Beam Therapeutics Inc. · NASDAQ:BEAM
Beam Therapeutics — Corporate Update (January 2022)
26 slides from Beam Therapeutics's corporate update, Jan 10, 2022
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: Coming era of one-time, curative therapies
Coming era of 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 3
Slide 4: Base editing is a next-generation approach to gene editing with single base precision
Nuclease editing Base editing Yes (guide RNA or ZF/TALE) Yes Random insertions and deletions Base editing is a next-generation approach to gene editing with single base precision Yes (guide RNA) No Predictable single base changes CRISPR, Zinc Fingers, TALEs 4 Precise targeting? Double strand breaks?
Slide 5: Single base DNA variants drive health outcomes
Single base DNA variants drive health outcomes Common disease Single base changes drive risk and protection from common diseases Rare disease Over half of genetic disease mutations are point mutations 5
Slide 6: Base editing is a highly-differentiated, potentially best-in-class gene editing technology
Base editing is a highly-differentiated, potentially best-in-class gene editing technology 6 Biology B Application A Specificity S Efficiency E Gene correction, activation, silencing, modification Simultaneous “multiplex” editing at many sites Highly specific and predictable editing profile Avoid ge
Slide 7: We are establishing a leading platform for precision genetic medicine
We are establishing a leading platform for precision genetic medicine Payload Manufacturing Delivery 7 Suite of gene editing technologies Base editing Nuclease editing RNA editing Prime editing Suite of delivery technologies Autologous cell therapy Allogeneic cell therapy mRNA LNP vectors Viral vect
Slide 8: Strategic collaboration with Pfizer for in vivo base editing
$300M upfront $1B+ in potential milestones 4-year research term; Pfizer option at DC nomination 3 targets, not included in Beam’s current programs Leverages Beam delivery technologies to target liver, muscle, CNS Beam option at end of P1/2 for 35% WW cost/net profit split on any one program Strategi
Slide 9: Additional strategic collaborations broaden therapeutic opportunities
Additional strategic collaborations broaden therapeutic opportunities and unlock value in Beam platform 9 Base editing for the prevention of cardiovascular disease Beam opt-in to 50% of US rights after Phase 1 Base editing for the treatment of complement mediated diseases $75M in upfront and near-te
Slide 10: Diversified portfolio of base editing programs
Diversified portfolio of base editing programs LNP = Lipid Nanoparticle; AAV = Adeno Associated Virus; HSC = Hematopoietic Stem Cell; ALL = Acute Lymphoblastic Leukemia; AML = Acute Myeloid Leukemia DELIVERY PROGRAM / DISEASE EDITING APPROACH RESEARCH LEAD OPTIMIZATION IND ENABLING PHASE I/II PIVOTA
Slide 11: Key progress and anticipated milestones
✓ Key progress and anticipated milestones 2021 Achievements 2022 Milestones ✓ FDA clearance of the BEAM-101 IND ✓ Beam LNP data in non-human primates ✓ First liver DC, BEAM-301: GSDIa R83C ✓ IND-enabling studies for BEAM-102 ✓ IND-enabling studies for BEAM-201 ✓ Non-human primate studies for Stargar
Slide 12: Autologous ex vivo cell process for editing hematopoietic stem cells
Autologous ex vivo cell process for editing hematopoietic stem cells Collect cells Electroporate Infuse cell product Condition patient Sickle Cell Disease Patient Sickle Cell Disease Patient 3 4 1 2 12 Program-specific gRNA and base editor mRNA
Slide 13: BEAM-101: High levels of editing and robust HbF induction
BEAM-101: High levels of editing and robust HbF induction after long-term in vivo engraftment >90% base editing at HBG1/2 promoters in multilineage cells1 >65% gamma globin protein levels in sorted erythroid cells2 13 Sickle Cell Disease: 100,000 patients in the US; severe pain, organ damage, early
Slide 14: BEAM-101 is the first clinical base editing program
BEAM-101 is the first clinical base editing program Safety endpoints Proportion of patients with successful neutrophil engraftment by day 42 Safety and tolerability assessments Efficacy endpoints Severe vaso-occlusive events Transfusion requirements Hemoglobin F levels Quality of life and ability to
Slide 15: BEAM-102: High editing of sickle mutation eliminating HbS globin
BEAM-102: High editing of sickle mutation led to significant elimination of HbS globin in patient donor cells 15 Makassar Globin (HbG) Sickle Globin (HbS) Elimination of sickling under low oxygen conditions >80% editing of HbS mutation Sickle Cell Disease: 100,000 patients in the US; severe pain, or
Slide 16: Best-in-class regimens for SCD patients, now and in the future
Uniquely positioned to potentially create best-in-class regimens for SCD patients, now and in the future Precise gene editing (non-cutting, non-viral) BEAM-101 BEAM-102 HSC conditioning with targeted antibodies (less toxic) In vivo editing after infusion of HSC-targeted LNPs (no transplant) 16 Wave
Slide 17: Developing LNPs for delivery of mRNA to HSPCs
Developing LNPs for the delivery of mRNA to Hematopoietic Stem & Progenitor Cells (HSPCs) Proprietary technology for high throughput LNP screening for delivery beyond the liver Each nanoparticle contains mRNA payload plus a unique DNA barcode Simultaneous in vivo screening of LNPs to select formulat
Slide 18: Allogeneic multiplex edited CAR-T cell process
Allogeneic multiplex edited CAR-T cell process Collect T-cells Electroporate and transduce Lymphodeplete and infuse Expand and freeze Healthy donor Multiple cancer patients Editor mRNA gRNA #1 gRNA #2 gRNA #3 gRNA #4 Lentiviral CAR 3 4 1 2 18
Slide 19: Advantages of multiplex base editing without double strand breaks
Significant advantages of multiplex base editing without double strand breaks DNA damage response to editing (apoptosis and p53 pathway) Chromosomal rearrangements Impact on cell expansion 4 edits: TRAC, CD52, PD1, CD7 3 edits: TRAC, B2M, PD1 4 edits: TRAC, CD52, PD1, CD7 19 Base editing Nuclease 40
Slide 20: BEAM-201: High level of cell engineering via multiplex base editing
BEAM-201: High level of cell engineering enabled by simultaneous multiplex base editing of four genes 20 Clinical process yields 96-99% editing, >90% quad edited1 Potent in vivo tumor clearance or control across a 25-fold dose2 Treatment Randomization Unedited BEAM-201 T-Cell Acute Leukemia: 15% of
Slide 21: Clinically validated technology for in vivo delivery to the liver
Clinically validated technology for transient, in vivo delivery to the liver Scalable manufacturing with lower COGS Proprietary Beam formulation showed up to 60% editing in NHPs at clinically-relevant dose of 1.0 mpk Non-viral delivery for in vivo base editing in liver 21 Infuse Formulate LNP Lipid
Slide 22: BEAM-301: ABE correction of GSDIa R83C mutation
BEAM-301: ABE correction of GSDIa R83C mutation associated with improved survival of R83C mice DC nomination in Dec 2021 – Beam’s first in vivo DC Near-normal serum metabolites, G6PC activity, hepatic morphology and lipid deposition Animal models suggest 11% editing may be sufficient for clinical be
Slide 23: In vivo direct correction of A1AT mutation with base editing
In vivo direct correction of A1AT mutation with base editing designed to address liver and lung disease 4.9-fold increase in functional A1AT secretion Reduction in toxic liver aggregates Control Correction Group 23 Alpha-1 Anti-trypsin Deficiency: 60,000 ZZ patients in US1; severe progressive lung a
Slide 24: Multiplex base editing of hepatitis B virus genome
Current antivirals do not eliminate the HBV genome, leading to viral rebound and preventing cure Multiplex base editing has potential to silence covalently closed circular DNA (cccDNA) Base editing also has potential to silence HBV integrated in human genome, without fear of chromosomal rearrangemen
Slide 25: Meet the Beam Team
Meet the Beam Team Significant team track record in discovery, development, approval of first-in-class medicines 25 John Evans Chief Executive Officer Giuseppe Ciaramella, PhD President, Chief Scientific Officer Courtney Wallace Chief Business Officer Susan O’Connor Chief Human Resources Officer Chr
Slide 26: Thank you
Thank you Beam