Precision Biosciences, Inc. · NASDAQ:DTIL
Precision Biosciences — Investor Presentation (January 2020)
34 slides from Precision Biosciences's investor presentation, Jan 15, 2020
Slide 1: Precision BioSciences: Dedicated to Improving Life
PRECISION BIOSCIENCES C; Dedicated to Improving Life. January 15, 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: Dedicated To Improving Life
Dedicated To Improving Life Overcome Cancer. Cure Genetic Disease. Feed the Planet. 3
Slide 4: Delivering on the Promise of Genome Editing
Delivering on the Promise of Genome Editing World class ARCUS genome Clinical stage team of ~200 editing platform built allogeneic CAR T Precisioneers for translation with full platform with that includes freedom to operate validating initial the pioneers in genome editing safety and response data S
Slide 5: Multiple Key Milestones Delivered in 2019
Multiple Key Milestones Delivered in 2019 Dosed first patients in Phase 1/2a trial with our lead CD19 allogeneic CAR T Opened first US in-house cGMP facility for genome edited allo CAR T cell therapies IND cleared for second allogeneic CAR T program targeting CD20 IND filed for third allogeneic CAR
Slide 6: ARCUS: Engineering Nature's Genome Editing System
ARCUS: Engineering Nature’s Genome Editing System ARCUS is derived from I-CreI, a homing Genome Editing by I-CreI in Algae endonuclease naturally evolved for highly Chlamydomonas reinhardtii precise genome editing 23S Gene Four Key Attributes I-CreI binds to its target site in the genome I-CreI 1. S
Slide 7: In-House cGMP Manufacturing for Key Components of Portfolio
In-House cGMP Manufacturing for Key Components of Portfolio • Manufacturing Center for Advanced Therapeutics (MCAT) is a 17,300 square foot cGMP clinical manufacturing facility • Allogeneic CAR T cells, mRNA (10g scale) and rAAV (400L scale) vectors for in vivo and ex vivo uses • Second phase expans
Slide 8: Deep Bench of Talent Across All Core Activities
Deep Bench of Talent Across All Core Activities Senior Leadership Includes recognized global pioneers in genome editing Team Over 200 Precisioneers including more than 85 PhD scientists and engineers Core Competencies Translational research, development, CAR T / AAV / mRNA manufacturing, clinical tr
Slide 9: Curing Genetic Disease: In Vivo Gene Correction
Curing Genetic Disease In Vivo Gene Correction 9
Slide 10: Why ARCUS for In Vivo Gene Correction?
Why ARCUS for In Vivo Gene Correction? 1 Safety Evolved to avoid random off-targeting, a critical 2 Delivery ARCUS “fits” in established gene delivery parameter for safety vehicles like AAV 3 Control of Edits ARCUS enables gene addition, gene knockout, 4 Proprietary ARCUS is the “drug” so freedom-to
Slide 11: 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 PCSK9 Familial hypercholesterolemia Transthyretin Familial amyloid polyneuropathy ApoC3 Lipoprotein lipase deficiency HAO1 Primary hyperoxaluria 11
Slide 12: Pioneering Liver Editing Collaboration with University of Pennsylvania
Pioneering Liver Editing Collaboration with University of Pennsylvania A collaboration with Jim Wilson and the Penn Orphan Disease Center was initiated August, 2016 to develop in vivo gene editing in the liver Scope Parameters • Indications: • Editing efficiency and durability - Familial hypercholes
Slide 13: 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 Transthyretin Familial amyloid polyneuropathy ApoC3 Lipoprotein lipase deficiency PCSK9 Familial hypercholesterolemia HAO1 nominated as
Slide 14: Primary Hyperoxaluria Type 1 (PH1) – Disease Overview
Primary Hyperoxaluria Type 1 (PH1) – Disease Overview 1 Rare genetic disease caused by loss-of-function mutations in the AGXT gene – Gene encodes an enzyme involved in glycine biosynthesis in the liver – Disease prevalence: 1-3/1,000,000 2 Gene mutations lead to the accumulation of calcium oxalate c
Slide 15: ARCUS Editing Effective in Pre-Clinical Models of PH1
ARCUS Editing Effective in Pre-Clinical Models of PH1 ARCUS treatment resulted in ~70% reduction in urine ARCUS efficiently knocked-out the HAO1 gene in non- oxalate in a PH1 mouse model human primates following AAV8 delivery • Prevented formation of renal CaOX crystals Mouse model Non-human primate
Slide 16: Overcoming Cancer: Off-the-Shelf CAR T
Overcoming Cancer Off-the-Shelf CAR T 16
Slide 17: Key Features of Precision's Allogeneic CAR T Platform
Key Features of Precision’s Allogeneic CAR T Platform Proprietary markers and Starting material Optimized donor cells selection criteria Product of 15+ years of ARCUS editing Single-step editing avoids off-targeting research CAR insertion CAR directly into TCR locus every time Issued Precision IP Pr
Slide 18: 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 End of Study Enrollment Infusion x1 Assessment Lymphodepletion Fludarabine 30 mg/m2/day + Cyclophosphamide 500 mg/m2/day Eligibilit
Slide 19: 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 20: Dose Levels Compared to Autologous CAR T
Dose Levels Compared to Autologous CAR T Significantly below the dose ranges of approved autologous CAR T products 1x109/kg 6x108 2x108 2.5x108 1x108/kg 1x108/kg 6x107 1x107/kg 1x107/kg 1x107 1x106 2x106 1x106 1x106/kg 1x106/kg 3x105 3x105 1x105/kg 1x105/kg DL1 DL2 Yescarta (NHL) DL1 DL2 Kymriah (AL
Slide 21: PBCAR0191 Summary: NHL Program
PBCAR0191 Summary: NHL Program CRS or ICANS Baseline disease Best Response (Mechanistic External PCR Internal Flow PFS demonstration of Expansion Expansion Patient ID burden Day ≥28 (Days)* cell expansion) (Study Days +) (Study Days +) Auto CAR T benchmarks Aggressive disease Positive 1-NHL-DL1 Part
Slide 22: PBCAR0191 Summary: ALL Program
PBCAR0191 Summary: ALL Program CRS or ICANS Baseline disease Best Response (Mechanistic External PCR Internal Flow PFS demonstration of cell Expansion Expansion Patient ID burden Day ≥28 (Days)* expansion) (Study Days +) (Study Days +) Auto CAR T benchmarks 95% marrow blasts Positive Yescarta – adul
Slide 23: Data Inform Trial Design – Aim to Further Optimize Clinical Activity
Data Inform Trial Design – Aim to Further Optimize Clinical Activity Key Protocol Amendments Recently Proposed to FDA 1 Option to re-dose after a response and subsequent disease progression 2 Split dosing into a single lymphodepletion 3 Higher total doses 4 Option to modify lymphodepletion (up or do
Slide 24: Early Activity and Safety Open Up Multiple Avenues
Early Activity and Safety Open Up Multiple Avenues Can we… Outcome Split Dosing …increase cell Optimized number protocol without tox? Repeat Dosing …maximize Dosing expansion & tailored to persistence? disease DL1 DL2 DL3 DL4 DL5 3x105 / kg 1x106 / kg 3x106 / kg 6x106 / kg 9x106 / kg …customize Shor
Slide 25: 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 • Safety profile • Accessibility • Efficacy profile 25
Slide 26: Off-the-Shelf CAR T Immunotherapy Pipeline
Off-the-Shelf CAR T Immunotherapy Pipeline Product Candidates Program Area Discovery Pre-clinical Clinical Rights PBCAR0191 (CD19) NHL and ALL – Phase 1 Data 2020 PBCAR20A (CD20) NHL, CLL, SLL – Phase 1 Dosing Q1 2020 PBCAR269A (BCMA) MM - IND cleared – Phase 1 Dosing 2020 26
Slide 27: Clear Emerging Leadership in Allogeneic CAR T
Clear Emerging Leadership in Allogeneic CAR T Objective tumor responses observed at first two dose levels Interim data suggest safety profile that compares favorably to auto CAR T Indication of dose-dependent mechanism of action Evidence allogeneic CAR T can be effective in true “off-the-shelf” sett
Slide 28: Feed the Planet: Elo Life Systems
Feed the Planet Elo Life Systems 28
Slide 29: Elo Life Systems is Focused on Healthy and Sustainable Food
Elo Life Systems is Focused on Healthy and Sustainable Food A Human Health Opportunity An Efficient Business Model ELO LIFE SYSTEMS Food companies need new inputs to respond to: Discovery Proof of Concept Lead Generation • Climate change. Critical raw materials like citrus and banana are under exist
Slide 30: Food Pipeline
Food Pipeline Product Discovery Greenhouse Field Program Lead Ultra-low Saturate Canola Oil* ZeroMelonTM (Melon Sweetener)* High Protein Chickpea *Partner funded in whole or in part 30
Slide 31: ZeroMelon: A Zero Calorie Sugar Substitute
ZeroMelonTM: A Zero Calorie Sugar Substitute • Elo’s ZeroMelonTM program leverages ARCUS to reactivate ARCUS-mediated reactivation of two latent dormant genes in watermelon to produce natural, zero mogroside V pathway genes in watermelon calorie sweetener mogroside V 15 • 250x sweeter than artificia
Slide 32: Upcoming Milestones Expected Across Portfolio
Upcoming Milestones Expected Across Portfolio IND accepted for BCMA CAR T Initiate dosing for CD20 CAR T Initiate dosing for BCMA CAR T Primary hyperoxaluria candidate selection Start ZeroMelonTM greenhouse studies NHL (CD19) clinical data update ALL (CD19) clinical data update 32
Slide 33: Key Takeaways
Key Takeaways Highly experienced team includes the pioneers in editing Proprietary ARCUS editing platform with leading specificity and freedom to operate Independent cGMP manufacturing capabilities Initial allogeneic CAR T clinical data validates core strategy Gene correction programs expand upside
Slide 34: Dedicated To Improving Life
Dedicated To Improving Life 34
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