On May 10, 2023 Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, reported first quarter 2023 financial results and provided an update on its BEACON clinical trial and pipeline progress (Press release, Beam Therapeutics, MAY 10, 2023, View Source [SID1234631344]).

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"As we work through the sentinel cohort process with our investigators, we are encouraged by the overall momentum of the BEACON trial, with multiple new sites activated, a growing number of patients on the wait list for future enrollment, and continuing progress preparing for internal GMP manufacturing in our North Carolina facility," said John Evans, chief executive officer of Beam. "In parallel to BEACON, we are preparing to initiate our second clinical trial this year, evaluating BEAM-201 in patients with a devastating form of blood cancer, while advancing our in vivo programs, BEAM-301 and BEAM-302, all of which remain on track. As leaders in the field of base editing, we have spent the last several years building a deep platform and pipeline and securing meaningful funding to support this ambitious set of goals. We plan to continue to follow the science, maintain a disciplined investment strategy, and preserve optionality in our portfolio, so that we can maximize our potential to create medicines that provide life-long cures for patients in need."

Hematology Program Updates

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Beam continues to advance its BEACON Phase 1/2 clinical trial evaluating BEAM-101 as a treatment for sickle cell disease (SCD), with five sites now active and a wait list for enrollment.
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In the sentinel cohort, which is designed to include three patients treated on a sequential basis, the first patient enrolled in the BEACON trial in late 2022 has been withdrawn from the study by the investigator due to personal, non-medical reasons prior to treatment with BEAM-101.
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Two additional patients have been enrolled in the sentinel cohort and are undergoing the screening procedures required to enable mobilization and ultimately treatment with BEAM-101.
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Beam expects to fully enroll the sentinel cohort in 2023.
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Enrollment of additional patients for the expansion cohort, which can occur in parallel with the sentinel cohort, is also anticipated to begin this year.
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The company continues to anticipate reporting initial data on multiple patients from the BEACON trial in 2024.

Immunology/Oncology Program Updates

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Beam is advancing BEAM-201, a multiplex-edited allogeneic CAR-T product candidate, for the treatment of relapsed/refractory T-cell acute lymphoblastic leukemia (T-ALL)/T-cell lymphoblastic lymphoma (T-LL). The company expects to begin enrollment and dose the first patient in its Phase 1/2 clinical trial of BEAM-201 by mid-2023.
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Beam also continues to invest in and advance its next-generation allogeneic strategies designed to improve cell persistence and expand the utility and accessibility of cell therapies in cancer and other diseases. The company plans to share updates on these efforts in 2023.
Genetic Disease (in vivo) Program Updates

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Beam is advancing two in vivo base editing product candidates, BEAM-301 and BEAM-302, leveraging lipid nanoparticles (LNPs) for delivery to the liver. The company expects to:
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Submit a regulatory application for authorization to initiate clinical trials for BEAM-301 for the treatment of glycogen storage disease 1a (GSD1a) by late 2023 or early 2024; and
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Submit a regulatory application for authorization to initiate clinical trials for BEAM-302 for the treatment of alpha-1 antitrypsin deficiency (AATD) in early 2024.
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In 2023, Beam plans to continue advancement of multiple additional in vivo liver editing programs, including both its wholly owned and collaboration programs, through lead optimization, and advance its LNP delivery technologies for delivery of base editing medicines to the liver and other tissues.
Manufacturing Updates

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Beam expects to initiate current good manufacturing practice compliant operations at its North Carolina manufacturing facility in late 2023.
Leadership Addition

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In March 2023, Beam appointed Gopi Shanker, Ph.D., as chief scientific officer. Dr. Shanker is a scientific leader with more than 20 years of drug development experience as well as deep expertise in novel genetic medicine modalities.
Upcoming Presentations at ASGCT (Free ASGCT Whitepaper)

Beam plans to present new in vivo data from its Engineered Stem Cell Antibody Paired Evasion (ESCAPE) platform, which it continues to advance in an effort to enable utility of its base editing investigational medicines in as many patients as possible, and from its base editing program for the treatment of Stargardt disease at the American Society of Cell and Gene Therapy 26th Annual meeting. The meeting is being held May 16-20, 2023, in Los Angeles.

Details of the presentations are as follows:

Title: Paired HSC Epitope Engineering of CD117 (Ckit) for Antibody-Mediated Autologous Hematopoietic Stem Cell Therapy Conditioning for the Potential Treatment of Hemoglobinopathies

Date & Time: Friday, May 19, 2023 12:00 pm – 2:00 pm PT

Session Title: Friday Poster Session

Title: (308) In Vivo Genetic Eye Disease Correction Using Split AAV-Mediated Adenine Base Editing

Date & Time: Friday May 19, 2023, 4:30 pm – 4:45 pm PT

Session Title: Gene Targeting and Gene Correction: Hemoglobin, Muscle, and Eye

First Quarter 2023 Financial Results

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Cash Position: Cash, cash equivalents and marketable securities were $1.1 billion as of March 31, 2023, as compared to $1.1 billion as of December 31, 2022.
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Research & Development (R&D) Expenses: R&D expenses were $99.6 million for the first quarter of 2023, compared to $65.4 million for the first quarter of 2022.
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General & Administrative (G&A) Expenses: G&A expenses were $23.5 million for the first quarter of 2023, compared to $19.2 million for the first quarter of 2022.
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Net Loss: Net loss was $96.5 million for the first quarter of 2023, or $1.33 per share, compared to $69.2 million for the first quarter of 2022, or $1.01 per share.
Cash Runway

Beam expects that its cash, cash equivalents and marketable securities as of March 31, 2023, 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 anticipated milestones for BEAM-101, BEAM-201, BEAM-301 and BEAM-302 described above, as well as continued investments in platform advancements and manufacturing capabilities.