On March 15, 2021 Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, reported recent business highlights and pipeline updates, as well as fourth quarter and full year 2020 financial results (Press release, Beam Therapeutics, MAR 15, 2021, View Source [SID1234576643]).
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"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 (ASH) (Free ASH Whitepaper) 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 (Free ASH Whitepaper) 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.