Beam Therapeutics Inc. · NASDAQ:BEAM
Beam Therapeutics — Investor Presentation (October 2023)
26 slides from Beam Therapeutics's investor presentation, Oct 19, 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 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 proteins (eg, ESCAPE) 4 Activate expression (eg, BEAM-101) X Knock out proteins (eg, BEAM-201) 2 Multiplex simultaneous edits (eg, four gRNAs in
Slide 6: Leading fully integrated platform for precision genetic medicines
We are establishing a leading fully integrated platform for precision genetic medicines FULLY INTEGRATED TECHNOLOGY AND CAPABILITIES MODULAR PLATFORM FOR RAPID DEVELOPMENT OF NEW BASE EDITING PROGRAMS
Slide 7: Portfolio strategy creates broad potential for wholly-owned programs and partnerships
Lead programs: Potentially de-risk technology, generate revenue, benefit patients with unmet need Future platforms: Expand addressable patient populations to create valuable, differentiated franchises Business strategy: Pursue both internal development of priority assets and external partnerships HE
Slide 8: Advancing a diversified pipeline into the clinic
PROGRAM / DISEASE DELIVERY EDITING APPROACH RESEARCH LEAD OPTIMIZATION IND ENABLING PHASE I/II PIVOTAL BEAM-101 Sickle Cell Disease Beta Thalassemia Ex vivo HSCs Activation of fetal hemoglobin ESCAPE Sickle Cell Disease Beta Thalassemia Ex vivo HSCs Multiplex CD117 edit-antibody pair BEAM-302 Alpha-
Slide 9: Key progress and anticipated milestones
Complete BEACON sentinel cohort enrollment and initiate enrollment of expansion cohort in 2023 Dose first patient in the BEAM-201 study Initiate preclinical studies for BEAM-301 and BEAM-302 NC manufacturing site GMP operational in late 2023 Data presentation on multiple patients from BEACON in 2024
Slide 10: 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 upregulation) Less toxic conditioning selects for edited cells – potential to expand to younger and broader patient
Slide 11: BEAM-101 designed for best-in-class profile
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 12: 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 13: 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 14: ESCAPE: CD117 signaling and HSC conditioning
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: BEAM-302: A potential one-time treatment of AATD
BEAM-302: A potential one-time treatment of AATD with potential to correct liver and lung disease Normal AAT Function Genetics Liver Respiratory AAT Deficiency Wild type SERPINA1 gene AAT protein is secreted, protecting lungs E342K* (PiZ) mutation AAT aggregates and causes liver damage/failure Low f
Slide 16: In vivo correction of PiZ mutation in mice with BEAM-302
In vivo correction of the causative AATD “PiZ” point mutation in mice with BEAM-302 BEAM-302 LNP Sequence for correction editing IV delivery NSG-PiZ Mouse 1-week Liver editing Numbers = % corrected alleles out of total alleles Corrected alleles include WT correction and WT correction plus D341G byst
Slide 17: BEAM-302 decreased liver aggregates
Correction of PiZ mutation in mice with BEAM-302* decreased liver aggregates Reduction in toxic liver aggregates Control Correction * Research grade BEAM-302
Slide 18: Measured secreted AAT in blood 1-week post-dose
Measured secreted AAT in blood 1-week post-dose AATD mice dosed with BEAM-302 had decreased serum PiZ AAT and increased corrected AAT
Slide 19: Increased serum AAT corresponded to increased functional AAT
Increased serum AAT in mice after BEAM-302 dosing corresponded to increased functional AAT Collect serum from dosed NSG-PiZ mice Incubate with neutrophil elastase Measure inhibition of elastase activity as Functional AAT ~3-fold increase Functional AAT * P<0.05, ***P<0.005, ****P<0.0005 One-way ANOV
Slide 20: 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 21: Preclinical data presented at ESGCT 2021
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 signific
Slide 22: 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 first patient dosed in August 2023 BEAM-201: Base edited allog
Slide 23: 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 24: Additional strategic and innovator deals
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: PCSK9 (VERVE-101 and VERVE-102), ANGPTL3 (VERVE 201), Undisclosed #3 Beam opt-in after P1: 5
Slide 25: 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 Susan O’Connor Chief Human Resources Officer Christine Bellon PhD, JD Chief Legal Officer T
Slide 26: Thank you
Thank you
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