On February 28, 2023 Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, reported its anticipated upcoming milestones across the company’s hematology, immunology-oncology, and genetic disease portfolios and reported fourth quarter and full year 2022 financial results (Press release, Beam Therapeutics, FEB 28, 2023, View Source [SID1234627803]).

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"Just five years since our founding, we achieved significant milestones in 2022, having advanced two ex vivo assets – BEAM-101 and BEAM-201 – into clinical-stage development, and two in vivo liver programs – BEAM-301 and BEAM-302 – on a path to regulatory filings," said John Evans, chief executive officer of Beam. "As we look ahead in 2023, we are focused on the execution of our clinical trials and advancing preclinical work that we expect will support regulatory filings for BEAM-301 and BEAM-302. Importantly, as our confidence in the power and differentiation of our base editing platform grows, we also continue to make important investments in new programs across each of our strategic pillars. This includes our ESCAPE platform in hematology, our next-generation of highly engineered cell therapies in immunology-oncology, and numerous new base editing programs targeting the liver on our own and with our collaborators. I am incredibly excited about the year ahead as we execute our clinical trials and focus on advancing an entirely new class of potentially one-time transformative medicines that could benefit so many patients in need."

Key 2023-2024 Anticipated Milestones and Investments

Hematology Portfolio

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Continue enrollment in sentinel cohort of BEACON Phase 1/2 clinical trial evaluating BEAM-101 as a treatment for sickle cell disease (SCD)
oFinish enrollment in the sentinel cohort in 2023
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Initiate enrollment in the expansion cohort in 2023
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Report initial data from multiple patients from one or both cohorts in 2024
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Continue to advance and invest in Engineered Stem Cell Antibody Paired Evasion (ESCAPE) conditioning platform throughout 2023
Immunology/Oncology Portfolio

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Enroll and dose first patient in Phase 1/2 clinical trial of BEAM-201 in patients with relapsed/refractory T-cell acute lymphoblastic leukemia (T-ALL)/T-cell lymphoblastic lymphoma (T-LL) by mid-2023
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Continue to invest in and advance potential next-generation allogeneic strategies designed to significantly improve cell persistence to expand the utility and accessibility of cell therapies in cancer and other diseases, with updates on these efforts to be provided in 2023

Genetic Disease Portfolio

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Advance preclinical studies for BEAM-301 in glycogen storage disease 1a (GSDIa) and, by late 2023 or early 2024, submit a regulatory application for authorization to initiate clinical trials for the program
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Advance preclinical studies for BEAM-302 for severe alpha-1 antitrypsin deficiency (AATD) and, in early 2024, submit a regulatory application for authorization to initiate clinical trials for the program
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Advance multiple additional in vivo liver editing programs through lead optimization in 2023, including both Beam wholly owned and collaboration programs
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Continue advancement of lipid nanoparticle delivery technologies targeting the liver and other tissues throughout 2023
Manufacturing

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Initiate current good manufacturing practice compliant operations at Beam’s internal North Carolina manufacturing facility in late 2023
Recent Nature Biotechnology Publication

In January 2023, Beam published preclinical research in Nature Biotechnology highlighting work that led to the creation of an improved class of cytosine base editors (CBEs), leveraging a TadA enzyme-based CBE (CBE-T), that demonstrated edits at levels comparable to traditional CBEs and that benefited from favorable attributes of TadA for precise, specific, and flexible base editing. In addition, the paper highlights research to identify cytosine and adenine base editors (CABEs) that can conduct both C-to-T and A-to-G edits with a single TadA deaminase—called CABE-Ts. Together with ABEs, CBE-Ts and CABE-Ts demonstrated programmable installation of all transition mutations using laboratory-evolved TadA variants with improved properties relative to previously reported CBEs.

Fourth Quarter and Full Year 2022 Financial Results

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Cash Position: Cash, cash equivalents and marketable securities were $1.1 billion as of December 31, 2022, compared to $ 965.6 million as of December 31, 2021.
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Research & Development (R&D) Expenses: R&D expenses were $86.3 million for the fourth quarter of 2022 and $311.6 million for the full year ended December 31, 2022, compared to $96.8 million for the fourth quarter of 2021 and $387.1 million for the full year ended December 31, 2021.
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General & Administrative (G&A) Expenses: G&A expenses were $22.7 million for the fourth quarter of 2022 and $87.8 million for the full year ended December 31, 2022, compared to $17.8 million for the fourth quarter of 2021 and $57.2 million for the full year ended December 31, 2021.
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Net Loss: Net loss attributable to common stockholders was $38.3 million, or $0.54 per share, for the fourth quarter of 2022 and $289.1 million, or $4.13 per share, for the year ended December 31, 2022, compared to $64.7 million, or $0.95 per share, for the fourth quarter of 2021 and $370.6 million, or $5.77 per share, for the full year ended December 31, 2021.
Cash Runway

Beam expects that its cash, cash equivalents and marketable securities as of December 31, 2022, will enable the company to fund its anticipated operating expenses and capital expenditure requirements at least into 2025. This expectation includes funding directed toward reaching each of the key milestones for BEAM-101, BEAM-201, BEAM-301 and BEAM-302 described above, as well as continued investments in platform advancements and manufacturing capabilities.