Beam Therapeutics Inc. · NASDAQ:BEAM
Beam Therapeutics — J.P. Morgan Healthcare Conference January 2024
26 slides from Beam Therapeutics's conference presentation, Jan 8, 2024
Slide 1: Precision Genetic Medicines Through Base Editing
PRECISION GENETIC MEDICINES THROUGH BASE EDITING JANUARY 2024 NASDAQ: BEAM
Slide 2: Cautionary note regarding forward-looking statements
Cautionary note regarding 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 preclin
Slide 3: Our vision is to provide life-long cures for patients
OUR VISION IS TO PROVIDE LIFE-LONG CURES for patients suffering from serious diseases POTENTIAL FOR one-time, curative therapies GENE EDITING FOR rare and common diseases PLATFORM FOR rapidly-programmable precision medicines
Slide 4: Base editing is an efficient, predictable gene editing technology
Base editing is an efficient, predictable and potentially best-in-class gene editing technology NUCLEASE CRISPR, ZFN, TALENs Double-stranded breaks Lack of control of gene sequence outcomes Precision targeting with CRISPR BASE EDITING BEAM THERAPEUTICS Enzymatic base conversion Highly efficient with
Slide 5: Base editing technology has multiple, highly versatile applications
Base editing technology has multiple, highly versatile applications CRISPR Protein Deaminase Guide RNA Activate expression Silence proteins Correct mutations Multiplex edits Modify proteins Turns on genes to restore or increase function Turns off any gene with disease-causing activity Repairs the mo
Slide 6: Comprehensive, fully-integrated platform for precision genetic medicines
We have built a comprehensive, fully-integrated platform for precision genetic medicines Guide RNA Design Production Base Editing A and C base editors Single and multiplex mRNA Design Production Cell Therapy Ex vivo HSC and T cells LNP In vivo Liver, HSCs and other tissues GMP Manufacturing External
Slide 7: Advancing a diversified pipeline into the clinic
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 (SCD) Ex vivo HSC Activation of fetal hemoglobin (HbF) ESCAPE Sickle Cell Disease Beta Thalassemia Ex vivo HSC Multiple
Slide 8: Highly differentiated priority programs with significant value creation potential
Highly differentiated priority programs with significant value creation potential Sickle Cell Disease Alpha-1 Antitrypsin Deficiency Best-in-class potential for BEAM-101 Increased probability of technical success for ex vivo gene editing and HbF upregulation Validated FDA regulatory pathway ESCAPE h
Slide 9: 2023 was a transformative year for CRISPR gene editing, base editing, and Beam
2023 was a transformative year for CRISPR gene editing, for base editing, and for Beam 2023 Highlights GENE EDITING First in vivo gene editing INDs cleared by FDA First in vivo liver base editing clinical data First CRISPR-based product approved for SCD BEAM First patients dosed with base edited the
Slide 10: 2024 is expected to be a year of significant catalysts for Beam
2024 is expected to be a year of significant catalysts for Beam 2024 Anticipated Catalysts BEAM-101 SCD Complete sentinel dosing and initiate expansion dosing in first half of 2024 Present clinical data on multiple patients in second half of 2024 ESCAPE SCD Initiate Phase 1-enabling preclinical stud
Slide 11: What if we could develop better one-time therapies for patients with SCD?
What if we could develop better one-time therapies for patients with SCD? SICKLE CELL DISEASE
Slide 12: Beam's multi-wave strategy for patients with SCD
Wave 3: ~100,000 Beam’s multi-wave strategy is focused on developing safer, more effective, and more accessible treatments for patients with SCD Eligible SCD Patient Population (U.S.) Wave 2: 30-40,000 Wave 1: ~10,000 Wave 2 ESCAPE: Multiplex HbF edit + CD117 selection edit Non-genotoxic conditionin
Slide 13: BEAM-101: Designed to be best-in-class genetic medicine for SCD
BEAM-101: Designed to be best-in-class genetic medicine for SCD BEAM-101 Potential SCD Patient HBB HBG1 HBG2 HBB HBG1 HBG2 Mutated hemoglobin gene (HBB) Direct reactivation of HbF genes Hemoglobin genes SCD Unmet Need Sickle cell hemoglobin (HbS) polymerization is root cause of sickle cell pathophys
Slide 14: Precise, single base editing results in highest editing efficiency in pre-clinical models
Precise, single base editing without need for double-stranded breaks or viral insertion results in highest editing efficiency in pre-clinical models BEAM-101: Potential for highest HbF induction and lowest residual HbS levels versus other approaches in the field Preclinical data presented at ASGCT 2
Slide 15: Anticipate presenting clinical data on multiple patients in second half of 2024
Anticipate presenting clinical data on multiple patients in the second half of 2024 BEAM-101: First clinical base editing program in the U.S., accelerating path to SCD patients and the market BEACON Phase 1/2 Study Design Select safety endpoints Proportion of patients with successful neutrophil engr
Slide 16: Plan to initiate Phase 1-enabling studies in 2024
Plan to initiate Phase 1-enabling studies in 2024 Wave 2 ESCAPE: Designed for selective depletion of diseased cells to enable non-genotoxic conditioning for SCD Stem cell factor (SCF) signaling via CD117 required for HSC survival and proliferation Single base edit changes the epitope on CD117 recept
Slide 17: Developing LNPs for delivery of base editors to blood stem cells
Wave 3 in vivo: Developing LNPs for delivery of base editors to blood stem cells Presented at ASH 2021 Expression of mRNA payload in NHP HSCs at clinically relevant doses In preclinical studies, Beam LNP technology allowed targeting of blood stem cells for delivery of mRNA payloads at clinically rel
Slide 18: What if we could use base editing to correct disease-causing mutations in vivo?
What if we could use base editing to correct disease-causing mutations in vivo? GENETIC DISEASES
Slide 19: BEAM-302: Aims to restore expression of functional AAT
BEAM-302: Aims to restore expression of functional AAT to address AATD-related lung and liver disease AATD Unmet Need PiZZ genotype is >95% of severe AATD population that typically develop progressive lung and/or liver disease 100,000 PiZZ individuals in the U.S.; ~10% diagnosed Current Available Tr
Slide 20: BEAM-302: Corrected the PiZ mutation and restored functional AAT in mouse model
BEAM-302: Corrected the PiZ mutation and restored functional AAT with a single dose in AATD mouse model Liver editing correlated with increased corrected serum AAT and decreased mutant PiZ AAT, at or below 0.75mpk Liver Editing Serum AAT Levels
Slide 21: BEAM-302: Phase 1/2 trial designed to achieve clinical proof-of-concept
BEAM-302: Phase 1/2 trial designed to achieve clinical proof-of-concept in patients across the spectrum of AATD Opportunity to achieve first ever clinical proof-of-concept of in vivo base editing leading to correction of a disease-causal mutation CTA submitted to UK; additional filings to follow Par
Slide 22: BEAM-301: Aims to normalize glycogen metabolism in patients with GSD1a
Chou & Mansfield. 2007. Curr. Gen. Ther.; Internal Beam estimates Wildtype G6PC gene G6PC R83C mutation BEAM-301: Aims to normalize glycogen metabolism in patients with GSD1a to prevent hypoglycemia and other disease manifestations Liver Unmet Need in GSD1a Patients with Severe R83C Mutation: Inabil
Slide 23: BEAM-301: Single dose significantly improved long-term survival in GSD1a mouse model
U.S. IND filing expected in first half 2024 BEAM-301: Treatment with a single dose significantly improved long-term survival in GSD1a mouse model Preclinical studies of BEAM-301 demonstrated a single dose significantly improved long-term survival out to a year in humanized R83C homozygous mice Untre
Slide 24: Pipeline and platform partnerships unlock additional value
Creative pipeline and platform partnerships unlock additional value and broaden therapeutic impact Strategic Deals resulting in $675M upfront and more than $1B in potential milestones Innovator Deals gaining rights to innovative and complementary technologies $300M upfront for 3 base editing targets
Slide 25: 2024 is expected to be a year of significant catalysts for Beam
2024 is expected to be a year of significant catalysts for Beam 2024 Anticipated Catalysts BEAM-101 SCD Complete sentinel dosing and initiate expansion dosing in first half of 2024 Present clinical data on multiple patients in second half of 2024 ESCAPE SCD Initiate Phase 1-enabling preclinical stud
Slide 26: Thank You
THANK YOU J.P. MORGAN HEALTHCARE CONFERENCE JANUARY 2024
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