Precision Biosciences, Inc. · NASDAQ:DTIL
Precision Biosciences — Investor Presentation (April 2020)
28 slides from Precision Biosciences's investor presentation, April 2020
Slide 1: Precision BioSciences - April 2020
PRECISION BIOSCIENCES ts Dedicated to Improving Life. April 2020 Cure genetic disease. Feed the planet.
Slide 2: Forward Looking Statements
Forward Looking Statements This presentation (together with any other statements or information that we may make in connection herewith) may contain forward-looking statements. All statements other than statements of present and historical facts contained in this prospectus, including without limita
Slide 3: Delivering on the Promise of Genome Editing
Delivering on the Promise of Genome Editing ARCUS genome Clinical stage World class team editing platform built allogeneic CAR T that includes the for translation with full platform with pioneers in freedom to operate validating initial genome editing safety and response data Scaled in-house In vivo
Slide 4: ARCUS: Precision's Proprietary Gene Editing Platform
ARCUS: Precision’s Proprietary Gene Editing Platform Key Advantages I-CreI ARCUS • Safety: minimizes off-target editing; natural “off switch” • Ease of delivery: Precision bioengineering small size permits both LNP and AAV delivery • Control of edits: efficient knock in or knock out Nature’s editing
Slide 5: Efficient, Scaled In-house Manufacturing Already Established
Efficient, Scaled In-house Manufacturing Already Established Ability to produce ARCUS-based CAR T and in vivo therapies MCAT: Manufacturing Center for Advanced Therapeutics 17,300 sq. ft. facility in Durham, NC Fully cGMP compliant Operational July 2019 Currently producing clinical trial material fo
Slide 6: Overcoming Cancer: Off-the-Shelf CAR T
Overcoming Cancer Off-the-Shelf CAR T 6
Slide 7: The Allogeneic CAR T Opportunity
The Allogeneic CAR T Opportunity Autologous Allogeneic CAR T CAR T • Early evidence of encouraging • High rates of efficacy in some cancers clinical efficacy • Can be effective where other options have failed • Potentially widely available and lower cost • High cost and challenging logistics leading
Slide 8: Four Key Requirements for Allogeneic CAR T Success
Four Key Requirements for Allogeneic CAR T Success 1 Scaled Manufacturing • Efficiently deliver 2 Optimal T Cell Phenotype • Healthy cell product 3 Improved Safety • True off-the-shelf 4 Clinical Activity • Demonstrated consistent, high safety profile efficacy quality cell product • High percentage
Slide 9: Key Features of Precision's Allogeneic CAR T Platform
Key Features of Precision’s Allogeneic CAR T Platform Single-step ARCUS editing - minimizes off-targeting - CAR directly inserted into TCR locus - helps preserve phenotype Optimized T cell CAR expressed from healthy with proprietary donor N6 co-stimulatory Short, 10-day domain manufacturing TCR TCR
Slide 10: Platform Is Delivering Against All Four Key Requirements
Platform Is Delivering Against All Four Key Requirements 1 Scaled Manufacturing 2 Optimal T Cell Phenotype % CAR+ Cells 3 Safety Profile NHL (n=6) ALL (n=3) 4 Clinical Activity 67% ORR Final Yield 40% GvHD in NHL CD19 Drug Candidate DL1 0% 0% (64M CAR T cells/vial) 30% DL2 0% 0% Batch Vial Count CRS
Slide 11: Precision's Off-the-Shelf CAR T Immunotherapy Pipeline
Precision’s Off-the-Shelf CAR T Immunotherapy Pipeline Product Candidates Program Area Discovery Pre-clinical Clinical Rights PBCAR0191 (CD19) NHL and ALL – Phase 1 Data Expected 2020 PBCAR20A (CD20) NHL, CLL, SLL – First Patient Dosed April 2020 PBCAR269A (BCMA) MM - IND cleared – Phase 1 Dosing Ex
Slide 12: PBCAR0191 Phase 1/2a: NHL and B-ALL
PBCAR0191 Phase 1/2a: NHL and B-ALL Safety Assessment Screening Treatment Period Follow-Up LTFU Study Day -14 -7 -5 -4 -3 0 28 60 90 180 360 PBCAR0191 Safety & Response Enrollment Infusion x1 End of Study Assessment Lymphodepletion Population • Adult patients with R/R B-NHL or R/R B-ALL Lymphodeplet
Slide 13: Adverse Events Compare Favorably to Autologous CAR T
Adverse Events Compare Favorably to Autologous CAR T System Organ Class NHL B-ALL Preferred Term, n(%) (n=6) (n=3) CRS (Cytokine Release Syndrome) – 2 (33%) 1 (33%) Grade 1 or Grade 2 ICANS (Immune Effector Cell Neurotoxicity) – 0 (0%) 1 (33%) Grade 1 or Grade 2 CRS Grade 3 or higher 0 (0%) 0 (0%) I
Slide 14: Initial Data Support Dose Dependent Clinical Activity
Initial Data Support Dose Dependent Clinical Activity NHL Cohort Data Best response day 28+ CAR T cell expansion # patients # patients with positive expansion (maximum days positive) 67% 1/3 ORR pts* 1 Complete response 3/3 pts 2 1 (Day 60†) Partial Partial 2/3 pts 2/3 pts responses response (Day 3*
Slide 15: PBCAR0191 Next Steps – Core Aim to Explore Higher Doses
PBCAR0191 Next Steps – Core Aim to Explore Higher Doses DL1 DL2 DL3 DL4 DL5 3x105 / kg 1x106 / kg 3x106 / kg 6x106 / kg 9x106 / kg Administration Single dose Single dose Single dose 2 doses 3 doses 3x105 cells/kg/dose 1x106 cells/kg/dose 3x106 cells/kg/dose 3x106 cells/kg/dose 3x106 cells/kg/dose Si
Slide 16: PBCAR20A and PBCAR269A Allogeneic CAR T Programs
PBCAR20A and PBCAR269A Allogeneic CAR T Programs Safety Assessment Screening Treatment Period Follow-Up LTFU Study Objectives Day -14 -7 -5 -4 -3 0 28 60 90 180 360 • Primary: safety and tolerability CAR T Safety & Response • Secondary: clinical (anti-tumor) Enrollment End of Study Infusion x1 Asses
Slide 17: Off-the-Shelf CAR T: The Best of Both Worlds
Off-the-Shelf CAR T: The Best of Both Worlds Off-The-Shelf Biologics CAR T First Line Last Line • Safety profile • Accessibility • Efficacy profile PBCAR Liquid tumors • Safety profile • Accessibility Future portfolio in solid tumors • Efficacy profile 17
Slide 18: Curing Genetic Disease: In Vivo Gene Correction
Curing Genetic Disease In Vivo Gene Correction 18
Slide 19: Four Key Requirements for Successful In Vivo Gene Correction
Four Key Requirements for Successful In Vivo Gene Correction 1 Safety • Minimize off-target gene editing 2 Ease of Delivery • Delivery vectors have limited capacity 3 Efficient, Versatile Editing • Knock in and/or repair of gene 4 Editing Drives Therapeutic Effect • High efficiency editing correspon
Slide 20: ARCUS Platform Delivers Against All Four Key Requirements
ARCUS Platform Delivers Against All Four Key Requirements 1 Safety PCSK9 ARCUS - NHP off-targeting assessment 2 Ease of Delivery 3 Efficient, Versatile Editing Frequency of ARCUS PCSK9 liver 60% editing events in NHP 4 Editing Drives Therapeutic Effect ~90% reduction in serum PCSK9 levels in NHPs 10
Slide 21: In Vivo Gene Correction Pipeline
In Vivo Gene Correction Pipeline Product Candidate Program Area Discovery Pre-clinical Clinical Rights HBV Chronic Hepatitis B – IND 2021 HAO1 Primary hyperoxaluria type 1 (PH1) Transthyretin Familial amyloid polyneuropathy ApoC3 Lipoprotein lipase deficiency PCSK9 Familial hypercholesterolemia PH1
Slide 22: Overview of Primary Hyperoxaluria Type 1 (PH1)
Overview of Primary Hyperoxaluria Type 1 (PH1) Primary Hyperoxaluria – key facts Prevalence of Rare genetic 1-3/1,000,000 Combined disease characterized ~40% Affects liver-kidney by accumulation of patients have adults transplant end-stage renal calcium oxalate in disease at the time and young child
Slide 23: Effective, Efficient ARCUS Editing Underpins Advancement of PH1
Effective, Efficient ARCUS Editing Underpins Advancement of PH1 ARCUS efficiently knocked-out the HAO1 gene ARCUS treatment resulted in ~70% reduction in non-human primates following AAV8 delivery in urine oxalate in a PH1 mouse model Non-human primate (whole liver) Mouse model 35 Buffer only Low do
Slide 24: Feed the Planet: Elo Life Systems
Feed the Planet Elo Life Systems 24
Slide 25: Elo is Focused on Delivering Healthy and Sustainable Food Sources
Elo is Focused on Delivering Healthy and Sustainable Food Sources Food companies need new inputs to respond to: A Human • Climate change Health • Consumer preference Opportunity Elo integrates ARCUS with enabling technologies to create greatly needed improvements to sources of food • Partner driven
Slide 26: Multiple Upcoming Milestones Expected Across Portfolio in 2020
Multiple Upcoming Milestones Expected Across Portfolio in 2020 IND accepted for BCMA CAR T Initiate dosing for CD20 CAR T Initiate dosing for BCMA CAR T PH1 candidate selection NHL (CD19) clinical data update ALL (CD19) clinical data update 26
Slide 27: Key Takeaways
Key Takeaways Highly experienced team includes the pioneers in editing Proprietary ARCUS editing platform confers fundamental advantages Independent cGMP manufacturing capabilities Early allogeneic CAR T clinical data validate core strategy In vivo programs to address significant unmet medical needs
Slide 28: Dedicated To Improving Life
Dedicated To Improving Life 28
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