UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
FORM
CURRENT REPORT
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| Item 2.02. | Results of Operations and Financial Condition. |
Although it has not finalized its full financial results for the fourth quarter and fiscal year ended December 31, 2022, Beam Therapeutics Inc. (the “Company”) announced in a press release on January 9, 2023 that it estimates that it had cash, cash equivalents and marketable securities of approximately $1.0 billion as of December 31, 2022.
The information contained in Item 2.02 of this Form 8-K regarding the Company’s estimated cash balance as of December 31, 2022 is preliminary, unaudited and is subject to completion of the Company’s financial statement closing procedures. This estimate also does not present all information necessary for an understanding of the Company’s financial condition as of December 31, 2022 and its results of operations for the three months and year ended December 31, 2022. Accordingly, undue reliance should not be placed on this preliminary estimate.
The information in this Item 2.02 is being furnished and shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”) or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended (the “Securities Act”) or the Exchange Act, except as expressly set forth by specific reference in such a filing.
| Item 7.01. | Regulation FD Disclosure. |
On January 9, 2023, the Company updated its corporate presentation that it intends to use in connection with presentations at conferences and meetings, including an investor presentation at the 41st Annual J.P. Morgan Healthcare Conference on January 9, 2023. The slides from the Company’s corporate presentation are furnished as Exhibit 99.1 to this Current Report on Form 8-K and are incorporated herein by reference.
The information in this Item 7.01 (including Exhibit 99.1 attached hereto) is being furnished and shall not be deemed “filed” for purposes of Section 18 of the Exchange Act or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference into any filing under the Securities Act or the Exchange Act, except as expressly set forth by specific reference in such filling.
| Item 8.01. | Other Events. |
On January 9, 2023, the Company issued a press release announcing progress across its base editing portfolio and outlining key anticipated milestones. The full text of the press release is filed as Exhibit 99.2 to this Current Report on Form 8-K and is incorporated herein by reference. The information contained on the website referenced in the press release is not incorporated herein.
Cautionary Note Regarding Forward-Looking Statements
Statements in this Current Report on Form 8-K about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute “forward-looking statements” within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the Company’s upcoming presentations at the 41st Annual J.P. Morgan Healthcare Conference; the Company’s plans, and the anticipated timing, to advance its programs; the Company’s expectations for transitioning to a multi-program clinical stage company; the therapeutic applications and potential of the Company’s technology, including with respect to sickle cell disease, glycogen storage disease 1a, relapsed/refractory T-cell acute lymphoblastic leukemia/T-cell lymphoblastic lymphoma, alpha-1 antitrypsin deficiency and the Company’s conditioning regimens; the clinical trial design for BEAM-201; the Company’s estimated cash, cash equivalents and marketable securities as of December 31, 2022 and its expectations related thereto; the sufficiency of the Company’s capital resources to fund operating expenses and capital expenditure requirements and the period in which such resources are expected to be available; and the Company’s ability to develop life-long, curative, precision genetic medicines for patients through base editing. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,”
“potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Each forward-looking statement is subject to important risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement, including, without limitation, risks and uncertainties related to: the Company’s ability to develop, obtain regulatory approval for, and commercialize the Company’s product candidates, which may take longer or cost more than planned; the Company’s ability to raise additional funding, which may not be available; the Company’s ability to obtain, maintain and enforce patent and other intellectual property protection for the Company’s product candidates; the potential impact of COVID-19 and its variants on the Company’s business; the uncertainty that the Company’s product candidates will receive regulatory approval necessary to initiate human clinical studies; that preclinical testing of the Company’s product candidates and preliminary or interim data from preclinical studies and clinical trials may not be predictive of the results or success of ongoing or later clinical trials; that enrollment and initiation of the Company’s clinical trials may take longer than expected; that the Company’s product candidates may experience manufacturing or supply interruptions or failures; risks relate to competitive products; whether the Company’s actual audited results will be consistent with its estimated cash, cash equivalents and marketable securities as of December 31, 2022; and other factors discussed in the “Risk Factors” and “Risk Factors Summary” sections of the Company’s Annual Report on Form 10-K for the year ended December 31, 2021, filed with the SEC on February 28, 2022, and in the “Risk Factors” section of the Company’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2022 filed with the SEC on November 7, 2022, and in other filings that the Company makes with the SEC in the future. These forward-looking statements speak only as of the date of this Current Report on Form 8-K. Factors or events that could cause our actual results to differ may emerge from time to time, and it is not possible for the Company to predict all of them. The Company undertakes no obligation to update any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by applicable law.
| Item 9.01. | Financial Statements and Exhibits. |
(d) Exhibits
| Exhibit No. |
Description | |
| 99.1 | Beam Therapeutics Inc. Corporate Presentation | |
| 99.2 | Press Release dated January 9, 2023 | |
| 104 | Cover Page Interactive Data File (embedded within the Inline XBRL document) | |
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, as amended, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
| BEAM THERAPEUTICS INC. | ||||||
| Date: January 9, 2023 | By: | /s/ John Evans | ||||
| John Evans | ||||||
| Chief Executive Officer | ||||||
PRECISION GENETIC MEDICINES THROUGH BASE EDITING Beam Therapeutics NASDAQ: BEAM Exhibit 99.1
This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding: the initiation, timing, progress and results of preclinical studies and research and development programs, including the initiation and progress of clinical trials, including our BEACON trial and our BEAM-201 trial; the advancement of our pipeline, including the advancement of BEAM-101, BEAM-201, BEAM-301, BEAM-302, additional CAR-T and liver programs, and Stargardt disease program in multiple preclinical studies; our current expectations and anticipated results of operations, including our expected use of capital; the potential activities and benefits under license and collaboration agreements and the formation of new collaborations; and the therapeutic applications and potential of our technology, including our potential to develop life-long, curative, precision genetic medicines for patients through base editing, including potential safety advantages, all of which are subject to known and unknown important risks, uncertainties and other factors that may cause our actual results, performance or achievements, market trends, or industry results to differ materially from those expressed or implied by such forward-looking statements. Therefore, any statements contained herein that are not statements of historical fact may be forward-looking statements and should be evaluated as such. Without limiting the foregoing, the words “anticipate,” “expect,” “suggest,” “plan,” “vision,” “believe,” “intend,” “project,” “forecast,” “estimates,” “targets,” “projections,” “potential,” “should,” “could,” “would,” “may,” “might,” “will,” and the negative thereof and similar words and expressions are intended to identify forward-looking statements. Each forward-looking statement is subject to important risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement, including, without limitation, risks and uncertainties related to: our ability to develop, obtain regulatory approval for, and commercialize our product candidates, which may take longer or cost more than planned; our ability to raise additional funding, which may not be available; our ability to obtain, maintain and enforce patent and other intellectual property protection for our product candidates; the potential impact of the COVID-19 pandemic; that preclinical testing of our product candidates and preliminary or interim data from preclinical studies and clinical trials may not be predictive of the results or success of ongoing or later clinical trials; that initiation and enrollment of our clinical trials may take longer than expected; that our product candidates may experience manufacturing or supply interruptions or failures; risks related to competitive products; and the other risks and uncertainties identified under the headings “Risk Factors Summary” and “Risk Factors” and elsewhere in our annual report on Form 10-K for the year ended December 31, 2021, our Quarterly Report on Form 10-Q for the quarter ended March 31, 2022, our Quarterly Report on Form 10-Q for the quarter ended June 30, 2022, our Quarterly Report on Form 10-Q for the quarter ended September 30, 2022, and in any subsequent filings with the Securities and Exchange Commission (the “SEC”) which are available on the SEC’s website at www.sec.gov. Additional information will be made available by our annual and quarterly reports and other filings that we make from time to time with the SEC. These forward-looking statements speak only as of the date of this presentation. Factors or events that could cause our actual results to differ may emerge from time to time, and it is not possible for us to predict all of them. We undertake no obligation to update any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by applicable law. Cautionary note regarding forward-looking statements .
Potential for one-time, curative therapies Gene editing for rare and common diseases Platform for rapidly-programmable precision medicines Our vision is to provide life-long cures for patients suffering from serious diseases
Base editing is a differentiated, potentially best-in-class gene editing technology Precise targeting? Yes (guide RNA or ZF/TALE) Yes (guide RNA) Durability of edit? Permanent Permanent Double strand breaks? Yes No Applications? Primarily knockout Correct, modify, activate, multiplex Editing predictability Random insertions and deletions 100s of uncharacterized edits Single base edits All edits fully characterized Efficiency of precise edit? Low – dividing cells only High – any cell type Nuclease CRISPR, ZFN, TALENs Base editing
A precise gene editing technology with highly versatile applications CRISPR Protein Deaminase Guide RNA Correct proteins (eg, BEAM-301, BEAM-302) 3 Modify surface proteins (eg, ESCAPE) 4 Activate expression (eg, BEAM-101) X Knock out proteins (eg, BEAM-201) 2 Multiplex simultaneous edits (eg, four gRNAs in BEAM-201) X + - 1 5 …And many other applications possible
We are establishing a leading platform for precision genetic medicine Payload Manufacturing Delivery Suite of gene editing technologies Base editing ABE: A-to-G (or T-to-C) editors CBE: C-to-T (or G-to-A) editors Additional kinds of base editors Nuclease editing RNA editing Prime editing Suite of delivery technologies Autologous cell therapy Allogeneic cell therapy mRNA LNP vectors Viral vectors Internal manufacturing capability 100,000 square foot cGMP clinical/commercial facility in NC, phased build, anticipated to be operational in 2023
Advancing a diversified pipeline into the clinic LNP = Lipid Nanoparticle; AAV = Adeno Associated Virus; HSC = Hematopoietic Stem Cell; T-ALL / T-LL = T-Cell Acute Lymphoblastic Leukemia / T-Cell Lymphoblastic Lymphoma; AML = Acute Myeloid Leukemia; ESCAPE: Engineered Stem Cell Antibody Paired Evasion DELIVERY PROGRAM / DISEASE EDITING APPROACH RESEARCH LEAD OPTIMIZATION IND ENABLING PHASE I/II PIVOTAL Ex vivo HSCs BEAM-101 Sickle Cell Disease Beta Thalassemia Activation of fetal hemoglobin BEAM-102 Sickle Cell Disease Correction of HbS sickle mutation ESCAPE Sickle Cell Disease Beta Thalassemia Multiplex CD117 edit-antibody pair Ex vivo T cells BEAM-201 T-ALL / T-LL CD7+ AML Multiplex silenced CD7 CAR-T In vivo LNP BEAM-301 Glycogen Storage Disease Ia Correction of R83C mutation In vivo LNP BEAM-302 Alpha-1 Antitrypsin Deficiency Correction of E342K mutation In vivo LNP Glycogen Storage Disease Ia Correction of Q347X mutation In vivo LNP Hepatitis B Virus Multiplex silencing In vivo LNP Complement Pathway (Apellis) Undisclosed 3 undisclosed targets (Pfizer) Undisclosed AAV Stargardt Disease Correction of G1961E mutation Refocusing on ESCAPE or in vivo delivery
Lead Programs: Potentially de-risk technology (higher probability of technical success, faster path), generate revenue, and benefit patients with high unmet need Future platforms: Expand addressable patient populations to create highly valuable, differentiated franchises through further innovation in editing and delivery HEMATOLOGY BEAM-101 ESCAPE for conditioning In vivo delivery GENETIC DISEASES BEAM-301, BEAM-302 Multiple new liver targets Barcoded LNP beyond liver Near term: Future platforms: IMMUNOLOGY- ONCOLOGY BEAM-201 Next-generation allogeneic platform (4-6+ edits) Beam is developing medicines across three franchises, each with near- and long-term potential ESCAPE: Engineered Stem Cell Antibody Paired Evasion
Key progress and anticipated milestones 2022 Achievements Upcoming Milestones ✓ ✓ ✓ ✓ ✓ Data presentation on multiple patients from BEACON in 2024 Regulatory filing for BEAM-301 by late 2023 / early 2024 Complete sentinel cohort enrollment and initiate expansion cohort of BEACON in 2023 Dose first BEAM-201 patient by mid 2023 Regulatory filing for BEAM-302 in early 2024 Refocused on new technology: ESCAPE & LNP Initiate IND-enabling studies for BEAM-301 Nominated BEAM-302 development candidate First subject enrolled for BEAM-101 Submit IND for BEAM-201 and respond to hold Refocused on next gen allogeneic strategies Strategic platform partnerships (Pfizer, Orbital) Hematology Immunology - Oncology Genetic disease Platform ✓ ✓
Designed for best-in-class profile: One-time therapy with potential for highest fetal hemoglobin (HbF) induction Direct editing of HbF genes to turn them on Potential for greatest reduction of disease-causing HbS due to hemoglobin switching Non-viral: No detectable random insertion Non-cutting: Lower risk for genotoxic stress and chromosomal abnormalities Investment in patient delivery to differentiate: Wholly owned manufacturing: control over quality and connection to patient services Investment in patient services: optimizing patient experience BEAM-101: Designed to treat sickle cell disease with a potentially one-time, direct, non-cutting activation of HbF Sickle Cell Disease: 100,000 patients in the US; severe pain crises, multi-organ damage, early mortality HPFH = Hereditary Persistence of Fetal Hemoglobin HBB HBG1 HBG2 A single base editor + gRNA edits regulatory element of both fetal hemoglobin genes, without cutting DNA Sickle hemoglobin (HbS) gene Duplicated fetal hemoglobin (HbF) genes HEMATOLOGY
Potentially best-in-class attributes of BEAM-101 product Base editing at HBG1/2 promoters1 HbF protein levels2 Preclinical data presented at ASGCT 2020; Edited human HSPCs analyzed 16 weeks after infusion in NBSGW mice (Mean±SEM, n=4-6); 1. Sorted human Lineage-CD34+ bulk bone marrow; 2. Sorted erythroid cells (GlyA+) HbS protein levels2 Edited human CD34+ cells followed by 16 week engraftment in mice >90% 65% <40% Potential for highest HbF induction and lowest residual HbS levels versus other approaches in the field Building capabilities for potential best-in-class patient delivery including internal manufacturing HEMATOLOGY
BEAM-101 is the first clinical base editing program in the U.S., accelerating path to patients and the market BEACON-101 Phase 1/2 Study Design Manufacturing Conditioning & Transplant Engraftment Follow-up Patient 1 Patient 2 Patient 3 Patients 4-45 Transfusion & Mobilization 6 months (+ / -) Select safety endpoints Proportion of patients with successful neutrophil engraftment by day 42 Safety and tolerability assessments Select inclusion criteria Patients with sickle cell disease (SCD) with severe veno-occlusive crises despite hydroxyurea or other supportive measures Age ≥18 to ≤35 years for initial cohort Select efficacy endpoints Severe vaso-occlusive crises Transfusion requirements Hemoglobin F levels Quality of life and ability to function Markers of red blood cell function and organ damage HEMATOLOGY
Well positioned to deliver potentially best-in-class regimens for SCD patients, now and in the future Precise gene editing (non-cutting, non-viral) Busulfan conditioning BEAM-101 (HbF) Less toxic conditioning selects for edited cells – potential to expand to younger and broader patient population ESCAPE-1 (HbF+CD117) ESCAPE-2 (Makassar+CD117) In vivo editing after infusion of HSC-targeted LNPs (no transplant) HbF + Makassar available Wave 1 Base Editing + HSC Transplant Wave 2 Improved Conditioning Wave 3 In vivo Delivery HEMATOLOGY * ESCAPE: Engineered Stem Cell Antibody Paired Evasion
Stem cell factor (SCF) signaling via CD117 is required for HSC survival and proliferation A single base edit changes an epitope on the CD117 receptor and is designed not to impact HSC biology Customized conditioning antibody depletes diseased unedited cells, but enables CD117-edited cells to “ESCAPE” and grow normally ESCAPE* designed for selective depletion of diseased cells, which may enable non-genotoxic conditioning Enrichment of edited cells in presence of antibody Paired CD117 antibody (ng/mL) HSC eHSC Cell Dies Cell Survives * ESCAPE: Engineered Stem Cell Antibody Paired Evasion HEMATOLOGY
GSD1a unmet need: Low G6PC activity can result in severe drop in blood glucose levels within 1-3 hrs Hypoglycemia may result in seizures or can be lethal Multiple organ dysfunction (e.g. renal and liver) BEAM-301 potential: Near-normal serum metabolites, G6PC activity, hepatic morphology, increased survival in mice Animal studies suggest 11% editing sufficient for restoring fasting glucose1 Key points: Beam’s first in vivo DC First DC in industry with in vivo direct correction gene editing2 Regulatory filing expected by late 2023 / early 2024 Chou & Mansfield. 2007. Curr. Gen. Ther. Based on publicly announced development candidates Wildtype G6PC gene G6PC R83C mutation BEAM-301 program aims to restore impaired glycogen metabolism which otherwise causes significant morbidity Liver Low G6P conversion High G6P conversion Glycogen Storage Disease Ia: 900 US R83C patients; severe hypoglycemia, liver & kidney dysfunction GENETIC DISEASE
Preclinical data presented at ESGCT 2021; 1. Homozygous huG6PC-R83C mice untreated or treated with LNP via temporal vein shortly after birth, and untreated mice survived less than 3 days with glucose therapy BEAM-301 program aims to restore impaired glycogen metabolism which otherwise causes significant morbidity ABE correction of GSDIa R83C mutation associated with improved survival of R83C mice1 Near-normal serum metabolites, G6PC activity, hepatic morphology and lipid deposition GENETIC DISEASE
Potential one time treatment to create permanent correction of E342K and enable normal A1 secretion and gene regulation Designed to address disease pathology in both the liver and lung In preclinical studies, lead candidate delivered up to 27% correction editing, that resulted in >3X increase in A1AT protein (> 11 uM protective threshold) at clinically-relevant dose of 0.75mpk In a minority of cells, correction resulted in wildtype allele plus D341G allele (bystander) that was observed to function normally BEAM-302 nominated for development; regulatory filing expected in early 2024 BEAM-302: Development candidate nominated for potential one-time treatment of AATD Corrected SERPINA1 (wildtype) E342K (PiZ) mutation SERPINA1 with D341G bystander edit GENETIC DISEASE Alpha-1 Anti-trypsin Deficiency (AATD): 60,000 ZZ patients in US; severe progressive lung & liver disease
BEAM-302 program has the potential to address both lung and liver pathology of AATD in one course treatment 4.9-fold increase in functional A1AT secretion Reduction in toxic liver aggregates Control Correction Group Preclinical data presented at ASGCT 2020; Editing in NSG-PiZ mice with either control (PCSK9) or correction (E342K) provided above results Representative in vivo studies of PiZZ mouse with precursor base editors GENETIC DISEASE
Multiplex base editing: Unlike nuclease editors, no detected chromosomal rearrangements, normal cell expansion, and no detected DNA damage response in preclinical studies Clinical-scale process: 96-99% editing, >90% quad edited1 BEAM-201 US IND cleared; first patient dosing expected by mid-2023 BEAM-201: Base edited allogeneic cell therapy candidate with an opportunity to treat aggressive CD7+ leukemias T-Cell Acute Leukemia: 15% of ALL, not treated by B-cell CARTs, few options for relapsed/refractory patients IMMUNOLOGY-ONCOLOGY Base editor mRNA TRAC gRNA: Prevent graft-vs-host disease CD52 gRNA: Enable allogeneic cell source PD1 gRNA: Prolong efficacy CD7 gRNA: Prevent fratricide from CD7 CAR C G C G C G C G Clinical-scale process yielded 96-99% editing, >90% quad edited1 Unedited BEAM-201 CD7 CAR Preclinical data presented at SITC 2020; 1. Simultaneous base editing at four target loci using clinical-scale process as measured by NGS.
BEAM-201: Significant advantages of multiplex base editing without double strand breaks Chromosomal rearrangements Impact on cell expansion 4 edits: TRAC, CD52, PD1, CD7 3 edits: TRAC, B2M, PD1 Base editing Nuclease Percent of cells with translocations1 Percent yield after editing2 Preclinical data presented at SITC 2020; 1. Base editing versus nuclease editing with the same four guide RNAs measured via G-banded karyotypes from 100 cells; updated analysis shows <0.1% translocations using first generation CBE (data unpublished) 2. Extensive guide screen across three targets, with BE4 and spCas9 sgRNAs selected for high editing efficiency and expansion in single-plex test, final cell yields compared between 3 edits, normalized to electroporation only control IMMUNOLOGY-ONCOLOGY <40% Multiplex editing more efficient with base editing which translates to better cell product Optimization of platform ongoing with focus on generating next generation “true allogeneic” products
BTX-ALO-001: Multiplex edited BEAM-201 enables evaluation in aggressive T-cell cancers using optimized lymphodepletion (LD) Select safety endpoints Incidence and severity of treatment emergent adverse events (TEAE) and treatment-related AEs, including serious AEs (SAEs) and DLTs Select inclusion criteria ≥18 to ≤50 yrs for dose exploration ≥1 yrs for peds after FDA review T-ALL or T-LL with one of following: Relapsed after 2nd CR Relapse after HSCT Primary refractory or R/R Eligible for allo HSCT (donor available) Select efficacy endpoints Proportion of T-ALL pts with CR or CRi or T-LL pts with CR or PR any time post BEAM-201 Proportion of pts eligible for HSCT based on response to BEAM-201 Proportion achieving MRD-negative status Duration of response, OS, etc Phase 1 dose exploration (≤36 pts) Phase 2 (~48 pts) Expansion Cohort 2 Expansion Cohort 1 Pediatric cohort (gated initiation of enrollment) LD Regimen 1 (Up to 3 CAR-T dose levels) LD Regimen 2 (Up to 3 CAR-T dose levels) Recommended phase 2 dose and LD regimen Phase 1 dose expansion (≤12 pts) IMMUNOLOGY-ONCOLOGY T-ALL = T cell acute lymphoblastic leukemia; T-LL = T cell lymphoblastic lymphoma; CR = Complete response; CRi = CR with incomplete count recovery; PR = Partial response; OS = Overall survival; HSCT = Hematopoietic stem cell transplant; DLT = Dose limiting toxicity; MRD = Minimal residual disease
Lead Programs: Potentially de-risk technology (higher probability of technical success, faster path), generate revenue, and benefit patients with high unmet need Future platforms: Expand addressable patient populations to create highly valuable, differentiated franchises through further innovation in editing and delivery HEMATOLOGY BEAM-101 ESCAPE for conditioning In vivo delivery GENETIC DISEASES BEAM-301, BEAM-302 Multiple new liver targets Barcoded LNP beyond liver Near term: Future platforms: IMMUNOLOGY- ONCOLOGY BEAM-201 Next-generation allogeneic platform (4-6+ edits) Beam is developing medicines across three franchises, each with near- and long-term potential ESCAPE: Engineered Stem Cell Antibody Paired Evasion
Additional strategic and innovator deals potentially unlock base editing value and broaden therapeutic impact License to Beam’s base editing technology for the prevention of cardiovascular disease 3 targets: VERVE-101 (PCSK9), VERVE-102 (ANGPTL3), Undisclosed #3 Beam opt-in after P1: 50% US (VERVE-101, VERVE-102) or 35% of WW (Target 3) cost/profit $75M in upfront payments for base editing for complement mediated diseases Beam opt-in to 50% of US rights after Phase 1 on one program Prime editing (PE) is a novel gene editing technology, complementary to base editing Beam provides delivery and CRISPR technology/know-how Beam has exclusive rights to PE: SCD transversion edit, any transitions (30% of mutations) $50M upfront for license to Cas12b nuclease for certain engineered cell therapies Non-exclusive license – Beam retains ability to use or repartner Cas12b $300M upfront, $1B+ in potential milestones 3 gene targets using Beam’s editing and delivery to target liver, muscle, CNS Beam option at end of P1/2 for 35% WW cost/net profit split on one program Next-gen RNA and delivery; Beam provides interim leadership and RNA/LNP capabilities Beam has meaningful equity stake in Orbital Beam access to Orbital IP for gene editing (exclusive) and certain fields (non-exclusive) Strategic deals Innovator deals
Meet the Beam Team Significant team track record in discovery, development, approval of first-in-class medicines John Evans Chief Executive Officer Giuseppe Ciaramella, PhD President, Chief Scientific Officer Courtney Wallace Chief Business Officer Susan O’Connor Chief Human Resources Officer Christine Bellon PhD, JD Chief Legal Officer Suzanne Fleming Chief Accounting Officer Terry-Ann Burrell Chief Financial Officer Brian Riley Chief Manufacturing Officer Amy Simon, MD Chief Medical Officer Manmohan Singh, PhD Chief Technology Officer John Lo, PhD Chief Commercial Officer
Thank you
Exhibit 99.2
Beam Therapeutics Reports Progress Across Base Editing Portfolio and Outlines Key Anticipated Milestones
BEACON Trial of BEAM-101 in Sickle Cell Disease Ongoing with Data from Multiple Patients Expected in 2024; Expansion Phase Initiation Expected in 2023
First Patient Dosing in BEAM-201 Trial in Patients with T-ALL/T-LL Expected by Mid-2023
Regulatory Submissions Planned for BEAM-301 by Late 2023/Early 2024 and BEAM-302 in Early 2024
North Carolina Manufacturing Facility Expected to Initiate GMP Operations in Late 2023
Approximately $1 Billion Estimated Cash, Cash Equivalents and Marketable Securities at Year-End 2022; Cash Runway Expected into 2025, through Anticipated Key Milestones for Lead Programs and Long-Term Platform Opportunities
CAMBRIDGE, Mass., January 9, 2023 – Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today reported progress across the company’s hematology, immunology-oncology and genetic disease portfolios and provided updates on anticipated upcoming milestones.
“Beam enters 2023 with significant momentum across all of our core pipeline areas and an expanding leadership position in the next generation of gene editing,” said John Evans, chief executive officer of Beam. “We have multiple clinical-stage candidates with BEAM-101 and BEAM-201, another two candidates – BEAM-301 and BEAM-302 – moving toward clinical trials, and an integrated platform of editing technologies, scalable manufacturing capabilities and diverse delivery modalities. We are also making key investments in long-term platform opportunities that may dramatically expand the reach and impact of base editing and create a sustainable pipeline of highly differentiated programs, including improved conditioning for transplant, allogeneic cell therapies and in vivo delivery. I’m so proud of the accomplishments this organization has achieved in our first five years of operations. We believe we now have a unique opportunity to deliver on the potential of our science and achieve our mission of bringing life-changing treatments to patients suffering from serious diseases.”
Hematology Portfolio
| • | BEAM-101: In November 2022, Beam enrolled the first patient in its BEACON clinical trial evaluating BEAM-101 as a treatment for sickle cell disease (SCD). Beam expects to complete enrollment in the sentinel cohort and initiate enrollment in the expansion cohort of BEACON in 2023, with plans to report data from multiple patients from one or both cohorts in 2024. BEAM-101 is a patient-specific, autologous hematopoietic stem cell (HSC) investigational therapy designed to offer a potentially best-in-class profile, incorporating base edits that are intended to mimic single nucleotide polymorphisms seen in individuals with hereditary persistence of fetal hemoglobin. BEAM-101 aims to potentially alleviate the effects of SCD or beta-thalassemia by leading to increases in fetal hemoglobin, which is expected to restore the formation of a functional hemoglobin tetramer and, in the case of SCD, inhibit hemoglobin S polymerization. |
| • | Platform Opportunity: Beam is advancing its Engineered Stem Cell Antibody Paired Evasion (ESCAPE) conditioning strategy in an effort to bring base editing treatments to more patients. ESCAPE aims to avoid toxicity challenges associated with currently available conditioning regimens for patients with SCD and beta-thalassemia ahead of autologous transplant. ESCAPE may also have applications in other diseases of the blood and immune system where transplant could deliver potential benefits but has been limited by toxicities associated with standard conditioning regimens. In December 2022, Beam presented in vivo proof-of-concept data at the American Society of Hematology Annual Meeting and Exposition (ASH) highlighting its potential. Beam has made significant investments in its ESCAPE platform and plans to continue its advancement in 2023. |
Immunology-oncology Portfolio
| • | BEAM-201: In December 2022, Beam received clearance from the FDA for its Investigational New Drug (IND) application for BEAM-201. The company has initiated a first-in-human Phase 1/2 clinical trial to evaluate the safety and efficacy of BEAM-201 in patients with relapsed/refractory T-cell acute lymphoblastic leukemia (T-ALL)/T-cell lymphoblastic lymphoma (T-LL) and expects to dose the first patient by mid-2023. The Phase 1 portion of the trial is expected to include up to 48 patients between the ages of 18 and 50, followed by a Phase 2 portion with approximately 48 patients. Key safety endpoints for the trial include treatment-emergent and treatment-related adverse events, and key efficacy endpoints include proportion of patients with complete or partial responses, proportion eligible for HSC transplant, and proportion achieving minimal residual disease negative status. Beam believes that BEAM-201 is the first quadruple-edited, allogeneic CAR-T cell investigational therapy in clinical-stage development. BEAM-201 is designed to target CD7 to treat relapsed/refractory T-ALL/T-LL, a severe disease affecting children and adults. |
| • | Platform Opportunity: Beyond BEAM-201, Beam continues to research potential next-generation allogeneic strategies that could dramatically expand the utility and accessibility of cell therapies in cancer and other diseases. Beam anticipates that multiple edits will be required to enable allogeneic cells to successfully avoid immune rejection and provide the cells with other desirable properties. Beam believes that multiplex base editing, with its high potency, efficiency in editing and lack of double-strand breaks, is well suited for making such highly engineered cells, and anticipates providing additional updates on this research in 2023. |
Genetic Disease Portfolio
| • | BEAM-301: IND-enabling studies for BEAM-301 continue, and by late 2023 or early 2024, the company plans to submit a regulatory application for authorization to initiate clinical trials for the program. BEAM-301 is a liver-targeting lipid nanoparticle (LNP) formulation of base editing reagents designed to correct the R83C mutation, the most common disease-causing mutation which results in the most severe form of glycogen storage disease 1a (GSDIa). GSDIa is an autosomal recessive disorder caused by mutations in the G6PC gene that disrupt a key enzyme, glucose-6-phosphatase, critical for maintaining glucose homeostasis. Patients with this mutation typically require ongoing corn starch administration, without which, they may enter into hypoglycemic shock within one to three hours. |
| • | BEAM-302: Beam also continues to advance its second liver-targeted in vivo program, BEAM-302, and in early 2024, plans to submit a regulatory application for authorization to initiate clinical trials for the program. BEAM-302 is designed to offer a one-time treatment to genetically correct the E342K point mutation (PiZZ genotype), which is most commonly responsible for severe alpha-1 antitrypsin deficiency (AATD). AATD is an inherited genetic disorder that can cause early onset emphysema and liver disease. |
| • | Platform Opportunity: Beam continues to advance its LNP delivery technologies using its barcode screening technology, which is designed to enable delivery of base editing treatments to the liver and tissues beyond, potentially expanding the number of diseases and patients that could benefit from base editing medicines. |
Manufacturing Facility
| • | Beam continues to expect operations at its North Carolina manufacturing facility to commence in the first quarter of 2023 and expects to initiate current good manufacturing practice compliant operations in late 2023. |
Cash Position and Runway
| • | Cash Position: Beam estimates that it had cash, cash equivalents and marketable securities of approximately $1.0 billion as of December 31, 2022. This estimate is preliminary, unaudited and is subject to completion of Beam’s financial statement closing procedures. This estimate also does not present all information necessary for an understanding of Beam’s financial condition as of December 31, 2022, and its results of operations for the three months and year ended December 31, 2022. Accordingly, undue reliance should not be placed on this preliminary estimate. |
| • | Cash Runway: Beam expects that its cash, cash equivalents and marketable securities as of December 31, 2022, 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 milestones for BEAM-101, BEAM-201, BEAM-301 and BEAM-302 described above, as well as continued investments in platform advancements and manufacturing capabilities. |
J.P. Morgan Healthcare Conference
Beam management will present and discuss Beam’s pipeline and business updates during a presentation at the 41st Annual J.P. Morgan Healthcare Conference today, Monday, January 9, 2023, at 11:15 a.m. PT. A live webcast will be available in the investor section of the company’s website at www.beamtx.com and will be archived for 60 days following the presentation.
About Beam Therapeutics
Beam Therapeutics (Nasdaq: BEAM) is a biotechnology company committed to establishing the leading, fully integrated platform for precision genetic medicines. To achieve this vision, Beam has assembled a platform that includes a suite of gene editing and delivery technologies and is in the process of building internal manufacturing capabilities. Beam’s suite of gene editing technologies is anchored by base editing, a proprietary technology that is designed to enable precise, predictable and efficient single base changes, at targeted genomic sequences, without
making double-stranded breaks in the DNA. This has the potential to enable a wide range of potential therapeutic editing strategies that Beam is using to advance a diversified portfolio of base editing programs. Beam is a values-driven organization committed to its people, cutting-edge science, and a vision of providing life-long cures to patients suffering from serious diseases.
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Investors are cautioned not to place undue reliance on these forward-looking statements, including, but not limited to, statements related to: our upcoming presentations at the 41st Annual J.P. Morgan Healthcare Conference; our expectations for transitioning to a multi-program clinical stage company; the therapeutic applications and potential of our technology, including with respect to SCD, GSDIa, T-ALL/TLL, AATD and our conditioning regimens; the clinical trial design for BEAM-201; our plans, and anticipated timing, to advance our programs; our estimated cash, cash equivalents and marketable securities as of December 31, 2022 and our expectations related thereto; the sufficiency of our capital resources to fund operating expenses and capital expenditure requirements and the period in which such resources are expected to be available; and our ability to develop life-long, curative, precision genetic medicines for patients through base editing. Each forward-looking statement is subject to important risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement, including, without limitation, risks and uncertainties related to: our ability to develop, obtain regulatory approval for, and commercialize our product candidates, which may take longer or cost more than planned; our ability to raise additional funding, which may not be available; our ability to obtain, maintain and enforce patent and other intellectual property protection for our product candidates; the potential impact of the COVID-19 pandemic, including its impact on the global supply chain; the uncertainty that our product candidates will receive regulatory approval necessary to initiate human clinical studies; that preclinical testing of our product candidates and preliminary or interim data from preclinical studies and clinical trials may not be predictive of the results or success of ongoing or later clinical trials; that enrollment and initiation of our clinical trials may take longer than expected; that our product candidates may experience manufacturing or supply interruptions or failures; risks related to competitive products; whether our actual audited results will be consistent with our estimated cash, cash equivalents and marketable securities as of December 31, 2022; and the other risks and uncertainties identified under the headings “Risk Factors Summary” and “Risk Factors” in our Annual Report on Form 10-K for the year ended December 31, 2021, our Quarterly Report on Form 10-Q for the quarter ended September 30, 2022, and in any subsequent filings with the Securities and Exchange Commission. These forward-looking statements speak only as of the date of this press release. Factors or events that could cause our actual results to differ may emerge from time to time, and it is not possible for us to predict all of them. We undertake no obligation to update any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by applicable law.
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Contacts:
Investors:
Chelcie Lister
THRUST Strategic Communications
chelcie@thrustsc.com
Media:
Dan Budwick
1AB
dan@1abmedia.com