Aclaris Therapeutics, Inc. · NASDAQ:ACRS
Aclaris Therapeutics — Investor Presentation (January 2018)
49 slides from Aclaris Therapeutics's investor presentation, Jan 8, 2018
Slide 1: Company Overview
Company Overview Dr. Neal Walker President and CEO January 2018 © Copyright 2018 Aclaris Therapeutics. All rights reserved. A-1 Illuminating Science Empowering Patients
Slide 2: Cautionary Note Regarding Forward-Looking Statements
Any statements contained in this presentation that do not describe historical facts may constitute forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements may be identified by words such as "believe", "expect", "may", "plan," "pote
Slide 3: Our Corporate Strategy: Building a Fully-Integrated Biopharmaceutical Company
APPLY UNIQUE LEADERSHIP INSIGHTS Our Corporate Strategy: Building a Fully-Integrated Biopharmaceutical Company A-101 A-101 45% Common Warts Initiated Phase 2 clinical trials June 2017 Immunology Portfolio ATI-50001/ATI-50002 (JAK 1/3 Inhibitors) Alopecia Areata Vitiligo ATI-450 (MK-2 inhibitor) Psor
Slide 4: Pipeline - Diversified Aesthetic and Medical Dermatology / Immunology Portfolio
4 Pipeline - Diversified Aesthetic and Medical Dermatology / Immunology Portfolio Program Indication(s) Discovery Pre-Clinical Phase 1 Phase 2 Phase 3 A-101(45%)Topical Common Warts ATI-50002 Topical Alopecia Areata ATI-50002 Topical Vitiligo ATI-50001 Oral Alopecia Areata “Soft” JAK Inhibitor Topic
Slide 5: Aesthetic and dermatology market growth
...and non-surgical procedures increased by 650% from 1997-20163 Global aesthetic market expected to reach $13.29 billion by 2021, growing at CAGR of 10.8%2 Global dermatology market valued at $20 billion in 2015, growing at CAGR of 7.7%1 “The types of procedures being performed is changing. Most of
Slide 6: Patient populations and treatment limitations
22+MM people in U.S.1; current treatments show only modest therapeutic effect and have significant limitations2,3 ANDROGENETIC ALOPECIA (MALE / FEMALE PATTERN HAIR LOSS) VITILIGO 1-2% global population impacted6 no FDA-approved medication to repigment the skin7 1 Nguyen et al, Laser Treatment of Non
Slide 7: ESKATA: First FDA-Approved Treatment for Raised SK
8 8 ESKATA: First FDA-Approved Treatment for Raised SK (hydrogen peroxide) topical solution, 40% (w/w) NDC: 71180-001-01 One Unit Rx Only Store at 20-25oC Single use applicator/0.7 mL. For Topical Use Only.
Slide 8: Favorable Market Dynamics
83+MM People in the US with SK1 18+MM visits to Derm for SK2 8+MM SK treatments2 83% of SK patients were interested in treatment and willing to pay out-of-pocket3 1 Bickers et al, The Burden of Skin Disease, J Am Acad Dermatology, 2006;55:490-500. 2 Data on File: Aclaris Therapeutics Burke Screener
Slide 9: ESKATA Positioning: Face and Neck
Body Locations Where Patients Would Use ESKATA (n=201) Market research demonstrates ESKATA’s value proposition is strongest for facial and neck lesions Patients are most adverse to scarring on the face and neck Patients prefer a topical treatment over an invasive procedure for SKs on the face and ne
Slide 10: US Patient Opportunity for ESKATA: Face Only
83+MM People in the US with SK1 17+MM of these Patients with Facial SKs2 44+MM of these Patients with Income to Spend on Cosmetic Treatments2 ESKATA Patient Funnel 5+MM of Patients Willing to Pay for ESKATA2 35+MM of these Patients Visiting or Interested in Visiting Relevant Providers2 10+MM of thes
Slide 11: Non-surgical Procedures: Quick Facts
Non-surgical Procedures: Quick Facts Physician/Surgeon Fees* Length of Procedure Number of Treatments Nonsurgical Procedures Best Candidate Back to Work Repeat treatments 4-6 months (onset of actions in 1-5 days) Botulinum Toxln (Botox, Dyspoi, Xeonin) Frown Ones, craw's feet $376 30 minutes No down
Slide 12: Territory Alignment
Territory Alignment Sales Director 1 Strategic Accounts 1 Sales Managers 6 Sales Reps 50 Inside Sales 2 HAWAII 13 > 90% possess five or more years of dermatology experience > 85% are multi-year President’s Club winners > 70% have previous buy and bill experience SEATTLE, WA 1.20 SACRAMENTO, CA 1.09
Slide 13: Sales Force Activity
Q1 Q2 Sales Force Training Market Readiness Activities AAD National Sales Meeting 14 Sales Force Activity: Approval to Launch Sales Force Training Intro/Reconnect to Target Accounts Establish ESKATA Centers of Excellence: Train Target Providers Schedule In-Service and/or Peer-to-Peer Programs Levera
Slide 14: ESKATA Campaign Proposed Timeline
1H 2018 2H 2018 ESKATA Campaign Proposed Timeline Now Approved! Ads Account Intro Early Experience Speaker Programs Now Approved! @ AAD San Diego, Feb 16-20 ESKATA HCP Campaign Key Conferences HCP Advertising Consumer PR Campaign Direct-to-Consumer Advertising Sampling Sales Force Promotion Promotio
Slide 15: A-101 45% Topical Solution Candidate For Common Warts
A-101 45% Topical Solution Candidate For Common Warts
Slide 16: Existing Patient Base Offers Significant Market Potential
1 IMS National Disease and Therapeutic Index 2016. 2 Bruggnik et al, Natural Course of Cutaneous Warts Among Primary Schoolchildren: A Prospective Cohort Study 2013, Annals of Family Medicine;11:5,2013;437-441. 3 Lipke M., An Armamentarium of Wart Treatments, Clinical Medicine & Research,4:4, 2006;
Slide 17: Summary of WART-202/203 Phase 2 Trial Results
Primary Endpoint: Mean change from baseline in the Physician’s Wart Assessment (PWA) score on target wart at day 56 (visit 10) using an analysis of covariance. Secondary Endpoints: The percentage of all treated warts that were clear at day 56 The percentage of all treated warts that were clear or ne
Slide 18: WART-203: Mean Change from Baseline in PWA Score
19 WART-203: Mean Change from Baseline in PWA Score on Target Wart at Day 56 * p < 0.05 ** p < 0.01 *** p < 0.001 0 -0.1 -0.2 -0.3 -0.4 -0.5 -0.6 -0.7 -0.8 -0.9 -1 Baseline Day 7 Day 14 Day 21 Day 28 Day 35 Day 42 Day 49 Day 56 0 -0.04-0.05 -0.14 -0.14 -0.17 -0.18 -0.15 -0.17 -0.22 -0.32* -0.58*** -
Slide 19: WART-203: Percentage of Treated Warts Clear
20 WART-203: The Percentage of All Treated Warts that are Clear on the PWA for Each Post-baseline Visit * p < 0.05 ** p < 0.01 *** p < 0.001 35.00% 30.00% 25.00% 20.00% 15.00% 10.00% 5.00% 0.00% Day7 Day 14 Day 21 Day 28 Day 25 Day 42 Day 49 Day 56 0.00% 0.71% 1.42% 2.13% 2.88% 5.67% 8.51% 9.22% 0.7
Slide 20: WART-203: Warts Clear or Near-Clear
7.09% 17.36% 22.15% 35.81%*** 42.95%*** 47.92%*** 45.21%*** 45.64%*** 3.60% 8.51% 11.35% 14.89% 12.95% 18.44% 16.31% 15.60% 0.00% 10.00% 20.00% 30.00% 40.00% 50.00% 60.00% Day 7 Day 14 Day 21 Day 28 Day 35 Day 42 Day 49 Day 56 A-101 45% Vehicle 21 WART-203: The Percentage of All Treated Warts that a
Slide 21: WART-203: Responder Analysis
0.00% 2.60% 5.06% 8.97%** 12.66%** 18.18%** 23.08%*** 25.32%*** 0.00% 0.00% 0.00% 0.00% 1.30% 2.56% 2.56% 2.56% 0.00% 5.00% 10.00% 15.00% 20.00% 25.00% 30.00% Day 7 Day 14 Day 21 Day 28 Day 35 Day 42 Day 49 Day 56 A-101 45% Vehicle 22 WART-203: Responder Analysis: Proportion of Subjects Achieving Ta
Slide 22: WART-203: Subjects with All Treated Warts Clear
0.00% 1.30% 2.53% 6.41%* 6.33% 11.69%* 15.38%** 18.99%*** 0.00% 0.00% 0.00% 0.00% 1.30% 2.56% 2.56% 2.56% 0.00% 2.00% 4.00% 6.00% 8.00% 10.00% 12.00% 14.00% 16.00% 18.00% 20.00% Day 7 Day 14 Day 21 Day 28 Day 35 Day 42 Day 49 Day 56 A-101 45% Vehicle 23 WART-203: Proportion of Subjects with all trea
Slide 23: WART-202: Mean Change from Baseline in PWA Score
0 -0.09 -0.26* -0.37** -0.39** -0.49** -0.55*** -0.69*** -0.77*** -0.03 -0.10 -0.14 -0.16 -0.21 -0.13 -0.21 -0.23 -0.9 -0.8 -0.7 -0.6 -0.5 -0.4 -0.3 -0.2 -0.1 0 Baseline Day 7 Day 14 Day 21 Day 28 Day 35 Day 42 Day 49 Day 56 A-101 45% Vehicle 24 WART-202: Mean Change from Baseline in PWA Score on Ta
Slide 24: WART-202: Percentage of Treated Warts Clear
2.94% 5.00% 7.84%* 8.57%* 11.65% 12.62% 14.85%** 20.75%*** 0.00% 0.00% 0.99% 1.08% 3.00% 2.04% 2.06% 2.94% 0.00% 5.00% 10.00% 15.00% 20.00% 25.00% Day 7 Day 14 Day 21 Day 28 Day 35 Day 42 Day 49 Day 56 A-101 45% Vehicle 25 WART-202: The Percentage of All Treated Warts that are Clear on the PWA for E
Slide 25: WART-202: Warts Clear or Near-Clear
8.82% 19.00%* 29.41%** 28.57%* 37.86%* 41.75%** 47.52%** 52.83%*** 3.00% 6.12% 8.91% 11.83% 15.00% 13.27% 14.43% 13.73% 0.00% 10.00% 20.00% 30.00% 40.00% 50.00% 60.00% Day 7 Day 14 Day 21 Day 28 Day 35 Day 42 Day 49 Day 56 A-101 45% Vehicle 26 WART-202: The Percentage of All Treated Warts that are C
Slide 26: WART-202: Responder Analysis
0.00% 2.94% 2.94% 2.90% 5.88% 5.97% 8.82%* 15.71%** 0.00% 0.00% 0.00% 0.00% 1.41% 0.00% 0.00% 1.37% 0.00% 2.00% 4.00% 6.00% 8.00% 10.00% 12.00% 14.00% 16.00% 18.00% Day 7 Day 14 Day 21 Day 28 Day 35 Day 42 Day 49 Day 56 A-101 45% Vehicle *p <0.05 27 **p <0.01 WART-202: Responder Analysis: Proportion
Slide 27: WART-202: Subjects with All Treated Warts Clear
0.00% 2.94% 2.94% 2.90% 4.41% 4.48%* 7.35%* 11.43%* 0.00% 0.00% 0.00% 0.00% 1.41% 0.00% 0.00% 1.37% 0.00% 2.00% 4.00% 6.00% 8.00% 10.00% 12.00% Day 7 Day 14 Day 21 Day 28 Day 35 Day 42 Day 49 Day 56 A-101 45% Vehicle * p < 0.05 28 WART-202:Proportion of Subjects with all treated Wart(s) (1-4) Clear,
Slide 28: Core Intellectual Property: A-101
Issued US Patent # 7,381,427 Directed to high concentration H2O2 methods of use for treating/removing SK. Orange Book listed for SK indication - estimated expiry date w/ potential PTE of 2½ yrs ~ 2025. Issued US Patent # 9,675,639 Encompasses Formulations / MOU / Applicators for A-101 ~ total of 70
Slide 29: JAK Inhibitor Candidates
JAK Inhibitor Candidates
Slide 30: ATI-50001/ATI-50002: JAK Inhibitors in Alopecia Areata, Vitiligo and AGA
ATI-50001/ATI-50002: JAK Inhibitors in Alopecia Areata, Vitiligo and Androgenetic Alopecia ATI-50001 (oral) and ATI-50002 (topical) – Selective JAK 1/3 inhibitor Additional topical JAK inhibitors in development Oral and topical rights Known MOA and biological response in humans Promoted hair regrowt
Slide 31: Alopecia Areata (AA)
6.8+ million people in the U.S. have had or will develop AA during their lives 25-50% of patients have persistent patchy AA 14-25% of patients progress to alopecia totalis or universalis AA is an autoimmune condition which ranges in severity: Patchy Alopecia Areata – patchy hair loss on scalp Alopec
Slide 32: Mechanism of JAK Inhibitors in Alopecia
Divito & Kupper, Nature Medicine 20, 989–990 (2014). 33 Mechanism of JAK Inhibitors in Alopecia HF of an AA patient control individual Hair follicle bulb (anagen phase) CD8+ NKG2D+ T cells T cell Proliferation Nucleus Follicular epithelial cell P STAT1 MHC class I +peptide Ruxolitinib Tofacitinib JA
Slide 33: Ruxolitinib and Tofacitinib in Alopecia Areata
Baseline Week 24 RUXO - Baseline SALT 64%. Duration of hair loss 12 years. 6 months 20mg BID. Last SALT 1% 34 TOFA - Baseline SALT 100%, 5 months 5mg BID, 2 months 10/5 mg BID, 3 months 10mg BID ongoing. Last SALT 39%. TOFA - Baseline SALT 46%, 5 months at 5mg BID, 2 months at 10/5mg, 2 months 10mg
Slide 34: ATI-50001: Prevents and Reverses Alopecia Areata in Mice
Control ATI-50001 chow ATI-50001 chow: 0.5g ATI-50001 disodium/kg diet N=10 N=10 ATI-50001 reverses AA ATI-50001 Prevents AA #1 Baseline 6WK #2 Baseline 6WK Baseline 6WK ATI-50001 chow Control N=4 N=4 35 Baseline 6WK #1 #2 ATI-50001: Prevents and Reverses Alopecia Areata in Mice
Slide 35: ATI-50002: Topical JAK Inhibitors are Effective In Vivo
Topical JAK Inhibitors Reversed AA in Mouse Model* Baseline Vehicle Control 7 weeks Topically Treated Baseline 7 weeks *Christiano Laboratory, Columbia University 36 ATI-50002: Topical JAK Inhibitors are Effective In Vivo
Slide 36: ATI-50001 Targets IFN-γ producing CD8+ Cytotoxic T cells
IFN-γ IL-17 IFN-γ CD4 CD4 CD8 Untreated AA AA treated w/ ATI-50001 Skin Draining Lymph Nodes Skin Major IFN-γ producing T cells are CD8+ T cells in AA mice. ATI-50001 targets IFN-γ producing CD8+T cells. 37 CD8 Untreated AA AA treated w/ ATI-50001 ATI-50001 Targets IFN-γ producing CD8+ Cytotoxic T c
Slide 37: Additional Potential Indications - Vitiligo
Vitiligo is a common autoimmune disease where melanin (pigment) is absent, causing lighter patches of skin to appear on various parts of the body1,2 Vitiligo impacts 1-2% of the global population irrespective of sex, race or age3 Disease onset occurs in about one-half of sufferers between the ages o
Slide 38: Additional Potential Indications – AGA
Androgenetic alopecia (male/female pattern hair loss) AGA, a genetic disorder, is the most common cause of hair loss1 Experienced by 70% of men and 40% of women at some point in their lives.1 In 2012, 35 million men and 21 million women suffered hair loss.2 Sufferers are highly motivated to seek tre
Slide 39: JAK IP estate
40 US & Global JAK IP estate consisting of >150 patents/applications (issued and/or pending) Exclusive license with Rigel Pharmaceuticals for ATI-50001 & ATI-50002 (COM) in dermatology US Natural expiry dates 2030-2034 + potential applicable PTE Corresponding patents & applications in 18 additional
Slide 40: Aclaris – The Next Chapter
Co-inventors of tofacitinib and former leaders of Pfizer kinase program (including JAK inhibitors) Kinome experts - chemists and biologists; combined 300+ years of drug discovery experience Significant experience in small molecule drug discovery through Phase II People Platform Assets JAK inhibitors
Slide 41: Early Stage Pipeline
ITK Inhibitors “Oral Anti-IL17” Atopic Dermatitis, Psoriasis Oral and soft topical formulation Alopecia Areata, Vitiligo, AGA, Inflammatory Disorders Highly selective, covalent and non-covalent. Oral and soft topical formulation JAK Inhibitors Psoriasis / Psoriatic Arthritis, RA, CAPS, Chronic Infla
Slide 42: Platform - KINect Innovation Engine
Type 1 active conformation 215 cysteine kinases Type 2 DFG out conformation 128 cysteine kinases Type 1.5 C-helix out conformation 68 cysteine kinases Concentrated effort in immunology: autoimmune disease and chronic inflammation Cysteinome targeted chemical library (60% of the kinome) Focused on a
Slide 43: Integrated drug discovery capabilities
Biomarker Assay Development Clinical Biomarker Assessment In vivo Efficacy and PK Studies PK/PD Relationship Release Assay Validation TRANSLATIONAL RESEARCH COMPUTATIONAL & MEDICINAL CHEMISTRY Leaders in Mechanistic Enzymology Custom Assay Development Compound: Target Interaction Enzyme Inhibitor Me
Slide 44: The Kinase Opportunity – Rational Targeted Drug Discovery
Creating New Medicines Targeting Previously Inaccessible Parts of the Kinome Kinase Drugs Represented $240B in Aggregate Global Sales from 2011-2015 Proprietary chemical library and integrated capabilities for interrogating the Kinome Solves challenges encountered in the class Selectivity Biochemica
Slide 45: Pipeline Targets
MK2: Innate immune response – clinically validated by Humira®, Actemra®, Kineret® ITK: T-cell receptor dependent autoimmune disease – clinically validated by Neoral®, Prograf®, Orencia® JAK: Inflammatory cytokine dependent inflammation – clinically validated by Xeljanz®, Jakifi® 46 Pipeline Targets
Slide 46: Antigen and Cytokine Receptor Signaling Inhibitors for Dermatology
Alopecia Areata: IFNg (JAK1/2) and IL-15 (JAK1/3) Vitiligo: IFNg (JAK1/2) and IL-15 (JAK1/3) Psoriasis: IFNg (JAK1/2), IL-12/23 (JAK2/Tyk2), IL-22 (JAK1/Tyk2) and IL-21 (JAK1/3) Atopic Dermatitis: IFNg (JAK1/2), TSLP (JAK1/2), IL-22 (JAK1/Tyk2) and IL-4/IL-21 (JAK1/3) All autoimmune disease driven b
Slide 47: Building a Fully Integrated Biopharmaceutical Company
48 Commitment to Patients Focus on “white-space” or underserved diseases where treatment gaps exist Research and Development Scientific leadership in dermatology and immunology- innovative clinical and regulatory strategies Innovative and differentiated clinical and regulatory strategies Executive T
Slide 48: Milestones 2018-2019
Milestone 2018 2019 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 A-101 SK Expected U.S. Launch Expected EU Approval A-101 Common Warts Phase 2 Data Initiate Phase 3 trial(s) Phase 3 Data File NDA ATI-50001/ATI-50002 (JAK Inhibitor) Initiate Phase 2 (ATI-50001) AA Trial Phase 2 (ATI-50001) AA Dose Range Data Phase 2 (ATI
Slide 49: Thank You
© Copyright 2017 Aclaris Therapeutics. All rights reserved. A-1 THANK YOU www.aclaristx.com
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