Company Overview. Dr. Neal Walker President and CEO. September Copyright 2016 Aclaris Therapeutics. All rights reserved.

Similar documents
COMPANY OVERVIEW. Dr. Neal Walker President and CEO. January Copyright 2017 Aclaris Therapeutics. All rights reserved. A-1 1

COMPANY OVERVIEW. Dr. Neal Walker President and CEO. June Copyright 2017 Aclaris Therapeutics. All rights reserved. A-1

Innovation In Ophthalmics

Developing the next generation of dermatology products to treat serious skin diseases

Innovation In Ophthalmology

Photocure ASA Executing the Strategy

Dynavax Corporate Presentation

Utrophin Modulation: A Universal Treatment Approach to DMD. End Duchenne Tour April 2018

Diagnostics for the early detection and prevention of colorectal cancer.

Bank of America Merrill Lynch 2016 Health Care Conference

Phase 2b/3 Topline Trial Results

David Zaccardelli, PharmD Chief Executive Officer. J.P. Morgan 37 th Annual Healthcare Conference January 8, 2019

Diagnostics for the early detection and prevention of colon cancer. Publication of DeeP-C Study Data in New England Journal of Medicine March 2014

Company Overview. Dr. Neal Walker President and CEO January 2019 EMPOWERING PATIENTS THROUGH REVELATIONARY SCIENCE

Lehman Brothers Eighth Annual Global Healthcare Conference March 30, 2005 Bruce A. Peacock President and Chief Executive Officer

AM-125 : Intranasal Betahistine

Inarigivir ACHIEVE Trial Results and HBV Clinical Program Update. August 2, 2018

Photocure ASA Executing the Strategy

N A S D A Q : E V F M

Annual Stockholder Meeting May 30, confidently live life with ease

Jefferies 2016 Healthcare Conference. Reid Huber, PhD Chief Scientific Officer

Diagnostics for the early detection and prevention of colon cancer. Fourth-Quarter 2014 Earnings Call February 24, 2015

Phase 3c Topline Results. Page 1

Corporate Presentation August 6, 2015

PATENCY-1 Top-Line Results

New Ideas. Better Medicines. Third Quarter Financial Results Conference Call

SAVARA CORPORATE PRESENTATION (NASDAQ: SVRA) NOVEMBER 2018

Savient's Pegloticase Data in Treatment-Failure Gout Patients Presented at 72nd Annual Meeting of the American College of Rheumatology Conference

Amicus Establishes Gene Therapy Pipeline for Lysosomal Storage Disorders (LSDs) Conference Call and Webcast September 20, 2018

Tamsulosin Hydrochloride 0.4 mg Capsule

Lauren Silvernail, CFO & VP Corporate Development to present at 2005 Thomas Weisel Partners Healthcare Conference on September 7, 2005 at 3:50 pm EST

ALLERGAN TO ACQUIRE ZELTIQ FOR $2.47B BEST-IN-CLASS COMPANY IN FAST GROWING BODY CONTOURING SEGMENT

Rimegepant Pivotal Phase 3 Trial Results - Conference Call March 26, Biohaven

34 th Annual J.P. Morgan Healthcare Conference

SAKURA 3 Open-Label Phase 3 Safety Study with DaxibotulinumtoxinA for Injection (RT002) for the Treatment of Moderate to Severe Glabellar Lines

Merrill Lynch's Global Pharmaceutical, Biotechnology, and Medical Device Conference. February 7, 2007

ARDELYX REPORTS POSITIVE T3MPO-2 PHASE 3 TRIAL RESULTS IN IBS-C

Targeted Therapeutics for Inflammatory Disease

LEERINK GLOBAL HEALTHCARE CONFERENCE. Marino Garcia EVP, Chief Strategy Officer February 15, 2017

January 2017 Investor Presentation. confidently live life with ease

Acasti Pharma Provides Business Update for the Third Quarter of Fiscal 2019

MARINE Study Results

Forward-looking Statement Disclaimer

Forward-Looking Statements

Pierre Legault CEO June 2, 2014

35 th Annual J.P. Morgan Healthcare Conference

PRO 140. First self-administered antibody therapy for HIV in late-stage clinical development. March

Memory Pharmaceuticals Establishes Plans for Clinical Program for MEM 3454 in Schizophrenia. -Broadens Roche Nicotinic Alpha-7 Alliance-

Investor Presentation June 2012 NASDAQ: CEMI

First self-administered antibody therapy for HIV in late-stage clinical trials. CytoDyn Annual Meeting of Stockholders August 24, 2017

ADAPTIMMUNE INVESTOR PRESENTATION. August 2016

Corporate Presentation May Transforming Immuno-Oncology Using Next-Generation Immune Cell Engagers

Q1 Results 2018 Webcast presentation 26 April 2018

Anti-IL-33 (ANB020) Program

Idenix Pharmaceuticals Building a Leading Antiviral Franchise. Cowen & Company 27 th Annual Healthcare Conference March 13, 2007 Boston

Advancing Innovative Therapies for Neurological Diseases

AGM Presentation For the year to 30 September February 2016

Targeted Therapeutics for Inflammatory Disease Jefferies Healthcare Conference

Aquinox Q2 /2015 Conference Call: LEADERSHIP Secondary Endpoint Update - AQX-1125 in BPS/IC. August 6, 2015

Oncology Therapeutics without Compromise APRIL 2011

TSX Venture: RVV OTCQB: RVVTF

Breathtaking science. Developing respiratory drugs to improve health and quality of life. H.C. Wainwright Global Life Sciences Conference April 2018

Introduction. (Nasdaq : PLSE)

REPROS THERAPEUTICS. Dedicated to Treating Male and Female Reproductive Disorders. Corporate Presentation. August 2017

confidently live life with ease Management Presentation

DARA Reports Year-End 2012 Financial Results

37 th Annual J.P. Morgan Healthcare Conference. January 9, 2019

GW Pharmaceuticals plc. Investor Presentation August 2014

Business Update & Financial Results for Q1 2018

37 th ANNUAL JP MORGAN HEALTHCARE CONFERENCE

Diagnostics for the early detection and prevention of colon cancer. Leerink Swann Global Health Care Conference February 2015

ANCHOR Study Results Overview

REPROS THERAPEUTICS. Dedicated to Treating Male and Female Reproductive Disorders. Corporate Presentation. September 2017

BioCryst Pharmaceuticals

Theravance Announces Positive Results from Phase 1 and Phase 2 Clinical Studies with TD-1211 in Development for Opioid-Induced Constipation

Investor Presentation December The vision to see past tomorrow

N a s d a q : I N S Y

Press Release. RedHill Biopharma Announces Enrollment of Last Patient in the BEKINDA Phase II Study for IBS-D

OBALON THERAPEUTICS, INC. (OBLN) The first and only FDA-approved swallowable, gas-filled intragastric balloon for weight loss

What s New in Alopecia Areata

CytoDyn Announces Initiation of Metastatic Triple Negative Breast Cancer Trial and Reiterates Phase 3 Goal in Cancer

VivaGel BV. Product Information and Independent Market Research 30 APRIL 2018 STARPHARMA HOLDINGS LIMITED ASX:SPL; OTCQX:SPHRY

Building a Stroke Portfolio. June 28, 2018

Oragenics Shareholder Update

Committed to Transforming the Treatment Paradigm for Migraine Prevention

Cowen Investor Conference March confidently live life with ease

Investor Presentation

Jefferies Healthcare Conference

NASDAQ: ZGNX. Company Presentation. October 2017

AVEO Oncology Announces Strategic Restructuring. AVEO to Host Conference Call Wednesday, June 5 at 8:30 a.m. ET

AVEO and Astellas Report Final Overall Survival Results from TIVO-1

Cowen Annual Healthcare Conference. March 2018

Supernus Pharmaceuticals

Market Potential for MDX Alcobra Investor Breakfast July 15, 2014

Theravance Biopharma, Inc. (NASDAQ: TBPH)

Calliditas Therapeutics Q2 Report Webcast August 16, 2018, 10:00 Presenters: Renée Aguiar-Lucander, CEO Fredrik Johansson, CFO

Sumitomo Dainippon Pharma to Acquire Tolero Pharmaceuticals, Inc. (US Biotechnology Company)

Aviragen Therapeutics, Inc. and Vaxart Inc. Joint Conference Call UNLOCKING THE FULL POTENTIAL OF ORAL VACCINES

Supernus Pharmaceuticals

Transcription:

Company Overview Dr. Neal Walker President and CEO Copyright 2016 Aclaris Therapeutics. All rights reserved. A-11

Disclaimer This presentation contains forward-looking statements, including statements regarding the treatment and market opportunity for SK, common warts, alopecia areata, androgenetic alopecia, vitiligo, and the future operations of Aclaris. These statements involve substantial known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance or achievements to be materially different from the information expressed or implied by these forwardlooking statements. We may not actually achieve the plans, intentions or expectations disclosed in our forward-looking statements, and you should not place undue reliance on our forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements we make. The forwardlooking statements in this presentation represent our views as of the date of this presentation. For further information regarding these risks, uncertainties and other factors you should read Aclaris Annual Report on Form 10-K for the year ended December 31, 2015, Aclaris Quarterly Report on Form 10-Q for the quarter ended June 30, 2016 and Aclaris other filings it makes with the Securities and Exchange Commission from time to time. We anticipate that subsequent events and developments will cause our views to change. However, while we may elect to update these forward-looking statements at some point in the future, we have no current intention of doing so except to the extent required by applicable law. You should, therefore, not rely on these forward-looking statements as representing our views as of any date subsequent to the date of this presentation. This presentation also contains estimates and other statistical data made by independent parties and by us relating to market size and other data about our industry. This data involves a number of assumptions and limitations, and you are cautioned not to give undue weight to such estimates. In addition, projections, assumptions and estimates of our future performance and the future performance of the markets in which we operate are necessarily subject to a high degree of uncertainty and risk. 2

The Aclaris Opportunity MANAGEMENT TEAM EXPERTISE IN DERMATOLOGY DRUG DEVELOPMENT PIPELINE ATTRACTIVE DERMATOLOGY MARKETS Founded and sold several companies Directly relevant experience in Dermatology Board-certified dermatologists as CEO and CSO Developed and commercialized multiple products A-101: Proprietary formulation of high concentration H 2 O 2 Seborrheic Keratosis Phase 3 Data 4Q 2016 Common Warts Phase 2 Data 3Q 2016 ATI-50001/ATI-50002/ATI- 50003: JAK 3 and 1/3 Inhibitors Alopecia Areata Vitiligo Androgenetic Alopecia (AGA) Time and capital efficient Highly concentrated prescriber base Large unmet market segments with no FDAapproved drugs Growing market for cash pay aesthetic and medical dermatology products Build a Fully Integrated Dermatology Company 3

Our Drug Candidates Exclusive, Worldwide Right to Commercialize A-101, A-102, ATI-50001, ATI-50002, and ATI-50003. A-101 Seborrheic Keratosis (topical) RESEARCH PRE-CLINICAL PHASE I PHASE II PHASE III Common Warts (topical) ATI-50001 Alopecia Areata (oral) ATI-50002 Alopecia Areata (topical) ATI-50001, ATI-50002 Vitiligo (oral, topical) ATI-50003 Androgenetic Alopecia (topical) Also developing A-102 topical gel as a lifecycle management opportunity for A-101 4

Seborrheic Keratosis (SK) Background 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 Untreated SK Patients seek diagnosis and treatment Fear of skin cancer Concern about appearance Discomfort from itching and inflammation Before Treatment 3 Months Post Cryosurgery Current options for SK removal: cryosurgery, curettage, electrodessication or excision Limitations of current removal options: Dyspigmentation (hypo or hyper) Scarring Pain Surgical - invasive Treatment of numerous SK is impractical 5

Potential to Be First FDA-approved Drug for SK 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 UNTREATED TREATED Background Developed a proprietary formulation of 40.0% H 2 O 2 Conducted formal dose-ranging studies MOA: drives apoptotic and necrotic cell death Inventor s Proof of Concept (with his initial formulation) 6

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% clear; 67.7% clear or near clear with 40% concentration Favorable safety profile SEBK 202 (n=172) Phase 2 Trunk and Extremities December 2014 Multicenter, parallel group Four lesions treated A-101 concentrations: 32.5%, 40.0% 1 or 2 applications Duration: 106 days Efficacy: Demonstrated statistically significant clearance of all 4 lesions in top dose group (Phase 3 primary end point) Favorable safety profile SEBK 203 (n=119) Phase 2 Face March 2015 Multicenter, parallel group One lesion treated A-101 concentrations: 32.5%, 40.0% 1 or 2 applications Duration: 106 days Efficacy: Statistically significant clearance Favorable safety profile 7

A-101 Phase 2 Trunk/Extremities Study: PLA Responder Analysis Percentage of Subjects Achieving Total Clearance Percentage of Subjects Achieving Total Clearance or Near Clearance 70.0% 60.0% 50.0% 20.0% 15.0% 10.0% 14.3% 16.1% 19.6% 40.0% 30.0% 20.0% 32.6% 42.4% 58.9% 5.0% 10.0% 23.7% 0.0% 5.4% 0.0% 0.0% Day 22 Day 106 P-value<0.01 0.0% Vehicle (n=58) A-101 32.5% (n=57) 10.1% 1.8% Day 22 Day 106 P-value<0.0001 A-101 40.0% (n=57) 8

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 70.0% 60.0% 50.0% 70.0% 60.0% 50.0% 40.0% 30.0% 20.0% 10.0% 28.0% 24.0% 46.0% 60.0% 40.0% 30.0% 20.0% 10.0% 33.3% 45.9% 61.5% 67.6% 0.0% 3.0% 0.0% Day 22 Day 106 0.0% 2.5% 5.0% Day 22 Day 106 P-value<0.001 P-value<0.001 9

Grading of SKs using PLA Scale in Clinical Trials Pre-Treatment with A-101 Post-Treatment with A-101 Subject: Subject: 04-52 04-92 Visit: Visit: 2 Date: Date: 17Jul2014 07Jul2014 Lesion: 1 Lesion: H 3 2 O 2 %:40.0 H PLA: 2 O 2 %: 3 40.0 PLA: 3 Subject: 04-52 Visit: Subj 8 04-92 Date: Visit: 8 23Oct2014 Date: 30Oct2014 Lesion: Lesion: 3 1 H 2 O 2 H%: 2 O40.0 2 %:40.0 PLA: PLA: 1 0 Subject: 75 Subject: Visit: 104-52 Visit: Date: 2 25Nov13 Date: Lesion: 2 07Jul2014 H 2 O 2 %:32.5 Lesion: PLA: 24 H 2 O 2 %: 40.0 PLA: 3 Subject: 75 Visit: 9 Date: 18Feb14 Lesion: 2 H 2 O 2 %: 32.5 PLA: 1 10

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 Primary endpoint: Proportion of subjects with clear on PLA scale 3 month drug-free follow-up Open-label (SEBK-303): 4 SK lesions Up to four applications Phase 3 Data - 4Q 2016 Plan to submit NDA 1Q 2017 11

A-101 Commercialization Strategy Buy and Bill Model Cash pay, minimally invasive procedure Lower cost relative to other aesthetic treatments (Botox, Fillers, Laser treatments) Concentrated Prescriber Base 5,000 dermatologists in US, accounting for over 70% of procedures performed Concentrated call point allows for high reach and frequency Disease Awareness Disease state awareness initiatives KOL engagement, conference presentations and publications Commercial Launch 50-60 person specialty sales team focused on high tier targets Comprehensive promotional campaign to include peer-influence programs Patient Engagement Campaigns focused on driving awareness and furthering interest in treatment options 12

A-101 CANDIDATE FOR COMMON WARTS 13

Existing Patient Base Offers Significant Market Potential 2 million patients seek treatment from physicians for common warts 1-43% of patients have more than one wart 2 59% of patients see a dermatologist 1 25% see a pediatrician 1 - Patients with warts have higher risk of developing new warts 3 11% see a family/general practitioner 1 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; 273 293. 14

Summary of A-101 Phase 2 Wart Clinical Trial Results Trial Common Wart Area Topline Data Trial Objective and Design Trial Outcome WART 201 (n=98) Phase 2 Trunk and Extremities August 2016 Multicenter, parallel group One wart treated A-101 concentrations: 40%, 45% compared to vehicle 8 applications Duration: 56 days Efficacy: Statistically significant clearance with 45% concentration Favorable safety profile Primary Endpoint: Mean change from baseline in the Physician s Wart Assessment (PWA) score at Visit 10 using a analysis of covariance Secondary Endpoints: Responder analysis: The proportion of subjects whose target wart is judged to be clear on the PWA at Visit 10. Responder analysis: The proportion of subjects whose target wart is judged to be clear or mild on the PWA at Visit 10. 15

Both Statistical and Clinical Significance Achieved on Primary Endpoint with A-101 45% Concentration MEAN CHANGE FROM BASELINE IN THE PWA SCORE AT VISIT 10 0 Baseline Day 7 Day 14 Day 21 Day 28 Day 35 Day 42 Day 49 Day 56 0-0.06-0.2-0.4-0.6-0.15-0.23-0.23-0.5-0.27-0.31-0.52-0.17-0.47-0.58-0.3-0.3-0.5-0.55-0.41-0.53-0.41-0.44-0.8-1 -0.69 p < 0.05-0.81-0.87 p < 0.01-1.0-1.2 A-101 45% A-101 40% Vehicle 16

Statistical Significance Achieved on Secondary Endpoints in Clearance of Common Warts with A-101 45% Concentration Responder Analysis PROPORTION OF SUBJECTS ACHIEVING WART CLEARANCE AT VISIT 10 PROPORTION OF SUBJECTS ACHIEVING CLEAR OR BARELY EVIDENT ON PWA AT VISIT 10 30 25 25.8 45 40 35 41.9 20 30 15 10 25 20 15 15.6 14.8 5 3.1 3.7 10 5 0 P- value = 0.02 0 A-101 45.0% A-101 40.0% Vehicle P- value = 0.02 17

Patient Treated with A-101 45% Concentration in study WART-201 Pre-Treatment with A-101 Post-Treatment with A-101 Subject: 04-52 Visit: 2 Date: 07Jul2014 Lesion: 3 H 2 O 2 %: 40.0 PLA: 3 Visit 2 (PLA 3) Visit 10 (PLA 0) 18

A-101 Solution 40% A-101 Solution 45% A-101 Solution Vehicle A-101 Solution 40% A-101 Solution 45% A-101 Solution Vehicle A-101 Solution 40% A-101 Solution 45% A-101 Solution Vehicle A-101 Solution 40% A-101 Solution 45% A-101 Solution Vehicle A-101 Solution 40% A-101 Solution 45% A-101 Solution Vehicle A-101 Solution 40% A-101 Solution 45% A-101 Solution Vehicle A-101 Solution 40% A-101 Solution 45% A-101 Solution Vehicle Skin Reactions Similar to Vehicle at Visit 10; Favorable Safety Profile 100% 90% 80% 70% 60% 50% 40% 30% 20% 10% 2 1 0 0% Edema PRE Erosion PRE Erythema PRE Excoriations PRE Scabbing PRE Ulceration PRE Vesicles/Bullae Vescicles/Bullae PRE No Reaction Mild Moderate 19

Based on Results, A-101 45% Concentration Considered for Further Development as Treatment for Common Warts 45% Concentration of A-101 Observed to be Safe and Effective Statistical Significance Achieved both statistical and clinical significance on the primary endpoint Achieved statistical significance in complete clearance of the warts Safety Profile Favorable safety profile was observed under the conditions of this study Occasional mild, transient local skin reactions observed during treatment; skin reactions were similar to vehicle Next steps Develop A-101 45% Concentration as the commercial dosage form for common warts Develop as RX drug for patient to use at home 20

ATI-50001/ATI-50002 Candidates for Alopecia Areata 21

Alopecia Areata (AA) Background AA is an autoimmune condition, characterized by patchy, nonscarring 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 AA Patchy 2/3 of affected individuals 30 years old at disease onset 25-50% of patients have persistent patchy AA 14%-25% of patients progress to totalis or universalis Current off label treatments include topical steroids, steroid injections, and minoxidil Recent translational research work by Dr. Angela Christiano Furthered genetic understanding of disease Identified JAK inhibitors as a potential treatment for AA Alopecia Universalis 22

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 Totalis and Alopecia Universalis ATI-50002 for topical administration in Patchy Alopecia Areata Development Strategy Planned submission of IND: 2H 2016 Initiation of clinical trial: 1H 2017 23

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 selective JAK3 inhibitors 24

Additional Potential Indications Androgenic alopecia (male and female pattern hair loss) AGA is the most common cause of hair loss and is 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 Topical JAK inhibitor Male with AGA Vitiligo Vitiligo impacts 1% to 2% of the overall global population irrespective of sex, race, or age 3 Disease onset occurs in about one-half of sufferers between the ages of 10 and 30 3 Oral and topical JAK inhibitor Female with AGA 1 Cassiopea. Androgenic Alopecia. 2 Bergeson, L. The Truth About Hair Loss and Baldness Cures. 11.08.2014. 3 Fitzpatrick T., et al. Vitiligo Facts. American Vitiligo Research Foundation Inc. 25

Milestones Milestone 2016 2017 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 A-101 SK Phase 3 Trials Initiated Phase 3 Data Submit NDA Submit MAA A-101 Common Warts Phase 2 Data ATI-50001/ATI-50002 Alopecia Areata Submit IND Commence POC trial 26

THANK YOU www.aclaristx.com Copyright 2016 Aclaris Therapeutics. All rights reserved. 27