Beam Therapeutics Inc. · NASDAQ:BEAM
Beam Therapeutics — Corporate Update (January 2025)
39 slides from Beam Therapeutics's corporate update, Jan 13, 2025
Slide 1: Precision Genetic Medicines Through Base Editing
PRECISION GENETIC MEDICINES THROUGH BASE EDITING NASDAQ: BEAM January 2025
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
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 …A C G - - - - G C A T… …A C G T C G C T T A T G C A T… …A - - - - - T G C A T
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 gene with disease-causing activity Repairs the most c
Slide 6: Key elements of Beam's leadership position in gene editing
Key elements of Beam’s leadership position in gene editing BASE EDITING PLATFORM Clinical Validation, Fully Integrated Manufacturing MULTIPLE CATALYSTS Expected in 2025 RAPID EXECUTION for Clinical Programs HIGH VALUE FRANCHISES with Best-in-Class Potential $850.7M in cash estimated as of December 3
Slide 7: A comprehensive, fully integrated base editing platform
A comprehensive, fully integrated base editing platform BASE EDITING PLATFORM Clinical Validation, Fully Integrated Manufacturing FULLY INTEGRATED CAPABILITIES GENE EDITING AND DELIVERY TECHNOLOGIES Base editing ex vivo cell therapy in vivo LNP INTERNAL GMP MANUFACTURING FACILITY Research Triangle,
Slide 8: Strategic focus areas with potential for significant value creation
Strategic focus areas with potential for significant value creation Best-in-class potential for BEAM-101 in sickle cell disease (SCD) ASH 2024 data Well-established FDA path to BLA Lifecycle strategy with ESCAPE and in vivo editing Platform for future hematology pipeline Best-in-class potential fo
Slide 9: Proven ability to execute on key R&D priorities
Proven ability to execute on key R&D priorities 40+ patients enrolled, 20+ doses manufactured and 13 patients dosed* with BEAM-101 in 1 year Opening of BEACON adolescent cohort approved by FDA First in vivo CTA clearances achieved (BEAM-302) with enrollment on track and sites active in UK, NZ, Austr
Slide 10: Significant potential catalysts on the horizon
Significant potential catalysts on the horizon BEAM-101 SCD (BEACON TRIAL) Dose 30 patients by mid-2025 Enroll and dose adolescent patients Present updated data in mid-2025 BEAM-302 AATD Present initial data on multiple cohorts in 1H 2025 ESCAPE SCD & BETA-THALASSEMIA Initiate Phase 1 healthy volunt
Slide 11: Advancing a diversified pipeline
Advancing a diversified pipeline PROGRAM / DISEASE or TARGET 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 (BEAM-103 & BEAM-104) SCD Beta-thalassemia Ex vivo HSC Multiple
Slide 12: 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 13: Multi-wave strategy for safer, more effective, more accessible SCD treatments
WAVE 3 ~100,000 WAVE 2 30-40,000 WAVE 1 ~10,000 Multi-wave strategy is focused on developing safer, more effective and more accessible treatments for patients with SCD ESCAPE: Multiple edits for non-genotoxic conditioning Designed to eliminate chemotherapy from ex vivo gene therapy and expand patien
Slide 14: Significant market potential for SCD gene therapy
WAVE 3 ~100,000 WAVE 2 30-40,000 Significant market potential for SCD gene therapy ESCAPE: Multiple edits for non-genotoxic conditioning Designed to eliminate chemotherapy from ex vivo gene therapy and expand patient population with: Broader range of disease severity Broader age range Increased will
Slide 15: BEAM-101 designed to be best-in-class genetic medicine for sickle cell disease
BEAM-101 designed to be best-in-class genetic medicine for sickle cell disease BEAM-101 Potential SCD Patient HBB HBG1 HBG2 HBB HBG1 HBG2 Mutated hemoglobin gene (HBB) Direct reactivation of fetal hemoglobin (HbF) genes Hemoglobin genes Hemoglobin expression Expression of sickle-causing hemoglobin H
Slide 16: What would an ideal outcome for BEAM-101 look like?
What would an ideal outcome for BEAM-101 look like? Sickle cell “trait” (SCT) (carrier with one mutation, typically asymptomatic) 60% HbA 40% HbS No circulating cells with HbSS genotype Normal (no mutations) 100% HbA 0% HbS No circulating cells with HbSS genotype Sickle cell disease (two mutations)
Slide 17: BEACON Phase 1/2 study rapidly advancing
BEACON Phase 1/2 study rapidly advancing Phase 1/2, non-randomized, open-label, single-arm, multicenter, safety and efficacy study of the administration of BEAM-101 to patients with SCD (NCT05456880) DMC, Data Monitoring Committee; HbF, fetal hemoglobin; HbS, sickle hemoglobin; RBC, red blood cell;
Slide 18: Emerging BEACON data show potential for significant differentiation
Emerging BEACON data show potential for significant differentiation of base editing and BEAM-101 for SCD Neutrophil engraftment following busulfan occurred <20 days Potential for fewer hospital days Improved red blood cell health and function after BEAM-101 Sickling parameters decreased to levels co
Slide 19: BEAM-101 treatment process aims to minimize time in hospital
Neutropenic Days Outpatient follow up (up to 24 months on study, with up to 13 years of additional follow up in LTFU study) Engraftment Dosing BEAM-101 and its treatment process aim to minimize mobilization and engraftment burden to reduce time in hospital Data cutoff Oct 28, 2024; Presented at ASH
Slide 20: Rapid and robust HbF induction, HbS reduction, and resolution of anemia
Patients treated with BEAM-101 achieved rapid and robust HbF induction, HbS reduction, and resolution of anemia Data cutoff Oct 28, 2024; Presented at ASH 2024 Female total Hb LLN-ULN: 11.5-15 g/dL; Male LLN-ULN: 13-17 g/dL. Hb, hemoglobin; HbA, adult hemoglobin; HbF, fetal hemoglobin; HbS, sickle h
Slide 21: BEAM-101 initial safety data
BEAM-101 initial safety data are consistent with busulfan conditioning and autologous HSCT Data cutoff Oct 28, 2024; Presented at ASH 2024 Related events include events where investigator has assessed relationship as possibly or definitely related to BEAM-101 *Includes events that were ≥Grade 3 in a
Slide 22: ESCAPE technology to bring a paradigm shift to conditioning
ESCAPE technology designed to bring a paradigm shift to conditioning for the first time in nearly 70 years 1956 1st HSCT TBI conditioning AML 1959 Busulfan approved for CML 1984 1st SCD patient alloHSCT TBI/CP conditioning 1999 IV busulfan approval for HSCT conditioning 2014 1st Lovo-cel SCD patient
Slide 23: ESCAPE technology is comprised of two components
ESCAPE technology for hemoglobinopathies is comprised of two components: ESCAPE technology designed for selective suppression of diseased cells to enable non-genotoxic conditioning Old diseased cells are targeted and suppressed by BEAM-103, creating space for the graft New edited cells “escape” bind
Slide 24: NHP autologous transplant model for ESCAPE conditioning approach
NHP autologous transplant model for our ESCAPE conditioning approach Multiplex base-editing and erythroid differentiation of Rhesus CD34+ cells Infusion product was manufactured with priority for maximizing total CD34+ cell dose for transplant Next generation sequencing Multiplex editing in Rhesus H
Slide 25: Preclinical proof-of-concept for ESCAPE antibody conditioning in NHPs
Preclinical proof-of-concept for ESCAPE antibody conditioning and engraftment in NHPs without chemotherapy Rapid and complete replacement of erythroid cells by edited cells F-cell levels reached ~70-80% post-transplant γ-globin (proxy for HbF) reached ~50% in all animals mAb dosing was well tolerate
Slide 26: Anticipated next steps and pathway to patients for ESCAPE
Anticipated next steps and pathway to patients for ESCAPE Phase 1 study of BEAM-103 Ab to evaluate PK/PD and safety in heathy volunteers PLAN TO INITIATE BY YE 2025 Follow-up NHP studies to optimize antibody dose regimen, dose response, and chimerism IN PROCESS Efficacy study of BEAM-103 and BEAM-10
Slide 27: BEAM-101 progress derisks BEAM-104
BEAM-101 progress derisks BEAM-104 Potential to accelerate clinical trial, BLA filing, launch and commercial ramp Synergy between BEAM-101 and ESCAPE technology (BEAM-104 and BEAM-103) support efficient development in SCD BEAM-101 BEAM-104 Regulatory pathway Clinical sites + CD117 guide RNA Commerci
Slide 28: Wave in vivo: Developing LNPs for delivery to blood stem cells
Wave 3 in vivo: Developing LNPs for delivery of base editors to blood stem cells Presented at ASH 2021 In preclinical studies, Beam LNP technology allowed targeting of blood stem cells for delivery of mRNA payloads at clinically relevant doses Research to adapt system to base editing payloads is ong
Slide 29: 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 30: BEAM-302 for alpha-1 antitrypsin deficiency
PiZ is caused by a single G > A point mutation in the SERPINA1 gene PiZ AAT is poorly secreted and less effective than normal PiM protein PiZZ genotype is >95% of severe AATD population, typically resulting in progressive lung and/or liver disease 100,000 PiZZ individuals in U.S.; ~10% diagnosed One
Slide 31: BEAM-302 increased corrected AAT and decreased serum PiZ AAT
BEAM-302 resulted in both increased serum total & corrected AAT and decreased serum PiZ AAT in vivo NSG-PiZ Mouse Presented at Alpha-1 Antitrypsin Deficiency 2023 Meeting
Slide 32: Single dose of BEAM-302 led to durable correction of PiZ variant
Single dose of BEAM-302 led to durable correction of the PiZ variant in AATD mouse model Long term studies were performed with precursor research grade reagents (1.5mpk) Presented at Alpha-1 National Conference 2024 SERPINA1 gene editing and AAT levels were either stable or increasing after a single
Slide 33: Phase 1/2 trial of BEAM-302 across the spectrum of AATD
Phase 1/2 trial of BEAM-302 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 Global site activation and enrollment ongoing Part
Slide 34: BEAM-301 aims to normalize glycogen metabolism in GSD1a
Chou & Mansfield. 2007. Curr. Gen. Ther.; Internal Beam estimates BEAM-301 aims to normalize glycogen metabolism in GSD1a to prevent hypoglycemia and other disease manifestations Liver Wildtype G6PC gene G6PC R83C mutation Non-functional G6Pase Glycogen Glucose Glucose-6- phosphate Pyruvate Acetyl-C
Slide 35: BEAM-301 single dose improved long-term survival in GSD1a mouse model
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 Untreated homozygous R83C mice die within weeks o
Slide 36: BEAM-301 Phase 1/2 trial in GSD1a patients
BEAM-301 Phase 1/2 trial in GSD1a patients with R83C mutation designed to achieve early clinical proof-of-concept KEY ENDPOINTS Safety and tolerability Time to hypoglycemia during fasting Changes from baseline in starch supplementation Beam will initially focus development of BEAM-301 in the U.S. U.
Slide 37: 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 38: Key elements of Beam's leadership position in gene editing
Key elements of Beam’s leadership position in gene editing BASE EDITING PLATFORM Clinical Validation, Fully Integrated Manufacturing MULTIPLE CATALYSTS Expected in 2025 RAPID EXECUTION for Clinical Programs HIGH VALUE FRANCHISES with Best-in-Class Potential $850.7M in cash estimated as of December 3
Slide 39: Patients are at the heart of our vision
Kyle SICKLE CELL DISEASE Dan and Kathi ALPHA-1 ANTITRYPSIN DEFICIENCY Alyssa and Gayle GLYCOGEN STORAGE DISEASE 1A Patients are at the heart of our vision