Intellia Therapeutics, Inc. · NASDAQ:NTLA
Intellia Therapeutics — Investor Presentation (January 2019)
25 slides from Intellia Therapeutics's investor presentation, Jan 7, 2019
Slide 1: Corporate Overview January 2019
Corporate Overview January 2019 Exhibit 99.1
Slide 2: Intellia Therapeutics' Legal Disclaimer
Intellia Therapeutics’ Legal Disclaimer This presentation contains “forward-looking statements” of Intellia Therapeutics, Inc. (“Intellia”) within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied
Slide 3: Our Mission
Our Mission Developing curative genome editing treatments that can positively transform the lives of people living with severe and life-threatening diseases
Slide 4: Elements for Our Success
Elements for Our Success Develop Curative CRISPR-Based Medicines Advance Our Science Be the Best Place to Make Therapies Build for Long-Term Sustainability
Slide 5: Overview: Modular Approach Drives Diversified Pipeline
Overview Modular Approach Drives Diversified Pipeline Full-Spectrum Genome Editing Company Significant in vivo preclinical validation Experienced management team At least two programs entering development in 2019 Well capitalized to achieve milestones ATTR AML > > CRISPR/Cas9 ATTR: Transthyretin Amy
Slide 6: Building a Full-Spectrum Genome Editing Company
Building a Full-Spectrum Genome Editing Company CRISPR creates the therapy CRISPR is the therapy Immuno-oncology Autoimmune diseases LNP: Lipid Nanoparticle Genetic diseases Modular Platform LNP CELL RNA CRISPR/Cas9
Slide 7: CRISPR/Cas9 is an Effective Tool for Modifying the Genome
CRISPR/Cas9 is an Effective Tool for Modifying the Genome KNOCKOUT Inactivation/deletion of disease-causing DNA sequence Insert new DNA sequence to manufacture therapeutic protein Correction of “misspelled” disease-driving DNA sequence REPAIR INSERT CRISPR/Cas9
Slide 8: CRISPR is the therapy: Delivering CRISPR/Cas9 to Treat Genetic Diseases
CRISPR is the therapy KNOCKOUT REPAIR INSERT + Knock out of toxic or compensatory genes Introduce functional DNA sequence Correct genes with few mutations DELIVERY SYSTEM: PROPRIETARY AND MODULAR LNP lipids mRNA gRNA Template design Template delivery system TEMPLATE Delivering CRISPR/Cas9 to Treat P
Slide 9: CRISPR creates the therapy: Engineering Cells for Immuno-Oncology
STEM CELL / T CELL INSERT COMBINATIONS (Insertion and Knockout) CRISPR creates the therapy KNOCKOUT Genes causing tolerability issues Genes controlling efficacy and safety to modify cell function Cancer-targeting genetic constructs ENGINEERING SYSTEM: PROPRIETARY AND MODULAR Targeted and efficient i
Slide 10: Genome Editing Platform Generates Full-Spectrum Pipeline
Genome Editing Platform Generates Full-Spectrum Pipeline LNP Select and design the modular components Genetic Disease Targets CELL Cell Function and Insertion Targets ATTR, AATD and PH1 AML and solid tumors AATD: Alpha-1 Antitrypsin Deficiency PH1: Primary Hyperoxaluria Type 1 Full-spectrum pipeline
Slide 11: Significant Preclinical Validation
Significant Preclinical Validation Knockout >95% reduction in TTR protein levels in NHPs >95% protein reduction (TTR and A1AT) in mice Well-tolerated editing durability in ATTR studies Insertion First to demonstrate insertion of F9 and SERPINA1 in the liver of mice First to achieve therapeutic level
Slide 12: R&D Programs Poised to Move to the Clinic
R&D Programs Poised to Move to the Clinic PROGRAM RESEARCH EARLY DEVELOPMENT PARTNER Transthyretin Amyloidosis Alpha-1 Antitrypsin Deficiency Primary Hyperoxaluria Type 1 Undisclosed Acute Myeloid Leukemia Additional Immuno-Oncology Programs Novartis Programs: - Selected Hematopoietic Stem Cells - A
Slide 13: Transthyretin Amyloidosis (ATTR)
1Ann Med. 2015; 47(8): 625–638. 50,000 ATTR patients worldwide1 Caused by accumulation of misfolded transthyretin (TTR) protein, which affects nerves, heart, kidneys and eyes 2-15 yrs Typical life expectancy from onset of symptoms1 Only chronic treatment options currently available ATTR Program Achi
Slide 14: Achieved Persistent Protein Reduction for Months in Mice
Achieved Persistent Protein Reduction for 12 Months in Mice TTR Editing TTR Protein Reduction Serum TTR High Dose Medium Dose Low Dose Single dose TTR Editing Liver Editing Single dose
Slide 15: Improved Delivery Formulations Achieve Therapeutic Liver Editing Levels in NHPs
Tested formulations show >75% liver editing and >95% reduction in circulating levels of TTR protein Liver editing of 35-40% is sufficient to achieve a reduction of >60% of TTR protein Therapeutically Relevant Range: >60% TTR Knockdown Improved Delivery Formulations Achieve Therapeutic Liver Editing
Slide 16: Early Development Considerations
Early Development Considerations First-in-human (FIH) study: Suitability of open label, dose escalation design Primary objectives: Safety, tolerability and indications of efficacy Secondary endpoints: Relevant biomarkers and symptomology measures to assess efficacy Potential expansion: Cohorts based
Slide 17: Acute Myeloid Leukemia (AML): First Wholly Owned Engineered Cell Therapy
1 NIH SEER Cancer Stat Facts: Leukemia – Acute Myeloid Leukemia (AML) 2 GlobalData EpiCast Report: Acute Myeloid Leukemia July 2017, 7MM: Seven Major Markets (includes U.S.) ~20,000 New cases in the U.S. in 20181 Cancer of the blood and bone marrow that is rapidly fatal without immediate treatment M
Slide 18: Wilms' Tumor (WT1) is an Attractive Tumor Target
Wilms’ Tumor 1 (WT1) is an Attractive Tumor Target Sugiyama et al., Jap J Clin Oncol, 2010 WT1 is Overexpressed in >90% of AML Blasts Cilloni et al., J Clin Oncol, 2009 WT1 is Overexpressed in >90% of Solid Tumors Normalized WT1 expression Normal expression Overexpression
Slide 19: Driving Our First Engineered Cell Therapy Towards the Clinic
Driving Our First Engineered Cell Therapy Towards the Clinic Cells with original TCR KO plus insertion of new TCRs kill patient-derived AML blasts Identified natural TCRs with equal binding to WT1 while effective against many cancers Removal of competing TCRs In collaboration with Ospedale San Raffa
Slide 20: Building for Long-Term Sustainability
Building for Long-Term Sustainability Modular Platform Delivery to the liver CAPABILITIES PIPELINE PROGRESSION Multiple INDs Human POC / Pivotal Trials LNP CELL Autologous Hematological Delivery to other tissues (CNS, muscle, etc.) Allogeneic Solid Tumors LNP CELL TTR AML AATD PH1 Immuno-Oncology Pr
Slide 21: Experienced Management Team
Experienced Management Team Driving Intellia to Deliver Innovative Therapies John Leonard, M.D. President and Chief Executive Officer Jennifer King, Ph.D. Senior Vice President, Business Development Nishla Keiser, Ph.D. Senior Vice President, Deputy General Counsel José Rivera Executive Vice Preside
Slide 22: Partnerships Provide R&D Capabilities to Enhance Pipeline Growth
Partnerships Provide R&D Capabilities to Enhance Pipeline Growth Multi-year collaboration Up to 10 in vivo targets Key deal components Liver-centric product development Platform development Partnership with leading pharmaceutical company Access to: Regeneron Genetics Center Animal model development
Slide 23: Intellia Milestones
Intellia Milestones 2019 ATTR: Initiate IND-enabling GLP toxicology studies AML: Nominate first engineered cell therapy development candidate by the end of 2019 Present additional in vivo insertion data at upcoming scientific conferences ATTR: Submit IND application 2020 ATTR: Achieved improved NHP
Slide 24: Elements for Our Success
Elements for Our Success Develop Curative CRISPR-Based Medicines Advance Our Science Be the Best Place to Make Therapies Build for Long-Term Sustainability
Slide 25: Intellia Therapeutics
Inteziia THERAPEUTICS
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