On February 28, 2022 Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, reported pipeline and business highlights, outlined key 2022 anticipated milestones and reported fourth quarter and full year 2021 financial results (Press release, Beam Therapeutics, FEB 28, 2022, View Source [SID1234609108]).
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"Throughout 2021, we made important advancements across our platform and portfolio, culminating in the clearance of our first IND submission late last year and our recently launched collaboration with Pfizer, both of which provide further validation for the potential of our base editing and delivery technologies," said John Evans, chief executive officer of Beam. "In 2022, we plan to continue this momentum by executing the first wave of our long-term strategy for sickle cell disease with the planned initiation of our trial with BEAM-101, marking our transition to a clinical-stage company, and our anticipated IND submission for BEAM-102. There is a significant need for novel treatments for sickle cell disease and other severe genetic blood disorders, and we believe that our strategy and suite of technologies – base editing, improved conditioning and in vivo delivery for editing HSCs – has the potential to make an important impact on the treatment landscape for these patients."
Mr. Evans continued, "In parallel, we plan to actively advance the remainder of our pipeline in 2022, notably with expected milestones including an IND submission for BEAM-201 for the treatment of relapsed and refractory acute T-cell leukemia and lymphoblastic lymphoma, IND-enabling work for BEAM-301 for the treatment of glycogen storage disease Ia, the naming of additional development candidates from our pipeline, and continued advancement of our comprehensive technology platform in precision genetic medicine. This is an exciting time for Beam, and I’m optimistic about the year ahead as we work to bring potentially life-changing medicines to patients."
Pipeline and Business Highlights
Executed Multi-Target Research Collaboration with Pfizer to Advance Novel In Vivo Base Editing Programs for a Range of Rare Diseases: Beam and Pfizer entered into a four-year research collaboration focused on in vivo base editing programs for three targets for rare genetic diseases of the liver, muscle and central nervous system. Under the terms of the agreement, Beam received an upfront payment of $300 million and, assuming Pfizer exercises its opt-in license rights for all three targets, is eligible for development, regulatory and commercial milestone payments for potential total deal consideration of up to $1.35 billion. Beam is also eligible to receive royalties on global net sales for each licensed program. Beam will conduct all research activities through development candidate selection, and Pfizer may opt in to exclusive, worldwide licenses to each development candidate, after which it will be responsible for all development activities, as well as potential regulatory approvals and commercialization, for each such candidate. Beam has a right to opt in, at the end of Phase 1/2 studies, upon the payment of an option exercise fee, to a global co-development and co-commercialization agreement with respect to one program licensed under the collaboration pursuant to which Pfizer and Beam would share net profits, as well as development and commercialization costs in a 65%/35% ratio (Pfizer/Beam). The collaboration has an initial term of four years and may be extended up to one additional year.
Outlined Long-term Strategy for Base Editing Programs in Sickle Cell Disease at ASH (Free ASH Whitepaper) 2021: At the 63rd American Society for Hematology Annual Meeting & Exposition in December 2021, Beam shared a long-term, staged development strategy for its base editing approach to treat sickle cell disease (SCD). Beam’s stepwise strategy involves three waves:
Wave 1: Ex Vivo Base Editing via Autologous Transplant
Beam is advancing ex vivo base editing programs, in which cells will be collected from a patient, edited and then infused back into the patient following a conditioning regimen, such as treatment with busulfan, the standard of care in hematopoietic stem cell (HSC) transplantation today. This approach will be deployed in the company’s BEAM-101 and BEAM-102 base editing programs and is intended to allow the company to pursue an efficient path for development using increasingly validated clinical endpoints and regulatory strategies.
Wave 2: Improved Conditioning
In parallel with Wave 1 development, Beam also aims to improve the transplant conditioning regimen for SCD patients undergoing HSC transplantation, reducing toxicity challenges associated with standard of care conditioning, a critical component necessary to prepare a patient’s body for effective treatment. Beam has a collaboration with Magenta Therapeutics to evaluate the potential utility of MGTA-117, Magenta’s novel antibody drug conjugate that is designed to spare immune cells and precisely target hematopoietic stem and progenitor cells. Beam is also conducting its own research into novel conditioning strategies. If successful, improved conditioning regimens could potentially be paired with BEAM-101 and BEAM-102, as well as other base editing programs in hematology.
Wave 3: In Vivo Base Editing via HSC-targeted LNPs
Beam is also 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 HSCs, eliminating the need for transplantation altogether. This approach could provide a more accessible option for patients, particularly in regions where ex vivo treatment is challenging. Building on its acquisition of Guide Therapeutics, Beam is using a DNA-barcoded LNP screening technology to enable high-throughput in vivo identification of LNPs with novel biodistribution and selectivity for target organs beyond the liver.
Presented Preclinical Data Highlighting Approach to Creating Multiplex Edited CAR T-cells to Target CD5-positive Tumors at SITC (Free SITC Whitepaper): At the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s 36th Annual Meeting in November 2021, Beam announced new preclinical research demonstrating the ability of the company’s multiplex edited CAR T-cells to target CD5-positive tumors, leading to tumor clearance in vivo. Beam’s process utilizes base editing designed to simultaneously silence five target genes, including CD5 and PD1, to create allogeneic anti-CD5 CAR T-cells with enhanced effector function for potential use as off-the-shelf treatments for T-cell malignancies.
Key 2022 Anticipated Milestones
Ex Vivo HSC Programs
Enroll the first subject in the Phase 1/2 clinical trial of BEAM-101 for the treatment of SCD, which is referred to as the BEACON-101 trial, in the second half of 2022
Submit an investigational new drug (IND) application for BEAM-102 for the treatment of SCD in the second half of 2022
Ex Vivo T Cell Programs
Submit an IND application for BEAM-201 for the treatment of relapsed/refractory T cell acute lymphoblastic leukemia/T cell lymphoblastic lymphoma (T-ALL/TLL) in the second half of 2022
Nominate a second CAR T development candidate in 2022
In Vivo LNP Liver-targeting Programs
Initiate IND-enabling studies for BEAM-301, a liver-targeting LNP formulation of base editing reagents designed to correct the R83C mutation, the most common disease-causing mutation of glycogen storage disorder Ia (GSDIa), in 2022
Nominate a second liver-targeted development candidate in 2022
Fourth Quarter and Full Year 2021 Financial Results
Cash Position: Cash, cash equivalents and marketable securities were $965.6 million as of December 31, 2021 (which does not include the upfront payment from the Pfizer collaboration), compared to $299.7 million as of December 31, 2020.
Research & Development (R&D) Expenses: R&D expenses were $96.8 million for the fourth quarter of 2021 and $387.1 million for the full year ended December 31, 2021, compared to $32.5 million for the fourth quarter of 2020 and $103.2 million for the full year ended December 31, 2020.
General & Administrative (G&A) Expenses: G&A expenses were $17.8 million for the fourth quarter of 2021 and $57.2 million for the full year ended December 31, 2021, compared to $8.4 million for the fourth quarter of 2020 and $29.6 million for the full year ended December 31, 2020.
Net Loss: Net loss attributable to common stockholders was $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 year ended December 31, 2021, compared to $95.5 million, or $1.69 per share, for the fourth quarter of 2020 and $195.9 million, or $4.19 per share, for the full year ended December 31, 2020.