Precision Biosciences, Inc. · NASDAQ:DTIL
Precision Biosciences — Investor Presentation (November 2020)
28 slides from Precision Biosciences's investor presentation, November 2020
Slide 1: Precision BioSciences Corporate Deck November 2020
PRECISION BIOSCIENCES C; Dedicated to Improving Life. November 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 presentation, including, without lim
Slide 3: Delivering on the Promise of Genome Editing
Delivering on the Promise of Genome Editing ARCUS genome editing Clinical stage World class team platform built for allogeneic CAR T that includes the translation with full platform with pioneers in freedom to operate validating initial genome editing safety and response data Scaled in-house cGMP ma
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 Q4 2020 PBCAR20A (CD20) NHL, CLL, SLL – First Patient Dosed April 2020 PBCAR269A (BCMA) MM - First Patient Dosed June 202
Slide 12: Allogeneic CAR T Study Designs
Allogeneic CAR T Study Designs Safety Assessment Screening Treatment and Follow-Up (365d) Lymphodepletion Flu/Cy Safety & Response Enrollment Assessment (Day 28) End of Study Population Dose Escalation Regulatory Designations Range PBCAR0191 Adult patients with • Fast Track for B-ALL 3.0 x 105 cells
Slide 13: PBCAR0191: Adverse Events Compare Favorably to Autologous CAR T
PBCAR0191: 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
Slide 14: PBCAR0191: Initial Data Support Dose Dependent Clinical Activity
PBCAR0191: 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 respo
Slide 15: Early Safety Profile Opens Three Paths towards Durable Responses
Early Safety Profile Opens Three Paths towards Durable Responses 15
Slide 16: Our Vision in Off-the-Shelf CAR T: The Best of Both Worlds
Our Vision in 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 16
Slide 17: Curing Genetic Disease: In Vivo Gene Correction
Curing Genetic Disease In Vivo Gene Correction 17
Slide 18: 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 19: 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, Sustained Editing Frequency of ARCUS PCSK9 liver 60% editing events in NHP 4 Editing Drives Therapeutic Effect ~90% reduction in serum PCSK9 levels in NHPs Hi
Slide 20: In Vivo Gene Correction Pipeline
In Vivo Gene Correction Pipeline Product Candidate Program Area Discovery Pre-clinical Clinical Rights HBV Chronic Hepatitis B HAO1 Primary hyperoxaluria type 1 (PH1) Transthyretin Familial amyloid polyneuropathy ApoC3 Lipoprotein lipase deficiency PCSK9 Familial hypercholesterolemia PH1 selected as
Slide 21: 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 22: 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 23: Feed the Planet: Elo Life Systems
Feed the Planet Elo Life Systems 23
Slide 24: 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 25: TR4 Pandemic-Resistant Cavendish Banana
TR4 Pandemic-Resistant Cavendish Banana Tropical Race 4 (TR4) is pushing the Why bananas matter Cavendish banana towards extinction • 100 billion bananas are consumed annually • Devastating fungal disease, Fusarium wilt, is a fast- spreading banana pandemic • Consumed by over 400 million people • TR
Slide 26: Multiple Upcoming Milestones Expected Across Portfolio
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 *The company expects to provide an update on this pr
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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