On November 8, 2023 Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, reported third quarter 2023 financial results and provided an update on the business and its clinical and pipeline progress (Press release, Beam Therapeutics, NOV 8, 2023, View Source [SID1234637247]).
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"This year, Beam has established an exceptional foundation for future growth, with significant financial strength, a broad portfolio of differentiated base editing programs in large addressable markets driving toward clinical milestones, and a fully operational suite of research, development, and manufacturing capabilities for precision genetic medicines," said John Evans, chief executive officer of Beam. "We have made critical decisions on where to focus and how to prioritize our investments over the coming years, creating significant momentum in our business and portfolio. In the BEACON trial, patient enrollment, mobilization, and manufacturing are progressing well, and we remain on track to treat the first trial patient with BEAM-101 this year and report clinical trial data on multiple patients next year. We’re also advancing our first in vivo program, BEAM-302, for patients with alpha-1 anti-trypsin deficiency with a regulatory filing expected in the first quarter of next year, followed by our planned IND submission for BEAM-301 shortly thereafter. External interest in our base editing technology continues to grow, illustrated most recently by our transaction with Lilly. With a strong balance sheet, a focused team, and leading capabilities, we are well positioned to advance our base editing programs and platform, which we believe have transformative potential for patients."
Pipeline Updates and Key 2023-2024 Anticipated Milestones
Beam recently announced portfolio priorities and plans to streamline its business operations to support potential near-term value drivers and long-term growth and, as such, is now focusing its efforts as follows:
Hematology Portfolio
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Beam continues to advance its BEACON Phase 1/2 clinical trial, an open-label, single-arm, multicenter study evaluating the safety and efficacy of BEAM-101 in adult patients with severe sickle cell disease (SCD). Treatment with BEAM-101, in which the edited cell product is delivered in an autologous bone marrow transplant, will occur on a sequential basis for the first three patients treated in the trial, and then will be given in parallel for all subsequent patients.
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Beam has continued to consent additional patients in the BEACON trial, all of whom are now moving in parallel through the screening, mobilization, and manufacturing activities required to enable treatment with BEAM-101. Beam anticipates that currently consented patients are sufficient to both fill the sentinel cohort (n=3) and to initiate the expansion cohort.
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The first patient in the trial is expected to be treated with BEAM-101 by year-end 2023.
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Beam will continue adding additional patients to the BEACON trial through the end of year and beyond, with a total target of 45 treated patients.
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The company anticipates reporting initial data on multiple patients from the BEACON trial in 2024.
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Beam continues to advance and invest in its Engineered Stem Cell Antibody Paired Evasion (ESCAPE) conditioning platform. ESCAPE aims to avoid toxicity challenges associated with currently available conditioning regimens for patients with SCD and beta-thalassemia ahead of autologous transplant.
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In addition, the company is exploring the potential for in vivo base editing programs for SCD, in which base editors would be delivered to the patient through an infusion of lipid nanoparticles (LNPs) targeted to hematopoietic stem cells (HSCs), eliminating the need for transplantation altogether.
Genetic Disease (in vivo) Portfolio
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Beam continues to advance its two lead in vivo base editing product candidates, BEAM-302 for the treatment of alpha-1 antitrypsin deficiency (AATD) and BEAM-301 for the treatment of glycogen storage disease Ia (GSDIa). BEAM-302 and BEAM-301 have the potential to be the first clinical programs to directly correct a genetic mutation back to a normal functional gene sequence.
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BEAM-302 is a liver-targeting LNP formulation of base editing reagents designed to correct the PiZ allele, the most common gene variant associated with severe AATD. The company expects to submit a regulatory application in the first quarter of 2024 for authorization to initiate clinical trials initially outside of the U.S., with an Investigational New Drug (IND) expected to be filed with the U.S. Food and Drug Administration (FDA) subsequently during early development.
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In September 2023, Beam reported the first preclinical data demonstrating the ability of BEAM-302 to significantly increase levels of corrected and functional alpha-1 antitrypsin (AAT) and reduce mutant PiZ AAT in multiple in vivo rodent disease models at clinically relevant doses in an oral presentation at the Alpha-1 Antitrypsin Deficiency 2023 Meeting in Naples, Italy. These findings support the potential of BEAM-302 to efficiently correct the disease-causal PiZ mutation after a single dose and potentially address both the liver and lung disease associated with AATD.
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An encore presentation at the ESGCT/SFTCG/NVGCT Collaborative Congress in October 2023 showed additional preclinical data supporting the durable effects of BEAM-302.
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BEAM-301 is a liver-targeting LNP formulation of base editing reagents designed to correct the R83C mutation, the most common disease-causing mutation that results in the most severe form of GSDIa. Given its rare nature and geographic distribution of disease burden, the company will focus development of BEAM-301 in the U.S. and expects to submit an IND application in the first half of 2024.
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In October 2023, Beam presented new preclinical data demonstrating the ability of a single administration of BEAM-301 to directly and durably correct the R83C mutation in vivo, with results lasting more than one year, in an oral presentation at the 30th Annual European Society of Gene & Cell Therapy (ESGCT) Congress in Brussels.
Immunology/Oncology Portfolio
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In September 2023, Beam announced dosing of the first patient in 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). The Phase 1/2 trial continues to enroll, and the company expects to report initial data in 2024.
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Beam plans to generate a focused clinical dataset for BEAM-201 and seek potential partnership for this and other potential ex vivo CAR-T programs, including Beam’s ongoing research into creating next-generation allogeneic cell therapies with multiplex base editing.
Research Portfolio
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Beam plans to focus near-term research and platform investments on specific applications leveraging Beam’s in vivo editing capabilities in the liver targeting both rare genetic and common disorders, as well as select opportunities in hematology and immunology/oncology.
Corporate Updates
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Manufacturing: Beam has initiated current good manufacturing practice (GMP) operations at its North Carolina manufacturing facility, where capabilities include both CD34 cell manufacturing and LNP production, with additional capabilities expected to be added in the future.
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Business Development: In October 2023, Beam announced that Eli Lilly and Company (Lilly) acquired certain rights under Beam’s amended collaboration and license agreement with Verve Therapeutics, Inc. (Verve), including Beam’s opt-in rights to co-develop and co-commercialize Verve’s base editing programs for cardiovascular disease, which includes programs targeting PCSK9, ANGPTL3, and an undisclosed liver-mediated, cardiovascular target. Beam received a $200 million upfront payment and a $50 million equity investment from Lilly. Beam is also eligible to receive up to $350 million in potential future development-stage payments from Lilly upon the completion of certain clinical, regulatory and alliance events for a total of up to $600 million in potential total deal consideration.
Third Quarter 2023 Financial Results
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Cash Position: Cash, cash equivalents and marketable securities, excluding upfront proceeds from Lilly, were $1.0 billion as of September 30, 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 $100.0 million for the third quarter of 2023, compared to $85.3 million for the third quarter of 2022.
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General & Administrative (G&A) Expenses: G&A expenses were $25.4 million for the third quarter of 2023, compared to $21.8 million for the third quarter of 2022.
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Net Loss: Net loss was $96.1 million for the third quarter of 2023, or $1.22 per share, compared to $109.6 million for the third quarter of 2022, or $1.56 per share.
Cash Runway
Beam expects that its cash, cash equivalents and marketable securities as of September 30, 2023, together with upfront and equity investment proceeds received from Lilly, will enable the company to fund its anticipated operating expenses and capital expenditure requirements into the second half of 2026. This expectation assumes anticipated cost savings related to the company’s portfolio prioritization and streamlining of operations and 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.