On May 11, 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 first quarter 2021 financial results (Press release, Beam Therapeutics, MAY 11, 2021, View Source [SID1234579686]).
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"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 (Free ASGCT Whitepaper) 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 (Free ASGCT Whitepaper)," 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) (Free ASGCT Whitepaper) 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 (Free ASGCT Whitepaper). 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 (Free ASGCT Whitepaper): 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 (Free ASGCT Whitepaper) 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.