On August 6, 2024 Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, reported second quarter 2024 financial results and provided updates across the company’s hematology and genetic disease franchises (Press release, Beam Therapeutics, AUG 6, 2024, View Source [SID1234645407]).

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"This quarter we’ve made significant progress across our rapidly expanding clinical portfolio, where each program utilizes the power and precision of base editing technology to provide potential best-in-class genetic medicines for patients," said John Evans, chief executive officer of Beam. "In our genetic disease franchise, we’re pleased to announce the clearance of our U.S. investigational new drug (IND) application for BEAM-301, our first U.S. in vivoregulatory filing. We’re focused on initiating site activation activities for BEAM-301 as well as continuing to enroll our BEAM-302 Phase 1/2 clinical trial in alpha-1 antitrypsin deficiency (AATD) following study initiation in June. We look forward to reporting the first data from the BEAM-302 trial next year. In addition, enrollment in the BEACON trial of BEAM-101 in sickle cell disease (SCD) has exceeded expectations, with more than 20 patients enrolled and six dosed, plus additional patients consented and in the screening process. Initial BEAM-101 clinical data have been submitted for presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting taking place in December, along with abstracts for the first clinical data for BEAM-201 as well as our first ESCAPE preclinical data in non-human primates."

Second Quarter 2024 and Recent Progress

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To date, more than 20 patients have cleared screening and enrolled in the BEACON Phase 1/2 clinical trial of BEAM-101, an investigational genetically modified cell therapy for the treatment of SCD. Of these patients, six have been dosed with BEAM-101, with the other enrolled patients going through pre-transplant stages including mobilization and manufacturing.
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In June, Beam reported data at the European Hematology Association (EHA) (Free EHA Whitepaper) Hybrid Congress highlighting its optimized, closed and automated manufacturing process for its base-edited CD34+ hematopoietic stem and progenitor cell genetic medicines, which is currently being deployed for the manufacturing of BEAM-101 in the BEACON Phase 1/2 clinical trial. The data, which include both preclinical and GMP clinical manufacturing experience to date, demonstrate that the use of base editing technology plus the advanced CD34+ manufacturing process employed by Beam are achieving reproducible and robust product yields and viability that meet high-quality standards.
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In June, Beam announced that the first patient was treated with BEAM-302, an investigational in vivo base editing medicine designed to precisely correct the underlying cause of severe AATD that is currently being evaluated in a Phase 1/2 clinical trial.
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The U.S. Food and Drug Administration has cleared the IND application for BEAM-301, an investigational in vivo base editing medicine designed to directly correct the R83C mutation, one of the primary disease-causing mutations of glycogen storage disease type Ia (GSDIa).
Key Anticipated Milestones

Hematology Franchise

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Initial data from the BEACON Phase 1/2 clinical trial have been submitted for presentation at the ASH (Free ASH Whitepaper) Annual Meeting, taking place December 7-10, 2024. Pending acceptance, Beam anticipates presenting data on all patients from the sentinel cohort as well as multiple patients from the expansion cohort.
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Beam continues to advance and invest in its Engineered Stem Cell Antibody Paired Evasion (ESCAPE) conditioning platform and anticipates initiating Phase 1-enabling preclinical studies for the program in 2024. Preclinical data for ESCAPE in non-human primates have been submitted for presentation at ASH (Free ASH Whitepaper).
Genetic Disease Franchise

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Beam continues to enroll the Phase 1/2 clinical trial of BEAM-302 in patients with AATD and expects to report initial clinical data in 2025.
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The company is now initiating site activation activities for the Phase 1/2 clinical trial for BEAM-301 in GSDIa with patient dosing expected to commence in early 2025.
Oncology

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Initial data from the Phase 1/2 clinical trial of 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), have been submitted for presentation at the ASH (Free ASH Whitepaper) Annual Meeting.
Second Quarter 2024 Financial Results

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Cash Position: Cash, cash equivalents and marketable securities, were $1.0 billion as of June 30, 2024, compared to $1.2 billion as of December 31, 2023.
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Research & Development (R&D) Expenses: R&D expenses were $87.0 million for the second quarter of 2024, compared to $97.6 million for the second quarter of 2023.
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General & Administrative (G&A) Expenses: G&A expenses were $29.6 million for the first quarter of 2024, compared to $24.7 million for the second quarter of 2023.
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Net Loss: Net loss was $91.1 million for the second quarter of 2024, or $1.11 per share, compared to $82.8 million for the second quarter of 2023, or $1.08 per share.
Cash Runway

Beam expects that its cash, cash equivalents and marketable securities as of June 30, 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, as well as continued investments in platform advancements and manufacturing capabilities, and excludes commercial spend related to the potential launch of BEAM-101.