Roivant Sciences Ltd. · NASDAQ:ROIV
Roivant Sciences — Investor Day (September 2021)
131 slides from Roivant Sciences's investor day presentation, Sep 28, 2021
Slide 1: Roivant R&D Day September 28, 2021
Roivant R&D Day September 28, 2021 Exhibit 99.1
Slide 2: Statement of Limitations (1/2)
Statement of Limitations (1/2) The following is a series of presentations (the “Presentation”) prepared by Roivant Sciences Ltd. and certain of its subsidiaries and affiliates (the “Company”). This Presentation is only for its intended investor audience, and for informational purposes only, and it i
Slide 3: Statement of Limitations (2/2)
Statement of Limitations (2/2) Key Performance Indicators This Presentation may include certain key performance indicators (“KPIs”). Management regularly reviews these and other KPIs to assess the Company’s operating results. Realized return on our investments in Vants and technology sold to Sumitom
Slide 4: Table of Contents
Table of Contents Roivant Overview 5 Roivant Discovery 19 Proteovant 35 Genevant 45 Dermavant 61 Immunovant 98 Aruvant 105 Kinevant 123
Slide 5: Roivant: Redefining Big Pharma from End to End
Roivant: Redefining “Big Pharma” from End to End Roivant in 2014 Roivant Today Discover Develop Commercialize 1. Create Value 2. Be Contrarian 3. Climb the Wall 4. Sweat the Details 5. Evolve or Die Our Principles 5
Slide 6: How We've Executed on Our Vision
How We’ve Executed on Our Vision Cited figures and associated investment multiples are Key Performance Indicators. Please refer to the information included on Slide 3 with respect to our KPIs. 1. Medicine, Vant launch, and approval figures include Alliance Vants transferred to Sumitovant, a wholly-o
Slide 7: Consecutive Positive Phase Studies
8 Consecutive Positive Phase 3 Studies Note: The FDA has approved relugolix for the treatment of adult patients with advanced prostate cancer and for the management of heavy menstrual bleeding associated with uterine leiomyomas (fibroids) in premenopausal women. The FDA has approved vibegron for the
Slide 8: Development Pipeline
Gene Therapy Cell Therapy Topical Biologic Small Molecule Preclinical Phase 1 Phase 2 Phase 3 Registration Note: All drugs in current pipeline are investigational and subject to health authority approval. Development Pipeline Modality TAPINAROF Psoriasis | Dermavant ► TAPINAROF Atopic Dermatitis | D
Slide 9: Tapinarof: Potential to Transform Treatment of Psoriasis and Atopic Dermatitis
Leveraged platform expertise to expand IP with multiple patents expected to provide protection until at least 2036 Hired leadership and provided investment that together delivered Phase 3 success Value Added by Roivant Platform Psoriasis Phase 3: Statistically significant improvement in PGA score of
Slide 10: ARU-1801 Well-Positioned Against Competitors
Well-Positioned Against Competitors1 Longstanding Roivant relationship with CCHMC enabled initial asset license and strong academic-industry partnership Manufacturing process improvements have enabled increased hemoglobin F expression and vaso-occlusive event (VOE) reduction Value Added by Roivant P
Slide 11: Immunovant IMVT-1401 Anti-FcRn Program
Rapidly initiated multiple Phase 2 trials to develop anti-FcRn antibody IMVT-1401 as a best-in-class or first-in-class subcutaneous injection Note: All drugs in current pipeline are investigational and subject to health authority approval. All trademarks are property of their respective owners. Base
Slide 12: Vant Model Enables Rapid Scaling
Vant Model Enables Rapid Scaling Alyvant Lokavant Datavant Proteovant Affivant Sio Arbutus New Vants from Additional In-Licensing Immunovant Dermavant Aruvant Genevant Cytovant Lysovant Kinevant New Vants from
Slide 13: The Roivant Model for Drug Discovery and Development
The Roivant Model for Drug Discovery and Development Find assets externally and in-license Discover drugs internally and advance into the clinic Plan to in-license multiple potentially category-leading drugs per year Develop drugs at Vants Identify drug targets and biological pathways Plan to submit
Slide 14: Entering the Era of Predictive, High-Precision Molecular Medicine
Entering the Era of Predictive, High-Precision Molecular Medicine 1980 2000 2021 2010 2016 SBDD DOCKING WATERS DYNAMICS INTEGRATED BIOPHYSICS, DYNAMICS, AND AI/ML We are positioned to realize the promise of rational drug design by treating drug discovery as engineering Ability to run atom-by-atom si
Slide 15: The Roivant Vision in Drug Discovery
TPD: targeted protein degradation. The Roivant Vision in Drug Discovery: Small Molecule Drug Design as Engineering Inputs “Investment lens” for target identification, powered by interdisciplinary team of R&D experts, investors and data scientists Outputs Drug candidates paired with Roivant’s extensi
Slide 16: What's Really Inside Our Engine?
What’s Really “Inside” Our Engine? PROPRIETARY TOOLS EXPERTISE Conformational Genetics Analyze genomic variants to define disease targets and map mutations to 3-D structures to predict novel MOA and conformational mechanism Druggability Assessment Predict high-energy water hotspots and druggable poc
Slide 17: Roivant Builds Technologies to Transform Biopharma
1. The implied enterprise value of the combined company at the conversion price cap of the new preferred equity investment made concurrently with the merger was $7.0 billion. This enterprise value implies an equity value of $6.1 billion (after netting out approximately $900 million of debt and other
Slide 18: Key Near-Term Potential Catalysts
All catalyst timings are based on current expectations but may be subject to change. All trademarks are property of their respective owners. Drugs are investigational and subject to regulatory approval. Key Near-Term Potential Catalysts Tapinarof NDA Filing in Psoriasis Mid-2021 Tapinarof Phase 3 In
Slide 19: From Chip to Clinic
From Chip to Clinic Integrating Advanced Simulation and AI Approaches to Design Novel Medicines for Challenging Disease Targets
Slide 20: Integrated Drug Discovery at Roivant
Integrated Drug Discovery at Roivant – From Chip to Clinic >600 GPUs >6000 CPUs Cloud for bursts Custom FPGA research SUPERCOMPUTER Experienced drug designers Unique ability to combine experimental & computational data 10,000 sq. ft. in-house laboratory FULLY INTEGRATED SCIENTIFIC EXPERTISE ~90 PhD
Slide 21: Leading Computational Discovery Capabilities in Physics and AI/ML
Leading Computational Discovery Capabilities in Physics and AI/ML Sample in silico Assays Identify novel binding sites on a protein and assess druggability Predict binding affinity and selectivity of a ligand to a protein, including ternary complexes Simulate conformational dynamics of a protein as
Slide 22: Improvements from Combining AI and Physics Approaches
Improvements from Combining AI and Physics Approaches χ: atomic electronegativity that characterizes how strongly an electron is associated with an atom J(0): atomic hardness that characterizes how much the atomic charge varies under a external field E: electrostatic potential energy q: atomic charg
Slide 23: Roivant Biophysics and Structural Biology Advantage
Roivant Biophysics and Structural Biology Advantage Generated hundreds of protein samples using 3 expression systems (mammalian, insect, and bacterial) Protein labeling for NMR Protein Science X-ray Crystallography Detect buried protein regions in binary and ternary complexes Augment simulations to
Slide 24: Conformational Modulation Assays with Integrated MD + Biophysics
Conformational Modulation Assays with Integrated MD + Biophysics x: atomic coordinates Er: restrained potential energy function E0: unbiased potential energy function R: experimental restraining energy De: experimental data conformation expt. bias experimental data simulation data Protein Protein+in
Slide 25: Witnessing Relevant Biological Timescales with Atomic Resolution
Witnessing Relevant Biological Timescales with Atomic Resolution Protein Folding Water reorganization Allosteric binding Agonism and conformational related events Loop dynamics and domain breathing Protein folding Timescale Relevant biological event Critical timescale to make project-driving predict
Slide 26: Free Energy Calculations and Atomic Decomposition
Peer to Schrödinger’s FEP+ for speed and accuracy of binding free energy calculations. Novel atomic decomposition of binding free energy lets our drug designers “see” areas for improvement. Free Energy Calculations and Atomic Decomposition for Better Designs
Slide 27: What Does the Predictive Sciences Platform Enable?
The era of predictive, high-precision molecular medicines across multiple modalities What Does The Predictive Sciences Platform Enable? Target ID Relating genetics to protein conformations Identification of novel MOAs Druggability and Novel Binding Sites Detection of cryptic/dynamic pockets Fragment
Slide 28: Design Cycle
Design Cycle – Ideate, Design, Predict, Make, Test, Analyze, Iterate Explore billions of new molecule designs Binding Free Energies Conformational Energies Off-target Selectivity ADME/Tox Models AI/ML Models Atomic Decomposition Proprietary method Answers “why” for good/bad binders Conformational Mo
Slide 29: Beyond Binding: Conformational Modulation and Induced Proximity
Beyond Binding: Conformational Modulation and Induced Proximity
Slide 30: Advanced Simulation Approach
Advanced Simulation Approach Bespoke Force Field All ligands are parameterized from scratch with QM Advanced Simulations Long timescale Biophysics constraints Conformational free energies Protein-Protein Interactions Docking Refinement Analysis Binding Free Energies Relative Absolute Energy decompos
Slide 31: Heterobifunctional Molecules for Protein Degradation
Example of How We Develop Methods to Overcome Critical Bottlenecks: Heterobifunctional Molecules for Protein Degradation E3 POI E3 ligand Protein of Interest (POI) POI Ub POI Ub Ub Ub Ub Proteasome Catalytic Cycle POI E2 Ub E3 POI E3 LYS Heterobifunctional molecule POI warhead E3 ligase Linker Free
Slide 32: Modeling the Ternary Structure is Critical to Predictive TPD
Modeling the Ternary Structure is Critical to a Predictive TPD Platform Binding Kinetics Binding Affinity Structure Dynamics Off-the-shelf computational methods are not suited to accurately predict the dynamic solution-state ternary complex
Slide 33: Fully Integrated Binding Simulation with HDX Data
Fully Integrated Binding Simulation with Hydrogen-Deuterium Exchange Data Most Accurate Ternary Structure Prediction Known Integration of experimental hydrogen-deuterium exchange mass spectrometry (HDX-MS) data offers unique advantage Final Statistics: Warhead-interface RMSD = 0.40 Å Ligand-interfac
Slide 34: Conclusions
Conclusions Roivant is a fully integrated drug discovery company – “From Chip to Clinic” We have built an industry-leading computational platform for molecular simulations and AI/ML to overcome critical bottlenecks in drug discovery The development of custom apps is key for drugging challenging targ
Slide 35: Degrading Proteins, Defeating Disease
Degrading Proteins, Defeating Disease
Slide 36: Degrading Proteins, Defeating Disease
Degrading Proteins, Defeating Disease By harnessing the human body’s innate cellular machinery to selectively ‘delete’ proteins, we aim to create new medicines to treat patients with debilitating diseases We are pursuing this expansive field with the scientific knowledge, proprietary technologies, b
Slide 37: Why Targeted Protein Degradation?
2015 Crews et al. Nature Chemical Biology; 2017 Crews et al. Cell Chemical Biology; 2019 Chamberlain et al. Cell Chemical Biology; 2019 Sun et al. Signal Transduction and Targeted Therapy Why Targeted Protein Degradation? Demonstrated efficacy, as a function of protein depletion, in tumors that are
Slide 38: Expanding the Druggable Proteome via the Ubiquitin-Proteasome System
Graphic: Adapted from Advances in targeting ‘undruggable’ transcription factors with small molecules. Henley M.J. Nature Reviews (2021). Unlocking The Vast Opportunity To Expand The Druggable Proteome By Exploiting the Ubiquitin-Proteasome System (UPS) Protein degradation via the UPS is a multiple s
Slide 39: Proteovant: Pioneer in Protein Degrader Discovery
Proteovant – Positioned As A Leading Pioneer In Protein Degrader Discovery and Development Formed new Vant focused on the discovery and development of novel targeted protein degraders Assembled a world-class team of discovery scientists, drug developers and business professionals Acquired Oncopia Th
Slide 40: World-Class Executive Team
World-Class Executive Team Positioned to Execute on Our Vision Drew Fromkin Chief Executive Officer 30+ years leadership in healthcare co’s, serves as Vant Portfolio Operating Partner. Previously CEO Tarveda Therapeutics; CEO of Clinical Data (CLDA – $1.5 Billion Sale); Head Corp Dev. Merck- Medco C
Slide 41: Proteovant's Differentiated Capabilities
Proteovant’s Leading Protein Degrader Discovery and Development Engine Is Fueled By Differentiated Capabilities Target Selection and Validation Driven by seasoned team of R&D, structural biology, and strategy experts Degrader Expertise Multi-year, exclusive partnership with the University of Michiga
Slide 42: Selected Pipeline Programs
Selected Pipeline Programs * Multiple programs For Investor Audiences Only Drugs are investigational and subject to regulatory approval. Balanced pipeline of protein degrader targets spanning Oncology and Immunology Advancing initial protein degrader programs from the Oncopia acquisition Enhancing p
Slide 43: VantAI: A Novel Paradigm For Rational Degrader Discovery
1 Restrained Protein Docking using LightDock 2 Enrichment: % of predicted ternary complexes within 10A Ligand RMSD (based on CAPRI) of crystalized ground truth glue ternary complexes reported in PDB (Benchmark structures: 6HOF, 6HOG, 6UML, 5HXB, 5FQD, 6UE5, 6UD7, 6PAI, 6SJ7, 6QOR, 6QOV, 6QOW, 6TD3,
Slide 44: Proteovant: Positioned To Lead
Well-financed to advance pipeline of protein degraders to the next level of value creation World-class team assembled to drive discovery and development of optimized protein degraders Advancing broad pipeline of protein degraders Long-term, exclusive discovery research partnership in protein degrada
Slide 45: Genevant Business and Technology
GENEVANT Business and Technology
Slide 46: Genevant Overview
Genevant Overview World-class delivery platforms to enable delivery of mRNA, siRNA, gene editing constructs, and other nucleic acids Best-in-class proprietary lipid nanoparticle (LNP) platform and proprietary ligand conjugate platform Focused business of collaboration around delivery expertise and t
Slide 47: Decades of Experience in Nucleic Acid Delivery
Decades of Experience in Nucleic Acid Delivery, Creative Corporate Partnering Peter Lutwyche, PhD Chief Executive Officer and President, GSC Former CTO, Arbutus Biopharma; Head of Pharmaceutical Development, QLT Developed and commercialized VISUDYNE® at QLT Over 20 years experience in nucleic acid-b
Slide 48: Industry-Leading Delivery Capabilities Enable Diverse NA Therapeutics
Industry-Leading Delivery Capabilities Enable Diverse NA Therapeutics LNP Ligand Conjugates mRNA vaccines Infectious Disease Personalized Oncology siRNA/ASO hepatocyte delivery siRNA/ASO other cell types Gene Editing hepatocyte/ other cell types mRNA/DNA (Hepatocyte/other cell types) Therapeutic Pro
Slide 49: LNP Platform
LNP Platform (© GENEVANTSCIENCES CORPORATION. 2021, ALL RIGHTS RESERVED.
Slide 50: Genevant's Lipid Nanoparticle Delivery Platform
Genevant’s Lipid Nanoparticle (LNP) Delivery Platform Multi-component lipid formulations encapsulating nucleic acid payload(s) within a lipid core Limited constraints on NA payload composition, structure or size Stable uniform dispersion of colloidal nanoparticles Efficient intracellular delivery to
Slide 51: Unparalleled Experience Designing Ionizable Lipids
Genevant has Unparalleled Experience Designing Ionizable Lipids In a head-to-head study comparing multiple LNP formulations varying only the ionizable lipid, a newer Genevant formulation outperformed third party formulations Superior potency and avoidance of immune stimulation relative to others, in
Slide 52: Genevant LNP Technology Has Entered the Clinic
Genevant LNP Technology has Entered the Clinic in More than a Dozen Product Candidates Representing Hundreds of Subjects Company Product Indication Activity Latest Phase ONPATTRO (patisiran) ATTR Amyloidosis Safely dosed for up to 25 months in some patients Efficacy of up to 94% TTR knockdown with p
Slide 53: Providence Therapeutics Interim Phase 1 mRNA COVID-19 Vaccine Data
Collaborator Providence Therapeutics Reported Favorable Interim Phase 1 Antibody Data for mRNA-LNP COVID-19 Vaccine* Data from 60 subjects; two doses, 28 days apart Compelling safety data also reported Both expts: (2 IM doses, 3-week interval). Measurements 14 days post boost. Robust Anti-spike Ab T
Slide 54: Hepatic Stellate Cell-Directed LNP Knockdown
Hepatic Stellate Cell (HSC)-Directed LNP: Strong Target-Specific Knockdown Building on track record of success, focusing on access to historically challenging tissues and cell types Hepatic stellate cells well established as central driver of fibrosis In preclinical studies, delivery of siRNA to HSC
Slide 55: Genevant LNP Manufacturing Technology
Genevant LNP Manufacturing Technology Controlled mixing process (not microfluidic or extrusion based) Broadly applicable to nucleic acids including plasmid DNA, siRNA, and mRNA High encapsulation efficiency Rapid, reproducible and robust Easily scalable Modular design is transferable GMP compliant
Slide 56: Ligand Conjugate Platform
Ligand Conjugate Platform
Slide 57: Genevant's siRNA-Ligand Conjugate Delivery Platform
Genevant’s siRNA-Ligand Conjugate Delivery Platform siRNA design stabilizes the conjugate and enhances cellular delivery Linker chemistry increases the Stability, potency, and duration of activity Ligand-targeting moiety mediates binding and internalization of conjugate: 5’ or 3’ coupled e.g., GalNA
Slide 58: siRNA-GalNAc Conjugates Mechanism of Action
siRNA-GalNAc Conjugates Mechanism of Action GalNAc-conjugated ligand mediates siRNA delivery GalNAc binds & internalized by ASGPR (Asialoglycoprotein Receptor); Clears serum glycoproteins via clathrin-mediated endocytosis High capacity uptake system Highly expressed and conserved across species siRN
Slide 59: RNAi 2.0 Enhanced Potency and Duration in NHP
First Generation Compared Favorably to Benchmark, RNAi 2.0 Provided Enhanced Potency and Duration of Effect in NHP RNAi 2.0 contains intrinsic endosomolytic properties Single subcutaneous dose NHP study; all groups used same TTR sequence to allow direct comparison Marked enhancement in potency Compa
Slide 60: Representative Corporate Partnering
Representative Corporate Partnering Co-develop + co-commercialize specified mRNA-LNP rare disease programs; 50-50 profit/cost share; additional LNP licenses for specified oncology target License to LNP for SAM RNA vaccine products for COVID-19 License to LNP for SAM RNA vaccine products for specifie
Slide 61: Dermavant Sciences
| Confidential. For Internal Review Purposes Only. WE WILL DERMATOLOGY Dermavant Sciences
Slide 62: Investment Summary: Poised to Transform Immuno-Dermatology
Investment Summary Poised to TRANSFORM Immuno-Dermatology | For Investor Audiences Only. Transformational 2 in 1 Lead Product Candidate Tapinarof, a novel chemical entity, was shown to have rapid onset and efficacy with clinically meaningful and statistically significant differences observed for all
Slide 63: Tapinarof Targets the Two Largest Markets in Immuno-Dermatology
Tapinarof Targets the Two Largest Markets in Immuno-Dermatology Psoriasis & atopic dermatitis markets projected to reach ~$31B in the US and ~$44B globally by 2026 | For Investor Audiences Only. Evaluate Pharma Data: Global Psoriasis and Atopic Dermatitis Prescription Drugs Market and Forecast 2019
Slide 64: Tapinarof: Transformational 2-in-1 Lead Product Candidate
Tapinarof Offers a Transformational 2-in-1 Lead Product Candidate Novel MOA delivering a unique & differentiated target product profile Chronic, inflammatory disease characterized by red patches & plaques with silvery scales on skin Large, mostly adult population (~8M people in U.S.)1 1 2 Limited to
Slide 65: Track Record in Developing Dermatology Products
Track Record of Success in Developing & Commercializing Innovative Dermatology Products at Multiple Companies | For Investor Audiences Only. All trademarks are property of their respective owners. Todd Zavodnick Chief Executive Officer Phil Brown MD, JD Chief Medical Officer Chris Chapman Chief Comm
Slide 66: Innovative Immuno-Dermatology Pipeline with Global Rights
Innovative Immuno-Dermatology Pipeline with Global Rights1 | For Investor Audiences Only. 1. Excludes certain Asian territories. Product candidate indication STAGE OF DEVELOPMENT Key milestone Preclinical Phase 1 Phase 2 Phase 3 Clinical Stage Development Programs TAPINAROF (DMVT-505) A topical ther
Slide 67: Tapinarof Cream Psoriasis Program
1For Internal Discussion Purposes Only TAPINAROF CREAM PSORIASIS PROGRAM Phil Brown MD, JD, Chief Medical Officer
Slide 68: Tapinarof: Novel Multi-Modal Mechanism of Action
Tapinarof: Novel Multi-Modal Mechanism of Action Inhibits inflammatory cytokines, promotes epidermal barrier restoration & decreases oxidative stress | For Investor Audiences Only. *Demonstrated in vitro. †Demonstrated ex vivo. ‡Demonstrated in mice models. AhR, aryl hydrocarbon receptor; Nrf2, nucl
Slide 69: PSOARING Program Executive Summary
PSOARING Program – Executive Summary Novel & differentiated attributes observed – NDA filed; PDUFA action expected in 2Q 2022 PSOARING 1 and PSOARING 2 PGA primary endpoints met (P<0.0001) and PASI75 secondary endpoint met In interim analysis (IA) of PSOARING 3 LTE data, 57.3% of patients achieved P
Slide 70: PSOARING Program Study Design
Phase 3 PSOARING Program – Study Design Over 1,000 patients enrolled in two identically-designed pivotal trials followed by long-term open-label extension study | For Investor Audiences Only. *Patients with PGA of 2 (mild) and PGA of 4 (severe) limited to ~10% each of the total randomized population
Slide 71: PSOARING Primary & Secondary Efficacy Results
PSOARING 1 & 2 Pivotal Program – Primary & Secondary Efficacy Results Primary efficacy endpoint met, as demonstrated by magnitude of PGA treatment success* & †PASI75 | For Investor Audiences Only. *PGA score of 0 (clear) or 1 (almost clear) and ≥2-grade improvement from baseline. †PASI75 response: ≥
Slide 72: PSOARING LTE: ~40% Achieved PGA 0
PSOARING 3 LTE Study – ~40% of Tapinarof Treated Patients Achieved PGA 0 PGA of 0 corresponds to complete disease clearance | For Investor Audiences Only. Overall (n=763) Patients who Entered LTE Trial on Tapinarof 1% QD & Continued on Tapinarof 1% QD (n=508) Patients who Entered LTE Trial on Vehicl
Slide 73: Rapid Onset of Action
| For Investor Audiences Only. Mean Change in PASI from Baseline to Week 12 (ITT, MI)* Phase 3 PSOARING Program – Rapid Onset of Action Statistically significant PASI improvement as early as Week 2 Exploratory Endpoint Achieved Rapid onset of activity with 20% reduction in disease activity by Week 2
Slide 74: Rapid Peak Pruritus Improvement
| For Investor Audiences Only. Minimum 4-point Improvement in Peak Pruritus NRS from Baseline to Week 12 (ITT, OC)* Phase 3 PSOARING Program – Rapid Peak Pruritis Improvement NRS of at least a 4-point reduction achieved as early as Week 2 Exploratory Endpoint Achieved Mean baseline peak NRS was 5.7
Slide 75: Tapinarof Achieves Primary Endpoint: Complete Clearance
Phase 3 PSOARING Program – Tapinarof Achieves Primary Endpoint Rapid & complete clearance of psoriasis in patient achieving primary endpoint | For Investor Audiences Only. BASELINE PGA = 3 PASI = 17.6 PGA = 0 PASI = 0 WEEK 4 WEEK 12 PGA = 2 PASI = 4 Results shown for one patient are not necessarily
Slide 76: Lower Extremity Disease Rapid Response
Phase 3 PSOARING Program – Tapinarof Achieves Primary Endpoint Lower extremity disease: rapid response in patient achieving primary endpoint | For Investor Audiences Only. Results shown for one patient are not necessarily indicative of results for other patients, additional trials or other uses BASE
Slide 77: Clinical Improvement in Patient Not Achieving Endpoint
Phase 3 PSOARING Program – Tapinarof Clinical Improvement Clinical improvement in a patient not achieving regulatory endpoint | For Investor Audiences Only. Results shown for one patient are not necessarily indicative of results for other patients, additional trials or other uses BASELINE PGA = 3 PA
Slide 78: Clear or Almost Clear for ~4 Months Off Treatment
PSOARING 3 – Clear or Almost Clear for ~4 months Off Treatment Remittive (off-therapy) effect observed among patients entering with PGA=0 | For Investor Audiences Only. Key Points Overall, median time to PGA ≥ 2 was 115 days (95% CI = 85 to 162 days, approximately 3 to 5 months) CI, confidence inter
Slide 79: Most Common Treatment-Related TEAEs
Phase 3 PSOARING Studies: Most Common Treatment-Related TEAEs ≥ 1% Consistent & predictable safety profile observed | For Investor Audiences Only. Count reflects numbers of patients reporting one or more AEs that map to grouped MedDRA preferred term. A patient is counted once only for each type of A
Slide 80: LTE Investigator-Assessed Irritation
PSOARING 3 LTE Study – Investigator-Assessed Irritation Favorable tolerability without regard to site of application or duration of use | For Investor Audiences Only. Investigator Assessed Irritation Scores* Across All Application Sites (Safety population) Tapinarof 1% QD (Pivotal) (n=508) Overall (
Slide 81: Tapinarof Cream Atopic Dermatitis Program
1For Internal Discussion Purposes Only TAPINAROF CREAM ATOPIC DERMATITIS PROGRAM
Slide 82: Atopic Dermatitis Phase 2b Efficacy Results
Tapinarof Atopic Dermatitis Phase 2b Trial – Efficacy Results Response rates: 49% of patients achieved IGA clear or almost clear and ≥2-grade improvement and 51% of patients achieved EASI75 after 8 weeks of treatment with tapinarof 1% QD | For Investor Audiences Only. Week 8 IGA score 0 or 1 and ≥2-
Slide 83: ADORING Program Study Design
Phase 3 ADORING Program – Study Design Two identically-designed pivotal trials followed by long-term, open-label extension | For Investor Audiences Only. Primary endpoint: Proportion of subjects who have a vIGA-ADTM 0 or 1 Baseline at Week 8 Secondary endpoints: Proportion of subjects with EASI 75 @
Slide 84: Commercial Overview
1For Internal Discussion Purposes Only COMMERCIAL OVERVIEW Chris Chapman, Chief Commercial Officer
Slide 85: Lack of Topical Innovation Offers Tapinarof Opportunity
Lack of Topical Innovation Offers Tapinarof Unprecedented Opportunity Establishing a potential new topical benchmark in psoriasis & atopic dermatitis | For Investor Audiences Only. Systemic Innovation Late-Stage Pipeline 2 topicals 4 targeted systemics Late-Stage Pipeline 3 topicals 7 targeted syste
Slide 86: U.S. Psoriasis Market Highly Concentrated
U.S. Psoriasis Market Highly Concentrated & Readily Accessible 80% of market value concentrated in tight payer, prescriber & geographic clusters | For Investor Audiences Only. National Payers Geography Prescribers 80% of TRx claims are processed by 4 national PBMS/Plans* 80% of Derm Commercial TRx v
Slide 87: Extensive Market Research Supports Early Access
Extensive Market Research Supports Early Access & Adoption More than 500 HCPs, >300 patients, & 65 payer interviews (~200M+ covered lives) | For Investor Audiences Only. *Due to the anonymity of participants across payer organizations, there is likely overlap of participants and plans (i.e., these a
Slide 88: 94% of HCPs Believe Tapinarof Addresses Unmet Need
94% of HCPs Believe Tapinarof Can Address an Unmet Need in Psoriasis Prescribers have a CLEAR & PRESENT understanding of the limitations of the current standard of care, TCS | For Investor Audiences Only. Source: Interviews conducted by Triangle Insights, November/December 2020 & January 2021 with d
Slide 89: Payer & Prescriber Interests Aligned
Payer & Prescriber Interests Aligned in the Need for Topical Innovation Tapinarof could offer payers the opportunity to manage spend with INNOVATION vs. RESTRICTION | For Investor Audiences Only. Source: N=17 payer interviews conducted by Triangle Insights Group, March & April 2021 Quotes from Payer
Slide 90: Key Attributes Set New Benchmark for Topical Innovation
Key Attributes Have Potential To Set New Benchmark for Topical Innovation | For Investor Audiences Only. Source: Qualitative market research conducted by Triangle Insights, January 2021 with dermatologists (n = 50) 1. What aspect of this product do you find most attractive? Overall Efficacy 1 7 Not
Slide 91: Prescriber, Payer & Patient Benefits Aligned
Tapinarof’s Prescriber, Payer & Patient Benefits are Uniquely Aligned Versatility may fundamentally change the psoriasis treatment paradigm | For Investor Audiences Only. Mild Disease <3% BSA Moderate Disease 3-10% BSA Severe Disease >10% BSA Chronic Monotherapy “Breaking The TCS Flare Cycle/ Changi
Slide 92: Finance, IP and Summary
1For Internal Discussion Purposes Only FINANCE, IP AND SUMMARY
Slide 93: Strategic Partnerships with GSK & Thermo Fisher
Strategic Partnerships with GSK & Thermo Fisher Support Global Supply Agreements ensure a high quality & predictable supply of drug substance and drug product | For Investor Audiences Only. Drug Product Manufacturing Barnard Castle, UK Drug Substance Manufacturing Cork, Ireland Clinical Manufacturin
Slide 94: Tapinarof IP Summary: Patent Protection Until at Least 2036
Tapinarof IP Summary: Patent Protection Until at Least 2036 | For Investor Audiences Only. * The remittive effect encourages payers to consider innovative contracts that cap annual refills or involve risk-sharing for the patient outcomes. Source: N=17 payer interviews conducted by Triangle Insights
Slide 95: Summary
Summary | For Investor Audiences Only. Dermavant PSOARING Phase 3 psoriasis program completed; PDUFA action expected 2Q 2022; Comprehensive commercial launch planning underway Dermavant poised to TRANSFORM Immuno-Dermatology $44.3B global opportunity across the two largest immuno-dermatology segment
Slide 96: Appendix
1For Internal Discussion Purposes Only Appendix
Slide 97: Folliculitis Examples From PSOARING
Folliculitis Examples From PSOARING 1 & 2 Most treatment-related events are localized to site of application & mild to moderate in nature | For Investor Audiences Only. Examples of two representative target lesions of two tapinarof treated patients from the PSOARING 1 and 2 clinical trials. DMVT-505
Slide 98: Roivant R&D Day
Roivant R&D Day Investor Presentation September 28, 2021
Slide 99: Forward-looking statements
Forward-looking statements This presentation contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as “may,” “might,” “will,” “expect,” “plan,” “anticipate
Slide 100: Rethinking Possibilities in Autoimmune Disease
Rethinking possibilities in autoimmune disease Love Trailblazing All Voices Bolder Faster Our vision: Normal lives for people with autoimmune diseases
Slide 101: Anti-FcRn Market: Potential Benefit Across Wide Range of Indications
Anti-FcRn Market: Potential therapeutic benefit across wide range of indications Fifteen indications announced by at least one anti-FcRn program ENDOCRINOLOGY Thyroid eye disease RHEUMATOLOGY Primary Sjögrens Syndrome Lupus Nephritis Systemic lupus erythematosus Rheumatoid arthritis DERMATOLOGY Bull
Slide 102: Even Well Controlled MG Patients Report Lifestyle Accommodations
Source: Analysis – Social Media Review MG n=975 / Qualitative research – MG patient journey n=28 / MG Patient Advisory Council n=6 / MG Patient Quantitative Survey (n=50) Even well controlled patients report lifestyle accommodations Anxiety around response and duration Significant impact on quality
Slide 103: Dosing Approach Preferences
Dosing approach preferences Source: Analysis – MG Patient Quantitative Survey (n=50) 94% of respondents with Myasthenia Gravis prefer chronic versus intermittent dosing Patient Survey Feedback: Preferred Treatment Approach
Slide 104: Batoclimab's Differentiated Attributes
Batoclimab’s (IMVT-1401) differentiated attributes provide a unique opportunity to address patients’ unmet needs Subcutaneous route of administration: Designed and developed for simple subcutaneous injection to provide human-centric, give and go dosing experience Significant impact on quality of lif
Slide 105: Developing Gene Therapies for Rare Diseases
Developing Gene Therapies for Rare Diseases
Slide 106: Aruvant: Growth Opportunity in Potentially Curative Gene Therapies
Aruvant represents a growth opportunity developing potentially curative gene therapies for rare diseases All product candidates are investigational and subject to regulatory approval. Ongoing Phase 1/2 trial Clinical data demonstrating curative potential Up to 100% reduction in vaso-occlusive events
Slide 107: Experienced Team in Gene Therapy
Experienced team in gene therapy, clinical development and manufacturing All trademarks are the property of their respective owners. Global Technical Operations Head 25 years technical ops leadership Multiple gene therapy development programs Successful rare disease approvals Novel assay development
Slide 108: Vaso-Occlusive Events Are Painful and Costly
The major complication of sickle cell disease are vaso-occlusive events (VOEs) which are painful and costly 1. Ballas SK, Lusardi M. Am J Hematol. 2005;79(1):17-25; 2. Piel FB, Steinberg MH, Rees DC. N Engl J Med. 2017;376(16):1561-1573; 3. Elmariah H, Garrett ME, De Castro LM, et al. Am J Hematol.
Slide 109: ARU-1801: One-Time Potentially Curative Therapy for SCD
ARU-1801 is a one-time potentially curative therapy for SCD with a differentiated toxicity profile. Uses self-inactivating lentiviral vector that contains a proprietary γ-globin gene for a novel, highly potent variant of fetal hemoglobin (HbF): HbFG16D Unique potency allows ARU-1801 to engraft with
Slide 110: ARU-1801 Uses a Patient's Own Stem Cells
ARU-1801 uses a patient’s own stem cells to make a unique fetal hemoglobin Produce Lentiviral Vector Collect CD34+ Stem Cells Transduce CD34+ Cells Ex Vivo Infuse Cells after a Single Dose of RIC 01 02 03 04
Slide 111: ARU-1801's Unique Attributes
ARU-1801’s unique attributes enable a potential differentiated product profile All product candidates are investigational and subject to regulatory approval. More hemoglobin F per vector copy Proprietary G16D point mutation drives higher HbF payload per vector copy High HbFG16D potency HbFG16D may h
Slide 112: RIC Benefits for Patients, Providers, and Payors
RIC has potential benefits for patients, providers, and payors Table reflects combination of gene therapy protocols, reported results from gene therapy trials, and literature on the use of these conditioning agents in other settings. *Dose adjusted to a targeted AUC for busulfan of 4200 μM*min. 1. b
Slide 113: 2021 Events Reinforce Importance of Safety in Gene Therapy
Recent events in 2021 reinforce importance of safety in GTx, an area where ARU-1801 is uniquely differentiated Vector was very unlikely a factor in either AML/MDS case1,2 Busulfan conditioning and baseline risk identified as possible causes1,2 Tisdale Blood Advances 2020 Bluebird bio press release a
Slide 114: Pre-Treatment VOEs Despite SOC Treatment
Before treatment with ARU-1801, patients had numerous VOEs despite SOC treatment -24 -12 0 +12 +24 Pt 1 Pt 2 Pt 3 Pt 4 Pre-treatment VOEs Pre-treatment Patient Management No VOEs observed 12 mo f/u Months before treatment Vaso-occlusive event Begin process development All product candidates are inve
Slide 115: Significant Improvement in VOEs
Significant improvement to date in VOEs including 100% resolution in recently treated patients -24 -12 0 +12 +24 Pt 1 Pt 2 Pt 3 Pt 4 93% 100% 100% 85% VOE Reduction Pre-treatment VOEs Post-treatment VOEs No VOEs observed 18 mo f/u No VOEs observed 12 mo f/u Months before/after treatment Vaso-occlusi
Slide 116: GTx with RIC Grows Addressable Population
Market research showed GTx with RIC grows addressable population and is favored choice vs myeloablative options Entry of ARU-1801 increases patients eligible for GTx Pts eligible for GTx Pts eligible for GTx Myeloablative GTx only market Myeloablative GTx + ARU-1801 market Results from market resear
Slide 117: ARU-1801 Path Forward
ARU-1801 Path Forward First patient dosed with updated manufacturing process ARU-1801 Ph 1/2 data Initiate ARU-1801 pivotal study H2 H2 H1:23 ü ü
Slide 118: ARU-2801 for Hypophosphatasia
ARU-2801 for Hypophosphatasia Data presented in All trademarks are the property of their respective owners.
Slide 119: ARU-2801 One-Time Gene Therapy for HPP
ARU-2801 is a one-time gene therapy for hypophosphatasia (HPP) that could replace a potential blockbuster enzyme replacement therapy (ERT) SOC and deliver potentially curative efficacy, without the limitations of chronic administration ARU-2801 is an adeno-associated virus gene therapy designed to t
Slide 120: Hypophosphatasia Is a Devastating Orphan Disorder
J Pediatr. 2019 Jun;209:116-124.e4. doi: 10.1016/j.jpeds.2019.01.049. Epub 2019 Apr 9. Fraser D: Hypophosphatasia. Am J Med. 1957, 22: 730-46. 10.1016/0002-9343(57)90124-9. Ann Hum Genet . 2011 May;75(3):439-45. doi: 10.1111/j.1469-1809.2011.00642.x. Epub 2011 Mar 24 Hypophosphatasia (HPP) is a deva
Slide 121: Chronic Injections with Standard of Care ERT
1. Strensiq FDA Label 2. Strensiq EPAR Product Information Drugs are investigational and subject to regulatory approval. SOC ERT for HPP, Strensiq, requires chronic administration and patients experience injection site reactions leaving high unmet need ARU-2801 can potentially eliminate these inhere
Slide 122: ARU-2801 Results in High, Durable ALP Levels and Survival
In HPP murine models, ARU-2801 treatment results in high, durable ALP levels and survival to 18 months (vs 3 weeks untreated) All drugs are investigational and subject to health authority approval. Matsumoto T et alt Molecular Therapy Methods and Clinical Development 2021 High ALP levels in HPP mode
Slide 123: Targeting Rare Autoimmune Diseases
Targeting Rare Autoimmune Diseases For Investor Audiences Only
Slide 124: Introduction
Introduction We are developing an anti-GM-CSF monoclonal antibody (namilumab) for sarcoidosis and potentially other autoimmune rare diseases Sarcoidosis is a systemic, multi-organ disease that results from a dysregulated immune response, with poor treatment options Our goal is to significantly impro
Slide 125: Sarcoidosis – Rare Autoimmune Disease
Baughman, et al. Annals ATS 2016 Denning, et al. European Respiratory Journal 2013 3. Sauer, et al. Annals ATS 2017; Baughman, et al. Sarcoidosis Vasculitis and Diffuse Lung Diseases 1997 Characterized by the accumulation of granulomas in organs and lymph nodes, believed to be due to an exaggerated
Slide 126: Inadequate Treatment Options for Sarcoidosis
Goal of therapy is to prevent or control organ damage, relieve symptoms and improve patients’ quality of life Corticosteroids are first-line therapy, but have significant side effects with long-term dosing Immunosuppressive therapy (methotrexate, azathioprine) and biologics (TNF inhibitors) are ster
Slide 127: GM-CSF is a Key Pathogenic Cytokine in Sarcoidosis
GM-CSF is a pro-inflammatory cytokine that plays a key role in the promotion and maintenance of sarcoid granuloma inflammation1 GM-CSF activates macrophages and other immune cells, which drive inflammation and tissue damage2 Upregulated GM-CSF forms granulomas in sarcoidosis in vitro models3 GM-CSF
Slide 128: Namilumab: Fully Human Anti-GM-CSF Antibody
Namilumab is a fully human anti-GM-CSF monoclonal antibody with broad potential in autoimmune diseases In multiple Phase 2 studies, anti-GM-CSFs have been well-tolerated and have demonstrated the potential to improve symptoms in autoimmune diseases including rheumatoid arthritis and giant cell arter
Slide 129: Limited Pipeline Competition for Pulmonary Sarcoidosis
Limited Pipeline Competition for Pulmonary Sarcoidosis Candidate Sponsor Mechanism of Action Development Phase Phase 1/2 Phase 2 ATYR1923 aTyr NRP2 modulator Completed CMK389 Novartis IL-18 Antibody Initiated Inhaled VIP Relief Immunosuppressant Announced Sources: clinicaltrials.gov; aTyr Pharma pre
Slide 130: Summary
Summary Sarcoidosis is a significant unmet clinical need Preclinical studies and patient samples indicate GM-CSF likely contributes to the pathogenesis of sarcoidosis Namilumab has the potential to significantly improve the treatment of sarcoidosis We are building a world class team with drug develo
Slide 131: Roivant Sciences
ROIVANT SCIENCES