Beam Therapeutics Inc. · NASDAQ:BEAM
Beam Therapeutics — Q3 2024 Earnings Presentation
38 slides from Beam Therapeutics's earnings presentation, Nov 5, 2024
Slide 1: Q3 Financial Results & ASH Abstracts Investor Webcast
Q3 Financial Results & ASH Abstracts Investor Webcast November 5, 2024
Slide 2: Beam conference call participants
Beam conference call participants Beam’s Sickle Cell Disease Strategy Q3 Business Update Introduction ASH Abstract: ESCAPE Preclinical Data ASH Abstracts: BEAM-101 Clinical Data Mr. Evans John Evans Chief Executive Officer Holly Manning VP Investor Relations & External Communications Giuseppe Ciaram
Slide 3: 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 therapeutic applications and potential of our techn
Slide 4: 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 5: The central hypothesis behind Beam: base editing vs nucleases
The central hypothesis behind Beam: Base editing is more precise, efficient, predictable and versatile than nucleases 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
Slide 6: Two platforms with potential to create transformative therapies
Two platforms with potential to create transformative therapies and significant value creation Hematology Liver Genetic Diseases Best-in-class potential for BEAM-101 for sickle cell disease (SCD) Increased probability of technical success for ex vivo gene editing and fetal hemoglobin (HbF) upregulat
Slide 7: Significant execution momentum in Q3
Significant execution momentum in Q3 across our priority hematology and liver genetic disease programs Hematology Liver Genetic Diseases First cohort dosing completed in the Phase 1/2 trial of BEAM-302 in AATD IND approved by FDA and site activation activities underway for BEAM-301 Phase 1/2 study i
Slide 8: Four Beam abstracts accepted for presentation at ASH
ORAL Initial Results from the BEACON Clinical Study of BEAM-101 in Sickle Cell Disease Sunday, Dec. 8, 10 a.m. PT Abstract #513 ORAL Preclinical Data for ESCAPE in a Rhesus Autologous Transplantation Model Sunday, Dec. 8, 10:45 a.m. PT Abstract #516 POSTER Initial Data from the Phase 1/2 Study of BE
Slide 9: Better one-time therapies for people living with SCD
What if we could develop better one-time therapies for people living with SCD? SICKLE CELL DISEASE
Slide 10: Beam's multi-wave strategy in 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 WAVE 2 30-40,000 WAVE 1 ~10,000 ESCAPE: Multiple edits for non-genotoxic conditioning Designed to eliminate chemotherapy from ex vivo gene therapy and expand
Slide 11: Beam's multi-wave strategy in 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 WAVE 2 30-40,000 ESCAPE: Multiple edits for non-genotoxic conditioning Designed to eliminate chemotherapy from ex vivo gene therapy and expand patient popula
Slide 12: BEAM-101: Designed to be best-in-class genetic medicine for SCD
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 SCD Unmet Need Sickle hemoglobin (HbS) polymerization is root
Slide 13: 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 14: Precise single-base editing results in highest editing efficiency in preclinical models
Precise, single-base editing without need for double-stranded breaks or viral insertion results in highest editing efficiency in preclinical models Preclinical data suggest BEAM-101 has potential for highest HbF induction and lowest residual HbS levels vs. other approaches Preclinical data presented
Slide 15: BEAM-101 Phase 1/2 Clinical Data
BEAM-101 Phase 1 Clinical Data AMY SIMON, M.D., CHIEF MEDICAL OFFICER
Slide 16: BEACON Phase 1/2 study design
BEACON Phase 1/2 study: Evaluating safety and efficacy of BEAM-101 in patients with SCD and severe vaso-occlusive crises 18-35 years age SCD (βS/βS; βS/β0; βS/β+ genotypes) with ≥4 severe vaso-occlusive crises (sVOCs) in 24 mos pre-screening No available matched sibling donor No history of overt str
Slide 17: Patient demographics and treatment characteristics
Patient demographics and treatment characteristics PATIENT DEMOGRAPHICS (N=6) Genotype, n βS / βS 5 βS / β0 1 Gender, n Male 3 Female 3 Age in years Range 19-27 Race African American 6 TREATMENT CHARACTERISTICS (N=6) Mobilization cycles 1 cycle 3 2 cycles 3 BEAM-101 dose, viable CD34+ cells x 106/kg
Slide 18: Preliminary safety data (N=6)
Preliminary safety data (N=6) Initial safety profile consistent with myeloablative conditioning with busulfan and autologous hematopoietic stem cell transplant (HSCT) One patient died due to respiratory failure likely related to busulfan conditioning 4 months after BEAM-101 infusion Unrelated to BEA
Slide 19: Preliminary safety data: Time to engraftment
Preliminary safety data: Time to engraftment (N=4*) Neutrophil Engraftment N=4 Platelet Engraftment N=4 Study Days after Drug Infusion Study Days after Drug Infusion 0 5 10 15 20 25 30 35 40 17 BEAM-101 Individual Patients BEAM-101 Median (Range) 20 BEAM-101 Individual Patients BEAM-101 Median (Rang
Slide 20: Preliminary efficacy data (N=4)
%HbF** Preliminary efficacy data (N=4*) 4% 72% 65% 9% 74% 17% 71% PATIENT 1 PATIENT 2 PATIENT 3 PATIENT 4 HbF HbS HbA Other Last RBC Transfusion: Day 7 Last RBC Transfusion: Day 7 Last RBC Transfusion: Day 17 Total Hemoglobin (g/dL) Last RBC Transfusion: Day 22 **%HbF / (F + S) Normal range of total
Slide 21: Preliminary efficacy data (N=4)
Preliminary efficacy data (N=4*) All patients achieved >60% HbF and <40% HbS of non-transfused hemoglobin at Month 1, sustained through all time points Markers of hemolysis (lactate dehydrogenase, indirect bilirubin, haptoglobin and reticulocyte counts) normalized or improved for all patients No VOC
Slide 22: Exploratory red blood cell function assays
Exploratory red blood cell (RBC) function assays After BEAM-101 treatment: Nearly all RBCs were expressing HbF by Month 1 Nearly all RBCs expressing solely HbS were eliminated by Month 2 Data cut as of July 2, 2024 Duplex flow cytometry performed on untransfused RBCs
Slide 23: Exploratory red blood cell function assays
Exploratory red blood cell (RBC) function assays Sickle Trait Average* Rate Sickling %/min * Median of maximum sickling observed from a ‘n’ of 10 sickle trait samples tested in a reference range study Reduction in RBC sickling (see graph) and cell adhesion to levels comparable to SCT samples (N=2) O
Slide 24: Summary: Emerging BEACON data show potential for differentiation
Summary: Emerging BEACON data show potential for significant differentiation of base editing and BEAM-101 for SCD Neutrophil engraftment following busulfan conditioning occurred <20 days Potential to enable fewer hospital days Initial biomarker data demonstrate near elimination of RBCs solely expres
Slide 25: ESCAPE Program Non-human Primate Data
ESCAPE Program Non-human Primate Data GIUSEPPE CIARAMELLA, PH.D., PRESIDENT
Slide 26: Beam's multi-wave strategy in 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 WAVE 2 30-40,000 WAVE 1 ~10,000 ESCAPE: Multiple edits for non-genotoxic conditioning Designed to eliminate chemotherapy from ex vivo gene therapy and expand
Slide 27: Beam's multi-wave strategy in 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 WAVE 2 30-40,000 ESCAPE: Multiple edits for non-genotoxic conditioning Designed to eliminate chemotherapy from ex vivo gene therapy and expand patient popula
Slide 28: ESCAPE designed 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 29: ESCAPE designed for selective depletion of diseased cells
ESCAPE technology designed for selective depletion of diseased cells to enable non-genotoxic conditioning Enrichment of edited cells in presence of antibody Paired CD117 antibody (ng/mL) Colony ratio (%) Old diseased cells are suppressed and eliminated by BEAM-103, creating space for the graft New e
Slide 30: Assessing ESCAPE in a non-human primate model
Assessing ESCAPE in a rigorous non-human primate (NHP) model of autologous transplant Proof-of-concept of non-genotoxic antibody conditioning Can engraftment of HSCs be achieved without chemotherapy? Can a therapeutic level of fetal hemoglobin induction be achieved? Study design: Complete ex vivo ge
Slide 31: Long-term engraftment of base edited CD34 cells in NHPs
Long-term engraftment of base edited CD34 cells after antibody conditioning in NHPs led to high % F cells and HbF % Proof of concept of long-term engraftment after antibody conditioning, without chemotherapy, in NHPs % F-cells and HbF % at therapeutic thresholds comparable to gene therapy after 3-6
Slide 32: Anticipated next steps for ESCAPE
Follow-up NHP studies to optimize antibody dose regimen, dose response and chimerism Additional data expected in 2025 GMP manufacturing initiated Initiate Phase 1-enabling tox studies by YE 2024 Phase 1 study of BEAM-103 antibody to evaluate PK/PD and safety in heathy volunteers Efficacy study of BE
Slide 33: 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 34: Significant step forward for Beam's hematology vision
Significant step forward for Beam’s hematology vision and base editing platform Potentially differentiated clinical profile, comparable to sickle cell trait Potential to eliminate chemotherapy from transplant and expand the SCD market Technology validated, with strong translation from preclinical to
Slide 35: BEAM-201 Phase 1/2 Clinical Data
BEAM-201 Phase 1 Clinical Data MULTIPLEX BASE EDITING FOR CELL THERAPY
Slide 36: BEAM-201 abstract highlights
BEAM-201 abstract highlights: Data demonstrate proof of concept of 1st quad-edited, allogeneic CAR-T cell therapy Early evidence of clinical efficacy with CAR-T cell doses <200 million, as measured by CRi/CR in 2/3 patients Pharmacokinetic (PK) and safety data support continued Phase 1 dose explorat
Slide 37: Significant catalysts on the horizon for Beam
Significant catalysts on the horizon for Beam Recent and Anticipated Catalysts BEAM-101 SCD Completed sentinel dosing and initiated expansion Present initial clinical data at ASH ESCAPE SCD & BETA-THALASSEMIA Initiate Phase 1-enabling preclinical studies in 2024 Present NHP preclinical data at ASH B
Slide 38: Q&A
VW A >, - Beam THERAPEUTICS Q&A