Precision Biosciences, Inc. · NASDAQ:DTIL
Precision Biosciences — Merger or Acquisition (June 2022)
24 slides from Precision Biosciences's merger presentation, Jun 22, 2022
Slide 1: Precision BioSciences & Novartis to Collaborate on In Vivo Gene Editing Program for Hemoglobinopathies
Precision BioSciences & Novartis to Collaborate on Potentially Curative In Vivo Gene Editing Program for Hemoglobinopathies June 22, 2022
Slide 2: Forward-Looking Statements
Forward-Looking Statements This presentation contains forward-looking statements, as may any related presentations, within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this herein and in any related presentation that do not relate to matters of his
Slide 3: Michael Amoroso, President & Chief Executive Officer
Michael Amoroso President & Chief Executive Officer
Slide 4: Precision Biosciences is Delivering on the Promise of Therapeutic Genome Editing
Precision Biosciences is Delivering on the Promise of Therapeutic Genome Editing to Transform the Future of Medicine ARCUS® Genome Editing Platform Precision and Versatility for In Vivo and Ex Vivo Therapeutics In Vivo Editing for Ex Vivo single-gene edit for Genetic Diseases Allogeneic CAR T immuno
Slide 5: In Vivo Gene Insertion Collaboration with Novartis for Sickle Cell Disease
In Vivo Gene Insertion Collaboration with Novartis for Sickle Cell Disease Builds on the unique gene insertion capabilities of + ARCUS® and further validates ARCUS as a premium genome editing platform Precision receives $75M upfront for a single target/single nuclease Collaboration with Novartis,
Slide 6: Novartis, the Partner of Choice for an In Vivo Gene Editing Hemoglobinopathy Transformational Therapy
Novartis, the Partner of Choice for an In Vivo Gene Editing Hemoglobinopathy Transformational Therapy • Collaboration with Novartis provides validation to Precision’s ARCUS platform “ We identify here a collaborative opportunity to imagine a unique therapeutic option for patients with hemoglobinopat
Slide 7: Derek Jantz, Ph.D., Chief Scientific Officer, Co-Founder
Derek Jantz, Ph.D. Chief Scientific Officer, Co-Founder
Slide 8: ARCUS is derived from I-CreI, a homing endonuclease from algae
I-CreI is 1 expressed 23S rRNA gene I-CreI 5’-CAAACTGTCGTGAGACGTTTTG-3’ 2 recognizes its target in the ARCUS is derived 3’-GTTTGACAGCACTCTGCAAAAC-5’ 23S rRNA gene from I-CreI, a homing endonuclease from algae I-CreI cuts and produces 3’ 5’-CAAACTGTCGTGA GACGTTTTG-3’ 3 evolved for precise overhangs 3
Slide 9: Safety
Safety This allows ARCUS to be expressed for extended periods of time ARCUS is inactive from an adeno-associated virus (AAV) vector without accumulating off- until it binds to its target gene edits. target DNA site 16 ARCUS nucleases have been evaluated in non-human primates representing, collecti
Slide 10: Delivery
Delivery ARCUS ARCUS is 364 amino acids. This enables delivery to tissues and cells using a variety of viral and non-viral delivery is small technologies including AAV. An ARCUS gene requires only a fraction of the carrying capacity of an AAV vector. This leaves the majority of the vector capacity a
Slide 11: Versatility
Versatility ARCUS can efficiently The 3’ overhangs produced by ARCUS potently stimulate DNA repair insert DNA into the by homology-directed repair (HDR). This enables transgenes to be genome inserted into the genome. Real world example: Targeted Insertion of an OTC Transgene in NHP Liver DAPI OTC Wa
Slide 12: Hemoglobinopathies are a Major World Health Problem
Hemoglobinopathies are a Major World Health Problem Sickle Cell Disease (SCD) Beta Thalassemia Affects the structure/function of hemoglobin, One of the most common genetic diseases caused by a disruption reducing the ability of red blood cells to transport of normal hemoglobin production oxygen • Co
Slide 13: We are Pursuing an In Vivo Approach to SCD
We are Pursuing an In Vivo Approach to SCD Our goal is a one-time, IV If successful, an in vivo therapy for SCD might be administered administration of drug in regions that do not have transplant centers. In vivo gene editing Ex vivo gene editing + 13
Slide 14: Gene Insertion to a Safe Harbor Locus in Hematopoietic Stem Cells
Gene Insertion to a “Safe Harbor” Locus in Hematopoietic Stem Cells ARCUS will be used to add an Permanent integration of an antisickling gene into a antisickling gene to hematopoietic “safe harbor” locus in HSCs is expected to prevent the stem cells (HSCs) sickle cell phenotype in mature erythrocyt
Slide 15: Gene Editing Pipeline Validation and Expansion Creates Value
Gene Editing Pipeline Validation and Expansion Creates Value 22 Pipeline Expansion Gene insertion, Complex Edits & Multiple Tissues in partnership with in partnership with 1 Lilly Novartis Clinical Validation PBGENE-DMD Knock-out Genes in Liver PBGENE-LLY2 PBGENE-HbE PBGENE-LLY3 PBGENE-PCSK9 PBGENE-
Slide 16: Cindy Atwell, Senior Vice President, Business Development
Cindy Atwell Senior Vice President, Business Development
Slide 17: Novartis Deal Meets all Precision Business Development Principles
Novartis Deal Meets all Precision Business Development Principles: Systematic and Disciplined Approach to Funding Business & Acquiring Capabilities Business Development Rules of Engagement for In Vivo Gene Editing Maximize upfront and near-term payments to complement financing Garner “premium” deals
Slide 18: Deal Structure Leverages Precision Expertise for ARCUS Nuclease Creation
Deal Structure Leverages Precision Expertise for ARCUS Nuclease Creation • Design and optimize a single, • Novartis to develop as a Focus on core potential in vivo treatment for custom ARCUS nuclease for capability creates sickle cell disease and beta “safe harbor” target site minimal distraction to
Slide 19: Summary of Deal Economics: $75M Upfront for Single Target
Summary of Deal Economics: $75M Upfront for Single Target $75M • $50M cash Upfront Payment • $25M equity at 20% premium to 10-day VWAP* ending 06/13/2022 Aggregate Milestones & Fees $1.4B milestones + research funding Royalties on Commercialized Products Mid-single digit to low-double digits Shares
Slide 20: Novartis Collaboration is Complementary to Precision's Existing Partnership with Lilly
Novartis Collaboration is Complementary to Precision’s Existing In Vivo Gene Editing Partnership with Lilly Research collaboration and license agreement aimed at treating + challenging genetic diseases Pre-IND R&D IND to Commercial 3 Initial collaboration for three targets, including Duchenne muscul
Slide 21: Michael Amoroso, President & Chief Executive Officer
Michael Amoroso President & Chief Executive Officer
Slide 22: Broad and Deep In Vivo Gene Editing Pipeline Demonstrates Unique Attributes of ARCUS
Broad and Deep In Vivo Gene Editing Pipeline Demonstrates Unique Attributes of ARCUS EDIT TYPE / CANDIDATE IND- EXPECTED PROGRAM INDICATION TISSUE TARGET RESEARCH PARTNER DELIVERY SELECTION ENABLING IND/CTA WHOLLY OWNED Familial PBGENE-PCSK9 hypercholesterolemia Liver PCSK9 Deletion/AAV 2022* Primar
Slide 23: Building the leading therapeutic gene editing company focused on high unmet needs
Technology: ARCUS, a premier genome editing platform People: Fortified Senior Leadership Building the leading therapeutic Team with 15+ years perfecting gene editing company focused on ARCUS protein engineering high unmet needs in oncology and Focus & Discipline: genetic diseases • 3 INDs in 3 years
Slide 24: Q&A
Q&A WD PRECISION
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