Precision Biosciences, Inc. · NASDAQ:DTIL
Precision Biosciences — Investor Presentation (June 2020)
27 slides from Precision Biosciences's investor presentation, Jun 2, 2020
Slide 1: Precision BioSciences: Dedicated to Improving Life
Exhibit 99.1 1 PRECISION BIOSCIENCES Dedicated to Improving Life. Cure genetic disease. Feed the planet.
Slide 2: 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 limitation, statements regarding
Slide 3: Delivering on the Promise of Genome Editing
Delivering on the Promise of Genome Editing ARCUS genome editing platform built for translation with full freedom to operate In vivo gene correction platform seeking to cure genetic and infectious diseases Wholly integrated food editing platform focused on human wellness and food security Scaled in-
Slide 4: ARCUS: Precision's Proprietary Gene Editing Platform
ARCUS: Precision’s Proprietary Gene Editing Platform Precision bioengineering I-CreI ARCUS Key Advantages Safety: minimizes off-target editing; natural “off switch” Ease of delivery: small size permits both LNP and AAV delivery Control of edits: efficient knock in or knock out Proprietary: more than
Slide 5: Efficient, Scaled In-house Manufacturing Already Established
Efficient, Scaled In-house Manufacturing Already Established Operational July 2019 17,300 sq. ft. facility in Durham, NC Currently producing clinical trial material for BCMA CAR T program Fully cGMP compliant Ability to produce ARCUS-based CAR T and in vivo therapies MCAT: Manufacturing Center for A
Slide 6: Overcoming Cancer: Off-the-Shelf CAR T
Overcoming Cancer Off-the-Shelf CAR T
Slide 7: The Allogeneic CAR T Opportunity
The Allogeneic CAR T Opportunity Autologous CAR T Allogeneic CAR T High rates of efficacy in some cancers Can be effective where other options have failed Early evidence of encouraging clinical efficacy High cost and challenging logistics leading to limited patient access Patient-to-patient product
Slide 8: Four Key Requirements for Allogeneic CAR T Success
True off-the-shelf safety profile Supports ease of use / physician adoption Minimize CRS / neurotoxicity Efficiently deliver consistent, high quality cell product Reach all eligible patients Control costs Healthy cell product High percentage naïve and central memory T cell phenotypes Ability to empl
Slide 9: 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 Key Features of Precision’s Allogeneic CAR T Platform Optimized T cell from healthy donor High yield, consistent drug product targeted Short, 10-day manufacturing CAR expressed with propri
Slide 10: Platform Is Delivering Against All Four Key Requirements
>50% naïve and central memory T cells Zero GvHD, severe CRS or neurotox; zero infections* Ability to manufacture consistent product at scale Encouraging early activity at low dose levels* Platform Is Delivering Against All Four Key Requirements 67% ORR in NHL 2 CRs achieved 28+ days 1 NHL / 1 ALL 18
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) PBCAR20A (CD20) PBCAR269A (BCMA) NHL and ALL – Phase 1 Data Expected 2020 NHL, CLL, SLL – First Patient Dosed April 2020 MM – IND cleared – Phase 1 Dosing Ex
Slide 12: Allogeneic CAR T Study Designs
Targeting CD19 PBCAR0191 Targeting CD20 PBCAR20A Targeting BCMA PBCAR269A Allogeneic CAR T Study Designs Population Dose Escalation Range Status Adult patients with R/R NHL (including MCL), or R/R B-ALL 3.0 x 105 cells/kg - 9.0 x 106 cells/kg Actively enrolling in escalating doses ODD granted for MC
Slide 13: PBCAR0191: Adverse Events Compare Favorably to Autologous CAR T
PBCAR0191: Adverse Events Compare Favorably to Autologous CAR T System Organ Class Preferred Term, n(%) NHL (n=6) B-ALL (n=3) CRS (Cytokine Release Syndrome) – Grade 1 or Grade 2 2 (33%) 1 (33%) ICANS (Immune Effector Cell Neurotoxicity) – Grade 1 or Grade 2 0 (0%) 1 (33%) 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+ # patients DL1 (n=3) DL2 (n=3) CAR T cell expansion # patients with positive expansion (maximum days positive) 2/3 pts (Day 3**) DL1 (n=3) DL2 (n=3) qPCR Flow qPCR Flow 2/3 pts (Day 21†) 3/3 pts (D
Slide 15: Early Safety Profile Opens Three Paths towards Durable Responses
Early Safety Profile Opens Three Paths towards Durable Responses
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 First Line Safety profile Accessibility CAR T Last Line Efficacy profile Safety profile Accessibility Efficacy profile PBCAR Future portfolio in solid tumors Liquid tumors ü
Slide 17: Curing Genetic Disease: In Vivo Gene Correction
Curing Genetic Disease In Vivo Gene Correction
Slide 18: Four Key Requirements for Successful In Vivo Gene Correction
Safety 1 Editing Drives Therapeutic Effect 4 Ease of Delivery 2 Efficient, Versatile Editing 3 Four Key Requirements for Successful In Vivo Gene Correction Knock in and/or repair of gene defects substantially increases utility of platform Specific and high efficiency Minimize off-target gene editing
Slide 19: ARCUS Platform Delivers Against All Four Key Requirements
Safety 1 ARCUS Platform Delivers Against All Four Key Requirements Full freedom-to-operate for ARCUS platform * as assessed by oligo capture technique PCSK9 data reported in part in Wang et al, Nature Biotechnology, 2018 Gen 3 ARCUS achieved undetectable levels of off target editing in NHPs Gen 3 n=
Slide 20: In Vivo Gene Correction Pipeline
Product Candidate Program Area Discovery Pre-clinical Clinical Rights HBV HAO1 Transthyretin ApoC3 PCSK9 Familial amyloid polyneuropathy Chronic Hepatitis B – IND 2021 Lipoprotein lipase deficiency Familial hypercholesterolemia In Vivo Gene Correction Pipeline PH1 selected as lead wholly-owned in vi
Slide 21: Overview of Primary Hyperoxaluria Type 1 (PH1)
Overview of Primary Hyperoxaluria Type 1 (PH1) Prevalence of 1-3/1,000,000 Affects adults and young children ~40% patients have end-stage renal disease at the time of diagnosis ARCUS mediated knockout of HAO1 gene in liver Prevent buildup of oxalate Aim to develop a one-time, permanent treatment Com
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 in non-human primates following AAV8 delivery NHP 1 NHP 2 NHP 3 NHP 4 Day 128 Knock-out % ARCUS treatment resulted in ~70% reduction in urine oxalate in a PH1 mouse model 0 7 14 21 28 35 42 49
Slide 23: Feed the Planet: Elo Life Systems
Feed the Planet Elo Life Systems
Slide 24: An Efficient Business Model
An Efficient Business Model A Human Health Opportunity Food companies need new inputs to respond to: Climate change Consumer preference Elo integrates ARCUS with enabling technologies to create greatly needed improvements to sources of food Partner driven Minimal capital investment Elo is Focused on
Slide 25: 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 ü ü
Slide 26: 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 $154.2m cash funds*, runway into 2H 2021; validating partne
Slide 27: Precision BioSciences: Overcome Cancer. Feed the Planet.
PRECISION BIOSCIENCES rc ria Y aul Overcome Cancer. Feed the Planet. Dedicated To Improving Life
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