less than 10% initially to 52% at a total RNA dose of 1.5 mg/kg. These formulations were also well tolerated by NHPs at 1.5 mg/kg with mild and transient liver enzyme elevations, and showed promising interim stability, maintaining potency after 8 weeks at -20⁰C.
    • Data Highlighting Beam's Approach to Confirming Precision Correction Using Novel Analytical Assays Presented at ASGCT: Base editing is emerging as a powerful next-generation editing technology; however, developing adequate analytical assays to confirm precise base editing at the protein level is critical and has proven historically challenging. In an oral presentation at ASGCT titled, "LC-MS Confirmation of Single Amino Acid Correction by Base Editing," Beam will describe its approach to using liquid chromatography mass spectrometry (LC-MS) for multiple analytical assays to confirm the precise correction of disease-causing mutations at the amino acid level, providing a unique solution for confirmation and quantitation of single amino acid corrections after base editing.
    • Data Highlighting Ability to Rationally Design Base Editors for Precise Editing Published in The CRISPR Journal: In April 2021, work describing Beam's approach to developing inlaid base editors (IBEs) was published in The CRISPR Journal. IBEs are architectural variants of base editors that demonstrate enhanced specificity and altered activity windows relative to foundational base editors. The work highlights Beam's application of its IBEs for BEAM-102, one of its base editing programs in development for the treatment of sickle cell disease.

    First Quarter 2021 Financial Results

    • Cash Position: Cash, cash equivalents and marketable securities were $503.5 million as of March 31, 2021, compared to $253.4 million as of March 31, 2020.
    • Research & Development (R&D) Expenses: R&D expenses were $190.1 million for the first quarter of 2021, compared to $21.5 million for the first quarter of 2020. R&D expenses for the first quarter of 2021 includes $155.0 million of expense related to in-process research and development acquired from of Guide Therapeutics, Inc.
    • General & Administrative (G&A) Expenses: G&A expenses were $10.3 million for the first quarter of 2021, compared to $6.8 million for the first quarter of 2020.
    • Net Loss: Net loss attributable to common stockholders was $201.6 million, or $3.35 per share, for the first quarter of 2021, compared to $31.7 million, or $1.03 per share, for the first quarter of 2020.

    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 that includes a suite of gene editing and delivery technologies and is in the process of building internal manufacturing capabilities. Beam's suite of gene editing technologies is anchored by base editing, a proprietary technology that enables precise, predictable and efficient single base changes, at targeted genomic sequences, without making double-stranded breaks in the DNA. This enables 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.

    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: our plans for scientific publications; our plans to enter the clinic; our expected timing for filing an investigational new drug application for BEAM-101, for initiating IND-enabling studies for BEAM-102 and BEAM-201, and for nominating our first development candidate from our liver portfolio; and the therapeutic applications and potential of our technology, including our ability to develop life-long, curative, precision genetic medicines for patients through base editing. Each forward-looking statement is subject to 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 potential impact of the COVID-19 pandemic; that preclinical testing of our product candidates and preliminary or interim data from preclinical and clinical trials may not be predictive of the results or success of ongoing or later clinical trials; that enrollment of our clinical trials may take longer than expected; 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 heading "Risk Factors" in our Annual Report on Form 10-K for the year ended December 31, 2020, our Quarterly Report on Form 10-Q for the quarter ended March 31, 2021, and in any subsequent filings with the Securities and Exchange Commission. These forward-looking statements (except as otherwise noted) 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:

    Chelcie Lister

    THRUST Strategic Communications

    Media:

    Dan Budwick

    1AB

    Condensed Consolidated Balance Sheet Data
    (in thousands)
         
      March 31,

    2021
     December 31,

    2020
    Cash, cash equivalents, and marketable securities $503,479  $299,671 
    Total assets  693,241   451,677 
    Total liabilities  270,812   206,116 
    Total stockholders' equity  422,429   245,561 
         
    Condensed Consolidated Statement of Operations
    (in thousands, except share and per share data)
         
      Three Months Ended March 31,
       2021   2020 
    License revenue $6  $6 
    Operating expenses:    
    Research and development  190,106   21,549 
    General and administrative  10,273   6,812 
    Total operating expenses  200,379   28,361 
    Loss from operations  200,373   (28,355)
    Other income (expense):    
    Change in fair value of derivative liabilities  (1,900)  (2,700)
    Change in fair value of contingent consideration liabilities  (305)   
    Interest and other income, net  1,018   597 
    Total other income (expense)  (1,187)  (2,103)
    Net loss $(201,560) $(30,458)
    Accretion of redeemable convertible preferred stock to redemption value, including dividends on preferred stock     (1,277)
    Net loss attributable to common stockholders $(201,560) $(31,735)
    Net loss per common share attributable to common stockholders, basic and diluted $(3.35) $(1.03)
    Weighted-average common shares used in net loss per share attributable to common stockholders, basic and diluted  60,210,120   30,725,077 
             


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  2. CAMBRIDGE, Mass., April 27, 2021 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (NASDAQ:BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that the company will present data highlighting its proprietary base editing approach in several presentations during the 24th American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, which will be hosted virtually May 11-14, 2021. In addition, Beam has been selected as a workshop presenter in the pre-meeting program taking place on May 10, 2021.

    "Since our founding in 2017, we have been diligently focused on advancing our proprietary base editing technology to develop treatments for patients suffering from severe diseases, and we are pleased…

    CAMBRIDGE, Mass., April 27, 2021 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (NASDAQ:BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that the company will present data highlighting its proprietary base editing approach in several presentations during the 24th American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, which will be hosted virtually May 11-14, 2021. In addition, Beam has been selected as a workshop presenter in the pre-meeting program taking place on May 10, 2021.

    "Since our founding in 2017, we have been diligently focused on advancing our proprietary base editing technology to develop treatments for patients suffering from severe diseases, and we are pleased to present updates showcasing the potential of our technology during ASGCT," said Giuseppe Ciaramella, Ph.D., president and chief scientific officer of Beam. "Taken together, these data highlight the depth of our understanding of base editing, including our work in delivery optimization to ensure efficient editing in the target organ, as well as analytical technology to confirm specificity and efficacy of our base editors, both of which are critical as we advance toward clinical development. We look forward to presenting these and additional data from our LNP program at ASGCT, as we work toward the nomination of our first development candidate from our liver portfolio and the planned IND application for our lead base editor program, BEAM-101, later this year."

    Pre-Meeting Program: Moving Genome Editing to the Clinic: From Technology to Therapeutics

    Session: Gene Editing Workshop

    Date and Time: Monday, May 10, 2021, 10:00 a.m. – 2:00 p.m. ET

    Poster Presentation: Using Base Editing and LNP Delivery to Correct Disease-Causing Mutations Underlying Genetic Liver Diseases

    Session: Synthetic/Molecular Conjugates and Physical Methods for Delivery

    Date and Time: May 11-14, 2021

    Abstract summary: Base editing enables programmable single-base genomic mutations and has the potential to permanently cure serious genetic diseases. Realizing its therapeutic potential requires three key elements, including the development of new base editors with appropriate specificity, editing window, and efficacy; sgRNA sequence and chemistry for optimal potency and target specificity; and safe and effective methods for mRNA and sgRNA delivery to target organs and intracellular compartments. For this study, Beam evaluated editing levels in the liver of non-human primates using a lipid nanoparticle (LNP) delivery system containing a novel adenine base editor (ABE) and reporter sgRNA. These data demonstrate that Beam's base editors in combination with LNP delivery have the potential to treat genetic liver diseases.

    Oral Presentation: LC-MS Confirmation of Single Amino Acid Correction by Base Editing

    Session: Pharmacology/Toxicology Studies or Assay Development

    Date and Time: Friday, May 14, 2021, 12:45 p.m. – 1:00 p.m. ET

    Abstract summary: Base editing is emerging as a powerful next generation editing technology; however, developing adequate analytical assays to confirm precise base editing at the protein level is critical and has proven historically challenging. In this study, Beam utilized liquid chromatography mass spectrometry (LC-MS) for multiple analytical assays to confirm the precise correction at the amino acid level by base editing, which proved to provide a unique solution for confirmation and quantitation of single amino acid corrections after base editing.

    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 that includes a suite of gene editing and delivery technologies and is in the process of building internal manufacturing capabilities. Beam's suite of gene editing technologies is anchored by base editing, a proprietary technology that enables precise, predictable and efficient single base changes, at targeted genomic sequences, without making double-stranded breaks in the DNA. This enables 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.

    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: our plans for scientific publications; our expected timing for filing an investigational new drug application for BEAM-101; our efforts toward the nomination of our first development candidate from our liver portfolio; and the therapeutic applications and potential of our technology, including our ability to develop life-long, curative, precision genetic medicines for patients through base editing. Each forward-looking statement is subject to 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 potential impact of the COVID-19 pandemic; that preclinical testing of our product candidates and preliminary or interim data from preclinical and clinical trials may not be predictive of the results or success of ongoing or later clinical trials; that enrollment of our clinical trials may take longer than expected; 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 heading "Risk Factors" in our Annual Report on Form 10-K for the year ended December 31, 2020, and in any subsequent filings with the Securities and Exchange Commission. These forward-looking statements (except as otherwise noted) 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:

    Chelcie Lister

    THRUST Strategic Communications

    Media:

    Dan Budwick

    1AB



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  3. Data Published in The CRISPR Journal Describe Inlaid Base Editors (IBEs) that Enhance Editing Efficiency and Capability, an Advancement of the Company's Pioneering Work in Developing Base Editors

    Data Demonstrates Utility of IBEs in BEAM-102, Enabling Conversion of Sickle Cell Hemoglobin Allele to the Naturally Occurring, Benign HbG-Makassar Variant in Patient-derived Hematopoietic Stem Cells

    CAMBRIDGE, Mass., April 20, 2021 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (NASDAQ:BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced the publication of work describing inlaid base editors (IBEs) in The CRISPR Journal. IBEs are architectural variants of base editors that have attributes of enhanced…

    Data Published in The CRISPR Journal Describe Inlaid Base Editors (IBEs) that Enhance Editing Efficiency and Capability, an Advancement of the Company's Pioneering Work in Developing Base Editors

    Data Demonstrates Utility of IBEs in BEAM-102, Enabling Conversion of Sickle Cell Hemoglobin Allele to the Naturally Occurring, Benign HbG-Makassar Variant in Patient-derived Hematopoietic Stem Cells

    CAMBRIDGE, Mass., April 20, 2021 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (NASDAQ:BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced the publication of work describing inlaid base editors (IBEs) in The CRISPR Journal. IBEs are architectural variants of base editors that have attributes of enhanced specificity and altered activity windows relative to foundational base editors. The work highlights Beam's application of its IBEs for BEAM-102, one of its base editing programs in development for the treatment of sickle cell disease (SCD).

    IBEs are an important innovation enabled by Beam's extensive know-how and understanding of structural biology and protein-guided DNA-targeting, which adds to the company's ever-expanding genome editing toolbox. Notably, the predictable, shifted editing window enabled by IBEs aids in further addressing disease-causing mutations normally out of reach with canonical base editors, with high efficiency and minimized off-target effects on the genome.

    "Beam is a pioneer in the field of base editing, and this work is a testament to the leadership of our team of scientists in expanding the role that base editing medicines can play in the treatment of a wide variety of diseases," said Giuseppe Ciaramella, Ph.D., president and chief scientific officer of Beam. "Direct correction of the sickle-causing mutation with traditional gene editing technologies has been limited by low efficiency. The therapeutic potential of our rationally designed IBEs is exciting. Not only have these differentiated base editors demonstrated the potential to efficiently convert the disease-causing sickle hemoglobin allele into a normal variant; they open the doors to potentially target a broad range of other genetic diseases."

    Beam's base editors use a DNA-targeted deaminase to create a precise chemical modification of a target DNA base with high precision and limited off-target consequences. IBEs expand the breadth of potential base editing targets by extending the range of editing windows that can be created for any given CRISPR-Cas protein used to target the DNA. By inserting the deaminase into the CRISPR protein at various strategic positions, repositioning the deaminase's active site, IBEs enable editing outside the traditional editing window. The broad modularity of these proprietary base editor designs was demonstrated using both the adenine base editor (ABE) and the cytosine base editor (CBE).

    Potential Utility of Beam's IBEs in BEAM-102

    BEAM-102 aims to treat SCD by directly editing the causative sickle hemoglobin (HbS) point mutation to recreate a naturally occurring normal human hemoglobin variant, HbG-Makassar. The Makassar variant has been reported to have the same function as the wild-type (HbA) allele and does not cause SCD. Findings in the paper show that Beam's IBE resulted in highly efficient editing levels of over 70% in CD34+ cells from both sickle trait (HbAS) and sickle cell disease (HbSS) individuals. Additionally, the IBE demonstrated significantly reduced guide-independent off-target editing in vitro.

    "Beam was launched in 2017 by world-leading innovators in gene editing who discovered that base editing is capable of making single-base changes with high efficiency and precise control of editing outcomes," said John Evans, chief executive officer of Beam Therapeutics. "With the development of our IBEs, we have once again set a precedent for the expansion and advancement of base editing as a new generation of gene editing technology. In addition, the patent applications on this work complement and extend Beam's comprehensive patent portfolio in the field of base editing. We are dedicated to fully realizing the potential of this technology for patients as we advance our portfolio, with plans to submit what we expect will be the first IND application for a base editor program in the second half of 2021."

    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 that includes a suite of gene editing and delivery technologies and is in the process of building internal manufacturing capabilities. Beam's suite of gene editing technologies is anchored by base editing, a proprietary technology that enables precise, predictable and efficient single base changes, at targeted genomic sequences, without making double-stranded breaks in the DNA. This enables 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.

    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 expected timing of filing our first investigational new drug application, our ability to advance programs to the clinic; and the therapeutic applications and potential of our technology, including our IBEs and our ability to develop life-long, curative, precision genetic medicines for patients through base editing. Each forward-looking statement is subject to 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 potential impact of the COVID-19 pandemic; that preclinical testing of our product candidates and preliminary or interim data from preclinical and clinical trials may not be predictive of the results or success of ongoing or later clinical trials; that enrollment of our clinical trials may take longer than expected; 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 heading "Risk Factors" in our Annual Report on Form 10-K for the year ended December 31, 2020, and in any subsequent filings with the Securities and Exchange Commission. These forward-looking statements (except as otherwise noted) 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:

    Chelcie Lister

    THRUST Strategic Communications

    Media:

    Dan Budwick

    1AB

     



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  4. Company On-track to Submit First IND with BEAM-101 in the Second Half of 2021

    Acquisition of Guide Therapeutics Supports Targeting of Diverse Tissues for In Vivo Delivery of Gene Editing

    Team Bolstered by Appointment of Amy Simon, M.D., as Chief Medical Officer and Kate Walsh to Board of Directors

    Balance Sheet Strengthened by $260 Million Common Stock Investment

    CAMBRIDGE, Mass., March 15, 2021 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (NASDAQ:BEAM), a biotechnology company developing precision genetic medicines through base editing, today reported recent business highlights and pipeline updates, as well as fourth quarter and full year 2020 financial results.

    "Beam has made significant progress in the last 12 months," said John Evans…

    Company On-track to Submit First IND with BEAM-101 in the Second Half of 2021

    Acquisition of Guide Therapeutics Supports Targeting of Diverse Tissues for In Vivo Delivery of Gene Editing

    Team Bolstered by Appointment of Amy Simon, M.D., as Chief Medical Officer and Kate Walsh to Board of Directors

    Balance Sheet Strengthened by $260 Million Common Stock Investment

    CAMBRIDGE, Mass., March 15, 2021 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (NASDAQ:BEAM), a biotechnology company developing precision genetic medicines through base editing, today reported recent business highlights and pipeline updates, as well as fourth quarter and full year 2020 financial results.

    "Beam has made significant progress in the last 12 months," said John Evans, chief executive officer of Beam. "Since our IPO in February 2020, we named three development candidates from our portfolio; initiated IND-enabling studies for BEAM-101; presented data highlighting our broad set of gene editing technologies; expanded access to novel delivery modalities through the acquisition of Guide Therapeutics; secured and initiated work on our build-to-suit manufacturing facility; enhanced our leadership team with the addition of Amy Simon as chief medical officer; expanded our board with the appointment of Kate Walsh; and further strengthened our capital position and operating runway. In the remainder of 2021, we plan to submit our first Investigational New Drug application for BEAM-101, initiate IND-enabling studies for BEAM-102 and BEAM-201, and nominate our first development candidate from our liver portfolio, positioning Beam to become a clinical stage company with an expansive pipeline moving quickly behind our lead assets. We are well positioned today to advance our platform and pipeline, and remain focused on achieving our vision of providing life-long cures for patients suffering from serious diseases."

    Recent Business Highlights

    • Acquisition of Guide Therapeutics (GuideTx) Expands Novel Base Editing Capabilities: In February 2021, Beam acquired GuideTx, a developer of nonviral drug delivery vehicles for genetic medicines, further expanding the potential reach of Beam's genetic medicines into target tissues beyond the liver. GuideTx's technology enables the screening of hundreds of nanoparticles in a single experiment, potentially generating in vivo drug delivery data at significantly greater rates compared to traditional screens. Beam believes that the proprietary screening method will enable identification of lipid nanoparticles with novel biodistribution and high selectivity for target cells. Using this platform and a proprietary library of cationic lipids, GuideTx has identified a broad array of novel lipid nanoparticle formulations.
    • Balance Sheet Enhanced with $260 Million Private Placement: In January 2021, Beam sold 2,795,700 shares of its common stock to certain institutional investors in a private placement. Aggregate gross proceeds from the offering were approximately $260 million, before deducting fees to the placement agents and other offering expenses payable by the Company.
    • Leadership Team Strengthened by the Addition of Amy Simon, M.D., as Chief Medical Officer: In March 2021, Beam appointed Amy Simon, M.D., as chief medical officer. Dr. Simon joins Beam from Alnylam Pharmaceuticals, where she spent over a decade in various roles with increasing responsibility for the clinical development of numerous RNAi-based medicines, most recently serving as vice president, clinical development and the lead clinician developing GIVLAARI® (givosiran) for acute hepatic porphyria, which was approved by FDA in 2019. Dr. Simon holds a B.A. in history and science from Harvard University and received her M.D. from Tufts University School of Medicine.
    • Leading Healthcare Executive, Kate Walsh, Appointed to Board of Directors: In January 2021, Kate Walsh, president and chief executive officer of the Boston Medical Center (BMC) health system, joined Beam's board of directors. BMC is a private, not-for-profit, academic medical center with a community-based focus and is the primary teaching affiliate of Boston University School of Medicine.

    Base Editing Progress

    • Updated Data from Novel Sickle Cell Disease Approaches Presented at 62nd American Society of Hematology Annual Meeting and Exposition (ASH 2020): In December 2020, Beam presented updated data from the company's complementary base editing approaches to treat hemoglobinopathies, BEAM-101 and BEAM-102, during poster sessions at ASH 2020. The BEAM-101 data presented demonstrated precise base editing with no off-target editing observed, supporting the company's planned IND submission in the second half of 2021. Additionally, in vivo data from the company's Makassar base editing program demonstrated long-term engraftment and editing retention at 16 weeks, with BEAM-102 achieving greater than 90% bi- and mono-allelic Makassar editing in SCD CD34+ HSPCs in vitro.



    • First Data Highlighting Base Editing Program for Glycogen Storage Disease Type Ia Presented at American Association for the Study of Liver Diseases' (AASLD) The Liver Meeting Digital Experience™: In November 2020, Beam presented the first data highlighting its novel base-editing strategy for correcting disease-causing mutations underlying Glycogen Storage Disease Type Ia (GSDIa) during a poster session at AASLD, demonstrating in vivo correction of the R83C and Q347X mutations by ABEs in the livers of two strains of transgenic mice, each carrying one of the two G6PC mutations. Next-generation sequencing data from whole liver extracts revealed significant correction for both R83C and Q347X, with nearly 40% and approximately 70% A-to-G conversion efficiency, respectively, of each mutation back to the normal gene sequence. These significant levels of mutation correction greatly surpassed those expected to restore glucose homeostasis, and functional studies are ongoing to correlate pathophysiology to extent of mutation correction by base-editing. Further, these levels of in vivo correction for GSDIa by base-editing were achieved without detectable creation of double-stranded breaks. In total, these data support base-editing technology as a promising approach for precise correction of causative mutations in GSDIa.

    Fourth Quarter and Full Year 2020 Financial Results

    • Cash Position: Cash, cash equivalents and marketable securities were $299.7 million as of December 31, 2020, compared to $91.8 million as of December 31, 2019. This cash balance does not include the proceeds from the January 2021 private placement.
    • Research & Development (R&D) Expenses: R&D expenses were $32.5 million for the fourth quarter of 2020 and $103.2 million for the full year ended December 31, 2020, compared to $20.2 million for the fourth quarter of 2019 and $54.6 million for the full year ended December 31, 2019.
    • General & Administrative (G&A) Expenses: G&A expenses were $8.4 million for the fourth quarter of 2020 and $29.6 million for the full year ended December 31, 2020, compared to $6.2 million for the fourth quarter of 2019 and $20.6 million for the full year ended December 31, 2019.
    • Net Loss: Net loss attributable to common stockholders was $95.5 million, or $1.69 per share, for the fourth quarter of 2020 and $195.9 million, or $4.19 per share, for the full year ended December 31, 2020, compared to $31.1 million, or $4.35 per share, for the fourth quarter of 2019 and $91.0 million, or $14.05 per share, for the full year ended December 31, 2019.

    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 that includes a suite of gene editing and delivery technologies and is in the process of building internal manufacturing capabilities. Beam's suite of gene editing technologies is anchored by base editing, a proprietary technology that enables precise, predictable and efficient single base changes, at targeted genomic sequences, without making double-stranded breaks in the DNA. This enables 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.

    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 expected timing of filing our first investigational new drug application, nominating our first development candidate from our liver portfolio, and initiating IND-enabling studies for BEAM-102 and BEAM-201; our ability to advance programs to the clinic; our ability to expand the reach of gene editing, including as a result of our acquisition of Guide Therapeutics; the strategic and other potential benefits of the Guide Therapeutics acquisition; the sufficiency of our cash position; and the therapeutic applications and potential of our technology, including our ability to develop life-long, curative, precision genetic medicines for patients through base editing. Each forward-looking statement is subject to 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 potential impact of the COVID-19 pandemic; that preclinical testing of our product candidates and preliminary or interim data from preclinical and clinical trials may not be predictive of the results or success of ongoing or later clinical trials; that enrollment of our clinical trials may take longer than expected; our ability to successfully integrate Guide Therapeutics' operations and employees and to realize the anticipated benefits of the acquisition; 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 heading "Risk Factors" in our Annual Report on Form 10-K for the year ended December 31, 2020, and in any subsequent filings with the Securities and Exchange Commission. These forward-looking statements (except as otherwise noted) 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:

    Chelcie Lister

    THRUST Strategic Communications

    Media:

    Dan Budwick

    1AB

    Condensed Consolidated Balance Sheet Data 
    (in thousands) 
          
     December 31, 
     2020  2019 
          
    Cash, cash equivalents and marketable securities$299,671  $91,848 
    Total assets451,677  156,099 
    Redeemable convertible preferred stock-  302,049 
    Total stockholders' equity (deficit)245,561  (201,104)



    Condensed Consolidated Statements of Operations
    (in thousands, except share and per share data)
                
     Three months ended December 31,  Year ended December 31, 
     2020  2019  2020  2019 
                
    License revenue$6  $6  $24  $18 
    Operating expenses:           
    Research and development32,451  20,217  103,179  54,619 
    General and administrative8,354  6,160  29,605  20,553 
    Total operating expenses40,805  26,377  132,784  75,172 
    Loss from operations(40,799) (26,371) (132,760) (75,154)
    Other income (expense):           
    Change in fair value of derivative liabilities(54,700) (1,800) (63,400) (5,400) 
    Interest and other income (expense), net35  321  1,568  2,228 
    Total other income (expense)(54,665) (1,479) (61,832)) (3,172)
    Net loss$(95,464) $(27,850) $(194,592) $(78,326)
    Accretion of redeemable convertible preferred stock to redemption value, including dividends on preferred stock-  (3,263) (1,277) (12,714)
    Net loss attributable to common stockholders$(95,464) $(31,113) $(195,869) $(91,040)
    Net loss per common share attributable to common stockholders, basic and diluted$(1.69) $(4.35) $(4.19) $(14.05)
    Weighted-average common shares used in net loss per share attributable to common stockholders, basic and diluted56,544,620  7,149,568  46,733,221  6,479,591 



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  5. CAMBRIDGE, Mass., March 15, 2021 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (NASDAQ:BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that drug development expert Amy Simon, M.D., has been appointed as the company's chief medical officer. Dr. Simon brings more than 20 years of clinical experience to Beam, serving in roles as a physician-scientist in academia and the biotechnology industry.

    "Amy is a dedicated physician and experienced drug developer who has worked across a wide range of disease areas, delivering innovative genetic medicines into the clinic and through approval," said John Evans, chief executive officer of Beam. "We have made significant progress advancing our lead…

    CAMBRIDGE, Mass., March 15, 2021 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (NASDAQ:BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that drug development expert Amy Simon, M.D., has been appointed as the company's chief medical officer. Dr. Simon brings more than 20 years of clinical experience to Beam, serving in roles as a physician-scientist in academia and the biotechnology industry.

    "Amy is a dedicated physician and experienced drug developer who has worked across a wide range of disease areas, delivering innovative genetic medicines into the clinic and through approval," said John Evans, chief executive officer of Beam. "We have made significant progress advancing our lead base editing programs through research, preclinical and now IND-enabling studies, with our first IND application for our lead candidate, BEAM-101 slated for the second half of this year. Amy's translational and clinical development expertise will be invaluable as we prepare for our next phase as a clinical-stage company. We are thrilled to welcome her to the Beam team."

    "Beam is built on an inspiring vision of bringing life-changing medicines to patients, and I couldn't be more excited to join this talented team," said Dr. Simon. "We have a unique opportunity to advance a broad portfolio of potentially one-time, disease-altering programs using base editing technology and a precision medicine approach supported by clear human genetics. I believe 2021 will be a transformative year for Beam as we leverage our strong research organization and further build out our clinical development organization. I look forward to partnering with the entire team to help bring new therapeutic options to patients suffering from serious diseases."

    Dr. Simon joins Beam from Alnylam Pharmaceuticals, where she spent over a decade in various roles with increasing responsibility for the clinical development of RNAi-based medicines, most recently serving as vice president, clinical development. During her tenure at Alnylam, she led the successful execution of clinical programs from natural history studies to Phase 1 through Phase 4 studies, regulatory interactions with both U.S. and ex-U.S. authorities, and drug approvals in the U.S. and EU. Dr. Simon was the lead clinician developing GIVLAARI® (givosiran) for patients with acute hepatic porphyria, which was approved by the Food and Drug Administration in 2019. Prior to entering the biotech industry, Dr. Simon worked in academia at Tufts University, serving as a professor and a director of the Asthma Center in the Pulmonary and Critical Care Division at Tufts University School of Medicine and as a professor at Tufts Graduate School of Biomedical Science where her laboratory conducted basic science research on asthma. She began her career in clinical practice, training as a resident in internal medicine and as a fellow in pulmonary and critical care medicine at Tufts Medical Center. Dr. Simon holds a B.A. in history and science from Harvard University, and an M.D. from Tufts University School of Medicine.

    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 that includes a suite of gene editing and delivery technologies and is in the process of building internal manufacturing capabilities. Beam's suite of gene editing technologies is anchored by base editing, a proprietary technology that enables precise, predictable and efficient single base changes, at targeted genomic sequences, without making double-stranded breaks in the DNA. This enables 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.

    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 expected timing of filing our first investigational new drug application, our ability to advance programs to the clinic; and the therapeutic applications and potential of our technology, including our ability to develop life-long, curative, precision genetic medicines for patients through base editing. Each forward-looking statement is subject to 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 potential impact of the COVID-19 pandemic; that preclinical testing of our product candidates and preliminary or interim data from preclinical and clinical trials may not be predictive of the results or success of ongoing or later clinical trials; that enrollment of our clinical trials may take longer than expected; 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 heading "Risk Factors" in our Annual Report on Form 10-K for the year ended December 31, 2020, and in any subsequent filings with the Securities and Exchange Commission. These forward-looking statements (except as otherwise noted) 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:

    Chelcie Lister

    THRUST Strategic Communications

     

    Media:

    Dan Budwick

    1AB

     



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