Aclaris Therapeutics, Inc. · NASDAQ:ACRS
Aclaris Therapeutics — Investor Presentation (May 2016)
33 slides from Aclaris Therapeutics's investor presentation, May 27, 2016
Slide 1: Corporate Presentation May 2016
© Copyright 2016 Aclaris Therapeutics. All rights reserved. A-1 1 May 2016 Corporate Presentation
Slide 2: Forward-looking statements and confidentiality notice
This presentation, the information contained herein and the materials accompanying it (collectively, this “presentation”) constitutes confidential information and is provided to you on the condition that you agree that you will hold it in strict confidence and not reproduce, disclose, forward or dis
Slide 3: The Aclaris Opportunity
The Aclaris Opportunity May 2016 3 Time and capital efficient Highly concentrated prescriber base Large unmet market segments with no FDA-approved drugs Growing market for cash pay aesthetic and medical dermatology products A-101: Proprietary formulation of high concentration H2O2 Seborrheic Keratos
Slide 4: Our Drug Candidates
Our Drug Candidates Exclusive, Worldwide Right to Commercialize All Current Candidates May 2016 4 Seborrheic Keratosis (topical) A-101* RESEARCH PRE-CLINICAL PHASE I PHASE II PHASE III Common Warts (topical) ATI-50001 Alopecia Areata (oral) ATI-50002 Alopecia Areata (topical) Also developing A-102 t
Slide 5: Seborrheic Keratosis (SK) Background
3 Months Post Cryosurgery Before Treatment SK is one of most common diagnoses made by dermatologists >83 million people with the disease in the U.S. 18.5 million patient visits to dermatologists 8.3 million procedures to remove SKs annually $1.2 billion - historic costs of treatments for SK Patients
Slide 6: A-101 is appealing concept for SK treatment
A-101 is appealing concept for SK treatment Topical, non-invasive Minimal discomfort; no need for anesthesia Reduced risk of pigmentary changes and scarring Ability to treat larger numbers of lesions Ability to hand off to ancillary staff Background Developed a proprietary formulation of 40.0% H2O2
Slide 7: Summary of Completed Phase 2 Trials for SK
Summary of Completed Phase 2 Trials for SK Trial SK Lesion Area Date Completed Trial Design Trial Outcome SEBK-201 (n=35) Phase 2 Trunk (Back) June 2014 Single center, intra-subject Four lesions treated A-101 concentrations: 25.0%, 32.5%, 40.0% 1 or 2 applications Duration: 78 days Efficacy: 32.4% c
Slide 8: A-101 Phase 2 Trunk/Extremities Study: PLA Responder Analysis
A-101 Phase 2 Trunk/Extremities Study: PLA Responder Analysis * 8 May 2016
Slide 9: A-101 Phase 2 Face Study: PLA Responder Analysis
A-101 Phase 2 Face Study: PLA Responder Analysis Percentage of Subjects With Target Lesion Clear Percentage of Subjects With Target Lesion Clear or Near-Clear * * * P-value<0.001 May 2016 9 * P-value<0.001 0.0% 3.0% 28.0% 46.0% 24.0% 60.0% 0.0% 10.0% 20.0% 30.0% 40.0% 50.0% 60.0% 70.0% Day 22 Day 10
Slide 10: Grading of SKs using PLA Scale in Clinical Trials
Grading of SKs using PLA Scale in Clinical Trials Subject: 04-52 Visit: 8 Date: 23Oct2014 Lesion: 3 H2O2%: 40.0 PLA: 1 Subject: 04-52 Visit: 2 Date: 07Jul2014 Lesion: 3 H2O2%: 40.0 PLA: 3 Subject: 04-52 Visit: 2 Date: 07Jul2014 Lesion: 4 H2O2%: 40.0 PLA: 3 Subj 04-92 Visit: 8 Date: 30Oct2014 Lesion:
Slide 11: A-101 Next Steps: Phase 3 Overview
A-101 40.0% is being used for Phase 3 clinical testing Initiated Phase 3 program – January 2016 Pivotal trials (SEBK-301/302): Two identical Phase 3 trials 4 lesions treated in total with at least one on face and one on trunk or extremities 400 subjects each Primary endpoint: Proportion of subjects
Slide 12: A-101 Commercialization Strategy
May 2016 12 A-101 Commercialization Strategy Cash pay, minimally invasive procedure Lower cost relative to other aesthetic treatments (Botox®, Fillers, Laser treatments) Buy and Bill Model 5,000 dermatologists in US, accounting for over 70% of procedures performed Concentrated call point allows for
Slide 13: ATI-50001/ATI-50002 Candidates for Alopecia Areata
ATI-50001/ATI-50002 Candidates for Alopecia Areata September 2015 CONFIDENTIAL 13
Slide 14: Alopecia Areata (AA) Background
AA is an autoimmune condition, characterized by patchy, non-scarring hair loss on the scalp and body Large unmet need: >6.6 million people in the U.S. have had or will develop AA at some point in their lives 2/3 of affected individuals ≤30 years old at disease onset 25-50% of patients have persisten
Slide 15: ATI-50001/ATI-50002: JAK Inhibitors in Alopecia Areata
Lead asset: Selective JAK 1/3 inhibitor from Rigel Exclusive, worldwide license and development collaboration Oral and topical rights Known mechanism of action and biological response in humans Promoted hair regrowth in mouse model of AA Drug Candidates: ATI-50001 for oral administration in Alopecia
Slide 16: Recent Business Development Transactions
Vixen (Columbia University IP) and Key Organics/JAKPharm Broadens our IP estate Methods of use covering JAK inhibitors for the treatment of: Alopecia Areata Androgenetic alopecia (female and male pattern hair loss) Additional hair loss disorders Next generation JAK inhibitors Covalently bound highly
Slide 17: Indication Expansion
December 2015 CONFIDENTIAL 17 Indication Expansion
Slide 18: Androgenic Alopecia
Androgenic Alopecia December 2015 CONFIDENTIAL 18
Slide 19: Androgenic Alopecia (AGA): Growing Market with Many Unmet Needs
An estimated 800,000 individuals are seeking some kind of treatment for hair loss worldwide 1 Women have been increasingly interested in solutions for AGA 2 The Rx treatment market was roughly $600M globally in 2013, and approximately $1.9B was spent on hair restoration surgery in 2012 3 There are t
Slide 20: Background: Hair cycle dynamics in AGA
In AGA, telogen (rest) becomes longer and anagen (growth) becomes shorter JAK-STAT signaling is elevated in telogen (resting) hairs Hypothesis:Treatment with a JAK inhibitor can start anagen (growth) Background: Hair cycle dynamics in AGA 20 May 2016
Slide 21: Inhibition of JAK-STAT signaling initiates anagen in normal mice
Inhibition of JAK-STAT signaling initiates anagen in normal mice Tofacitinib Vehicle SAG Ruxolitinib 7 days 14 days 21 days Category Differentially expressed in Ruxolitinib and Tofacitinib T0-T5, FC>1.5 or <0.5, P<0.05 P-value Melanogenesis Tyrp1, Syt4, Mlana, Pmel, Dct, Tyr Sos1, Dbf4, Pax3, PIK3ca
Slide 22: Dynamics of hair cycle
Dynamics of hair cycle In Androgenetic Alopecia (AGA), the duration of anagen decreases with each successive hair cycle, whereas the duration of telogen becomes prolonged. This leads to a reduction in the anagen-to-telogen ration and corresponds to periods of excessive hair shedding. Prolongation of
Slide 23: Vitiligo
Vitiligo December 2015 CONFIDENTIAL 23
Slide 24: Background on Vitiligo: Market is Large and Underserved
The global market for vitiligo was estimated at $1.4B in 2011 and is expected to reach $2.7B by 2019 4 There are no approved treatments and the pipeline is considered to be weak. Most commonly used medications are generic, used off-label and considered average efficacy 4 Key unmet needs in medicatio
Slide 25: Vitiligo: JAK Inhibitor proof-of concept case report
Vitiligo: JAK Inhibitor proof-of concept case report Tofacitinib Citrate for the Treatment of Vitiligo: A Pathogenesis-Directed Therapy Brittany G. Craiglow, MD; Brett A. King, MD, PhD JAMA Derm 2015;151(10)1110-2 Baseline: White macules and patches evident Baseline: numerous white macules & patches
Slide 26: Patient with coexistent vitiligo and alopecia areata
Patient with coexistent vitiligo and alopecia areata Rapid skin repigmentation on oral ruxolitinib May 2016 Baseline Week 4 Week 8 Week 12 Week 16 Week 20 Harris et al, JAAD 2016 26
Slide 27: Near-Term Milestones
Milestone 2016 2017 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 A-101 SK Phase 3 Trial Initiated Submit NDA A-101 Common Warts Phase 2 Trial Underway ATI-50001/ATI-50002 Alopecia Areata Submit IND Commence Clinical Trial May 2016 27 Near-Term Milestones
Slide 28: Thank You
© Copyright 2016 Aclaris Therapeutics. All rights reserved. THANK YOU www.aclaristx.com 28
Slide 29: Mechanism of action of the JAK inhibitors
29 Mechanism of action of the JAK inhibitors May 2016 Adapted from Gadina, Arth & Rheum V68, no.1 (2016) pp31-4
Slide 30: Interfering with the IFN-γ/CXCL10 pathway using JAK inhibitors
Interfering with the IFN-γ/CXCL10 pathway using JAK inhibitors to develop new targeted treatments for vitiligo May 2016 IFN-γ/CXCL10 signaling pathway in vitiligo. Binding of IFN-γ to its receptor (IFN-γR) activates the JAK-STAT pathway and leads to CXCL10 secretion in the skin. CXCL10 promotes recr
Slide 31: Androgenetic Alopecia scalp contains predominantly telogen hairs
Androgenetic Alopecia scalp contains predominantly telogen hairs May 2016 31
Slide 32: Inhibition of JAK-STAT promotes human hair follicle growth
Inhibition of JAK-STAT promotes human hair follicle growth May 2016 Day 0 Day 10 Day 30 Vehicle Tofacitinib Ratio of vehicle/tofacitinib = 1.57 Ratio of vehicle/tofacitinib = 3.13 Ratio of vehicle/tofacitinib = 2.26 Tofacitinib Ruxolitinib Vehicle 32 0 2 4 6 8 10 12 0 2 4 6 8 10 12 0 2 4 6 8 10 12
Slide 33: Inhibition of JAK-STAT promotes dermal papilla inductivity
Inhibition of JAK-STAT promotes dermal papilla inductivity May 2016 Phospho STAT 3 Phospho STAT 5 Isotype hDP spheres Control hDP spheres Tofacitinib N/S hDP spheres Ruxolitinib 33
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