Beam Therapeutics Inc. · NASDAQ:BEAM
Beam Therapeutics — Investor Presentation (January 2023)
25 slides from Beam Therapeutics's investor presentation, Jan 9, 2023
Slide 1: Precision Genetic Medicines Through Base Editing
PRECISION GENETIC MEDICINES THROUGH BASE EDITING Beam Therapeutics NASDAQ: BEAM Exhibit 99.1
Slide 2: Forward-looking statements
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, i
Slide 3: Potential for one-time, curative therapies
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
Slide 4: Base editing is a differentiated, potentially best-in-class gene editing technology
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 predict
Slide 5: A precise gene editing technology with highly versatile applications
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 g
Slide 6: We are establishing a leading platform for precision genetic medicine
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 de
Slide 7: Advancing a diversified pipeline into the clinic
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 Evasi
Slide 8: Beam is developing medicines across three franchises
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 i
Slide 9: Key progress and anticipated milestones
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 B
Slide 10: BEAM-101: designed to treat sickle cell disease with one-time HbF activation
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: Lowe
Slide 11: Potentially best-in-class attributes of BEAM-101 product
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 eryt
Slide 12: BEAM-101 is the first clinical base editing program in the U.S.
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
Slide 13: Well positioned to deliver best-in-class regimens for SCD patients
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 ES
Slide 14: Stem cell factor signaling via CD117 is required for HSC survival
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
Slide 15: GSD1a unmet need and BEAM-301 potential
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
Slide 16: ABE correction of GSDIa R83C mutation improved survival in mice
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 signifi
Slide 17: BEAM-302: development candidate for potential one-time treatment of AATD
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 A1A
Slide 18: BEAM-302 could address both lung and liver pathology of AATD
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 contro
Slide 19: BEAM-201: base edited allogeneic cell therapy candidate
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
Slide 20: BEAM-201: advantages of multiplex base editing without double strand breaks
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
Slide 21: BTX-ALO-001: trial design for BEAM-201 in T-cell cancers
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 cri
Slide 22: Beam is developing medicines across three franchises
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 i
Slide 23: Additional strategic and innovator deals broaden therapeutic impact
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-1
Slide 24: Meet the Beam Team
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 Christ
Slide 25: Thank you
Thank you
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