On February 25, 2025 Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, reported fourth quarter and full year 2024 financial results and reiterated anticipated milestones across the company’s hematology and genetic disease franchises (Press release, Beam Therapeutics, FEB 25, 2025, View Source [SID1234650517]).

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"We are incredibly proud of the significant progress and momentum across our hematology and liver-targeted genetic disease franchises over the last year," said John Evans, chief executive officer of Beam. "On the heels of the initial positive results from the BEACON trial for BEAM-101 in patients with sickle cell disease, we recently achieved our adult enrollment target in the study and enrolled our first adolescent patients. Additionally, we remain on track to deliver initial data for our lead in vivo program, BEAM-302 in alpha-1 antitrypsin deficiency, in the first half of 2025, where we have the potential for a one-time treatment to address both the lung and liver manifestations of disease. With a strong financial position and important catalysts on the horizon, we are well equipped to continue driving forward our mission of providing life-long cures to patients suffering from serious diseases."

Fourth Quarter 2024 and Recent Progress

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The company has achieved its adult enrollment target for the BEACON Phase 1/2 clinical trial of BEAM-101, an investigational genetically modified cell therapy for the treatment of sickle cell disease (SCD). In addition, multiple adolescent patients have cleared screening and enrolled in the trial.
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In December, Beam presented initial results for the BEACON trial at the 66th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, which demonstrated that treatment with BEAM-101 induced robust and durable increases in fetal hemoglobin and reductions in sickle hemoglobin, rapid neutrophil and platelet engraftment, and normalized or improved markers of hemolysis. The presentation subsequently received the "Best of ASH (Free ASH Whitepaper)" distinction, and an encore of the data was presented at the 2025 Tandem Meetings of ASTCT and CIBMTR.
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Also at ASH (Free ASH Whitepaper), Beam presented proof-of-concept data in non-human primates for its Engineered Stem Cell Antibody Evasion (ESCAPE) platform demonstrating engraftment of base-edited cells using only antibody conditioning and no chemotherapy. The company initiated Phase 1-enabling preclinical toxicology studies for ESCAPE in December.
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The company continues to advance global regulatory and site activation activities for BEAM-302, an in vivo base editor being developed for the potential treatment of alpha-1 antitrypsin deficiency (AATD), with sites now open in the United Kingdom, New Zealand, Australia and Netherlands.
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In January, Beam activated the first clinical trial site for the Phase 1/2 clinical trial of BEAM-301, an in vivo base editor being developed for the potential treatment of glycogen storage disease type 1a (GSD1a).

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In December, Beam appointed Sravan Emany as chief financing officer. In addition, Chirfi Guindo, chief marketing officer of Human Health at Merck & Co., Inc., was appointed to its board of directors.
Key 2025 Anticipated Milestones

Hematology Franchise

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In the BEACON Phase 1/2 clinical trial of BEAM-101 in adults with severe SCD, Beam expects to present updated data in mid-2025.
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Beam expects to dose 30 patients in the BEACON trial by mid-2025.
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The company expects to initiate a Phase 1 healthy volunteer clinical trial of BEAM-103, the ESCAPE monoclonal antibody, by the end of 2025.
Liver-targeted Genetic Disease Franchise

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Beam expects to report initial data from multiple cohorts from the Phase 1/2 study of BEAM-302 in AATD in the first half of 2025.
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Patient dosing in the Phase 1/2 clinical trial of BEAM-301 in GSD1a is expected to commence in early 2025.
Fourth Quarter and Full Year 2024 Financial Results

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Cash Position: Cash, cash equivalents and marketable securities were $850.7 million as of December 31, 2024, compared to $1.2 billion as of December 31, 2023.
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Research & Development (R&D) Expenses: R&D expenses were $101.4 million for the fourth quarter of 2024 and $367.6 million for the full year ended December 31, 2024, compared to $140.1 million for the fourth quarter of 2023 and $437.4 million for the full year ended December 31, 2023.
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General & Administrative (G&A) Expenses: G&A expenses were $28.7 million for the fourth quarter of 2024 and $111.5 million for the full year ended December 31, 2024, compared to $43.3 million for the fourth quarter of 2023 and $116.8 million for the full year ended December 31, 2023.
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Net Income (Loss): Net loss attributable to common stockholders was $90.4 million, or $1.09 per share, for the fourth quarter of 2024 and $376.7 million, or $4.58 per share, for the year ended December 31, 2024, compared to net income attributable to common stockholders of $142.8 million, or $1.77 per basic share and $1.73 per diluted share, for the fourth quarter of 2023 and net loss attributable to common stockholders of $132.5 million, or $1.72 per share, for the year ended December 31, 2023.
Cash Runway

Beam expects that its cash, cash equivalents and marketable securities as of December 31, 2024, will enable the company to fund its anticipated operating expenses and capital expenditure requirements into 2027. This expectation includes funding directed toward reaching each of the key anticipated milestones for BEAM-101, ESCAPE, BEAM-301 and BEAM-302 described above.